clinical research center business plan

Writing Business Plans for a Life Science Startup or Clinical Program

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The business plan is an important tool for raising capital, finding strategic partners, recruiting, and providing an internal guide on how to drive a company’s growth.

The plan should include an executive overview, introduction to the management team, market and competitive analyses, value proposition, operating plan, financial projections, and potential risks.

The plan should be concise, well written, and dynamic. Details behind key assumptions should be included.

Common business plan pitfalls include focusing only on the product without framing it in the context of the consumers/patients, the market dynamics, and the ecosystem in which it will be launched, as well as giving financials that are too aggressive and precise given the stage the company is in.

New founders should consider engaging experts to help test assumptions as they develop the key parts of the business plan, including the financial projections.

Many of the same concepts for writing a business plan for a startup apply to creating a business plan for a new clinical program or expanding operations within a health system.

Introduction

Building a life science startup is a long and complex endeavor, and the skills required are very different from the knowledge and training that academic scientists undergo. The process of developing a business plan ensures the team has tested their vision/strategy, and the plan can be used as a roadmap to guide their operations. It also serves as an important communication document when seeking investment in the business. Topics familiar to experienced grant writers—such as the significance, innovation, and approach topics from National Institutes of Health (NIH) grant applications—are relevant and necessary for framing the business plan. The plan also helps to keep the management team accountable, but sage entrepreneurs will recognize that they will often have to revise the original plan over time, based on clinical trial results, regulatory milestones, and market changes. There are several kinds of business plans, generally described as either one-page plans (business model canvas or lean launchpad) and traditional, full-length plans. While one-page business plans are a useful and simple tool for rapid, early iteration, the startup team will ultimately have to write a more detailed plan to secure funding (“ Business Model Canvas ”'; Osterwalder and Pigneur ). In this chapter, we focus on the later and will outline the key elements of a full-length biomedical business plan, highlight the pitfalls to avoid, and provide potential resources for new founders to get their plans started.

It is important to note that developing a sound business plan is also highly relevant for supporting smaller-scale clinical programs, operational investments, and intrapreneurship efforts (see the chapter on " Intrapreneurship: Strategic Approaches for Managing Disruptive Innovation in Your Clinical and Research Projects "). Please see the section at the end of the chapter that outlines the key differences for these types of business plans.

Key Elements of a Business Plan

A) executive s ummary.

The executive summary is a one to four page overview of the existing problem/need in the market and how the new product or service has a unique value proposition that addresses that need (Markowitz) . It is the reader’s first impression of the business, and investors often read only the summary, so it is important for it to be compelling ( Figure 1 ). Consider it a concise but more formal “elevator pitch” that highlights what the company is and why the product or service will win in the market (Cohen) . The remainder of the executive summary should include the short- and long-term goals, key points in the strategic plan, the business model, summary of financial projections, and information about employees and location (Valentin) .

Elements in an Executive Summary.

B) Management team and advisors

This section introduces the management team, their backgrounds, and how their expertise aligns with their particular roles in the business. In addition to the management team, there should be a Scientific Advisory Board (SAB), which helps to guide technology decisions, and a Board of Directors (BoD) which helps to guide all key decisions for the company. SAB and BoD members should be chosen based on their ability to provide industry knowledge and key industry connections that can help the company grow. Having industry experts involved in the company gives assurance—especially to investors—that the technology and operations have been vetted and are supported by those who know the market well.

C) Market Overview

This section should describe the overall landscape of the market, including the size of the market, key segments, historical and expected market growth, and key drivers or trends that may impact the problem the startup is trying to solve.

Market size estimates for life science businesses often include the incidence and prevalence of the disease/condition related to the problem, the estimated medical cost burden—in the U.S. or globally—associated with the condition, and the estimated cost for the consumer/patient with the current available solutions (see the chapter “ Conducting Insightful Market Research ”). It may be useful to describe any relevant preclinical and clinical data that support why this problem exists and highlight the market need. While a large market size is usually viewed positively, it is important that the information provided focus on the market size relevant to the startup’s specific solution. For example, a business plan for an intervention to treat prescription opioid addiction should include details about the global market for opioid drugs, the most recent trends and the expected growth rate of prescribed opioid use and abuse, the morbidity and mortality associated with opioid abuse, and the estimated annual cost for the treatment of opioid abuse and addiction, including the subsequent medical and mental health costs. To demonstrate market size in this example, estimates would include the number of emergency department visits, hospitalizations, intensive care unit admissions, and procedures for overdose or opioid abuse–related complications, as well as the current number, length, and cost of drug rehabilitation admissions.

In terms of the current market, it is useful to describe how the market is divided into customer/patient segments, which may be by geographic region, demographics, psychographic, or customer type (in healthcare, this might be pediatric vs. adult, or inpatient hospital vs. outpatient setting), among others, who are using a certain type of product/service. In the above example of the opioid market, there is segmentation in application (pain relief, anesthesia, cough suppression, diarrhea suppression, or treating addiction) and by geography (North America, Europe, Asia-Pacific, South America, and the Middle East/Africa, which can be further broken down by key countries), which can then be mapped to the types of opioid products with different mechanisms of action (for example, short acting vs. long acting opioids). If the market has clear customer/market segments, there are likely different drivers of demand in each of them, which should be well understood since the startup will want to provide product/service offerings that meet the needs of those segments. Describing the market structure, how this market is segmented, and projected growth rate of target segments will make it easier to determine which segments are the most valuable and to describe how they will be targeted.

As the market data are outlined, it is important to tie back to the product/service offering and how it is uniquely positioned to fulfill the unmet need(s) within the particular target markets (see the chapter “ Identifying Unmet Needs: Problems that Need Solutions ”). Use market research, economic trends, and even patient and provider behaviors, if appropriate, to determine what sector(s) of the market the product fits best. Understand the needs of patients, providers, and hospitals and why the startup’s strategy will meet these needs, in order to better prepare the marketing plan for the product. Moreover, it is important to highlight the attributes of the market that support the offering, such as a large addressable market size, rapid growth in the market segment(s) of interest, and/or the level of competition in these segments. These are dimensions that are critical to stakeholders when they evaluate the business plan.

D) Competitive Landscape

The problem the startup is seeking to solve is one that multiple incumbents are likely already addressing today, either directly or indirectly. This section should provide an overview of the current offerings in the market, where they fall short, and how the new offering fills a current gap in the market. In the above sections, the problem should have been outlined and framed in a way that there would be critical dimensions that matter to stakeholders (patients, physicians, providers, payers). It would be helpful to describe how these competitive offerings compare across these dimensions, which sets up the next section on value proposition.

Understanding the competitors’ product and services, market share, current and past strategies, strengths and weaknesses, the threats they pose to the startup, and the opportunities they make available are integral to a thorough and useful analysis of the competition. This is not just an exercise to learn about other businesses; it will also help identify the strengths and weakness of the startup’s business strategy (see the chapter “ Startup Company Formation and Management ”). Consider developing a basic profile of each of the current direct competitors in the market with these characteristics and include it in the Appendix.

Typical sources of information are company websites and marketing materials (Hisrich et al.) .  Academic libraries can also provide a wealth of information through their subscriptions. Other helpful strategies include browsing media outlets for press releases and public relations information, social media, and former customers’ testimonials on how they perceive the competition.

E) Value Proposition

Building off the market need and competitive landscape analysis, the value proposition of the startup’s solution should be articulated in this next section. Against the dimensions that matter to stakeholders, this section of the plan should describe how the new solution will outperform the competition. The value proposition statement is a key way to succinctly demonstrate the measurable benefit that the patient or provider would get from the new product or service, and why patients or health care providers would choose it over existing solutions. Money savings, time, and convenience add to the value of a product. It is important to explain this in a way that can be understood by both scientific and nonscientific audiences. Describe the product/service without revealing too much proprietary information since the business plan may be distributed beyond the intended recipients.

The research results that led to the development of the new product should be shared. This may include pilot data, preclinical/animal model studies, and/or clinical trials, depending on what stage of testing the product has undergone (see the chapter “ Pre-Clinical Animal Models ”). Provide preliminary data and reference specific publications that support the product. In addition, any results of prototype testing should be included.

In the life sciences realm, even if the product solution meets the needs of a patient, ensuring that it fits into the medical ecosystem is imperative. Understanding the infrastructure of a hospital, including the physicians, the administrators, the insurance payers, and whether or not the new offering will improve a patient’s quality of life or improve outcomes such that payers will reimburse the startup for its technology is critical to success. It is also important to articulate (if applicable) whether or not the offering can be dropped into existing treatment algorithms/processes or if changes will need to be made to how work is done to adopt the solution. If a lot of re-training or adjustments around the rest of the ecosystem are required, the value proposition will be more challenging since a lot of changes will need to be made to adopt the solution.

F) Operating Plan

With the market and competitive landscape outlined, and the company’s value proposition defined, this section should describe how the company will execute to capitalize on the opportunity. The operating plan should begin with a thorough explanation of the business model—how the company will work successfully with clients, suppliers, manufacturers, and partners to generate profit. Include here the organizational structure of the company. Next, describe all critical technical, regulatory, and strategic milestones. Finally, outline any functional details about daily execution (Friend and Zehle) .

There are numerous business models, and any one industry may have several examples of successful companies using different approaches. For example, is the company going to adopt an integrator model, where they will build out everything needed to launch the offering, or will they adopt an orchestrator model, where they will partner with people for certain core competencies (e.g., manufacturers) to bring the solution to market? The operating plan should describe the selected model and explain why it is preferable to alternatives. Referencing the leading players highlighted in the competitive landscape section and contrasting against their business models may also be helpful.

Healthcare businesses must deal with reimbursement, fee schedules, billing systems, managed care contracts, and licensing, along with operational issues. The operating plan must address how these challenges will be handled and how the company will get paid, either through insurance reimbursement, by employers, on a fee-for-service basis, or directly by consumer payments. The long sales cycle in most health care businesses is particularly challenging for startups and requires keen long-term planning.

Reimbursement by third-party payers to hospitals and physicians is one of the determinants in whether or not a product will ultimately make it to market, whether it will be used by healthcare providers and patients, and how accessible the product will be. For most technology in healthcare, the payers account for most of the purchasing. Understanding the payers’ reimbursement process, their reimbursement terms, their method for determining the amount to be paid to the provider, and their policy on out-of-pocket cost sharing with the patients is integral as the reimbursement method will impact return on investment for the business (see the chapter “ Reimbursement Strategies and CPT Codes for Device Development ”).

After establishing the business model, it is important to provide an overview of the significant milestones the company foresees. Include any remaining technical development goals, any regulatory approvals the company will face, and other strategic imperatives, such as licenses to related technology, critical partnerships, or protecting intellectual property. Biotechnology and biomedical devices may also need to go through extensive regulatory and legal processes before approval. These processes are outlined elsewhere in this textbook (see the chapter “ FDA Device Regulation: 510(k), PMA ” and, “ FDA Drug Regulation: Investigational New Drug Applications ”).

Each of these milestones should include a description of the task, due date, budget, and responsible person. Due dates and budgets should be ranged since it is difficult to have 100% clarity; milestones function as the management team’s commitment to investors, and the company’s ability to complete these goals will be assessed.

Young businesses should also provide details about the market entry strategy to penetrate the targeted market effectively and to reach revenue and profit expectations. To develop this strategy, engaging with and understanding the ecosystem early on can help improve the design of the new offering and ensure that the solution can be reimbursed. Ideally, during this process one should meet with representatives across the ecosystem to understand what they care about and what the startup will need to deliver in order for them to embrace the new offering.

The technical side of the operating plan should include tactical steps and a timeline for implementing the plan and making the business operational. As a reference point, mention what has been done thus far. Explain how the business will operate, describing the current production process but also the planned process once the company is at scale. Include high-level details about labor, materials, technology, facilities, equipment, manufacturing processes, distribution plan, supply chain, and quality-control measures.

G) Financial Projections

In this section, the team must articulate the financials of the company and show that they have a solid understanding of their expenses, future revenue, and the projected timeline for achieving revenue goals. There will be many assumptions that go into these estimates, so it is important to provide ranges and to explain the assumptions behind the projections. Potential investors will review them to gauge the robustness of an entrepreneur’s understanding of the challenges that lie ahead.

A startup’s financial statements should detail the anticipated financial performance over time (for example: expenses, assets, liabilities, and working capital). Since the financial performance of the startup is dependent on future events (e.g., regulatory approvals or clinical trial partnerships), the financial projections will likely need to be in the format of a ‘pro forma’ budget, which projects future revenues and expenses based on a set of assumptions. Projections of financial statements should go far enough into the future to help readers see where the business can go when it matures or reaches an exit point. Outlined below are the three major parts of the financial plan ( Kolchinsky ; Friend and Zehle ).

The income statement shows the revenue, expenses, and profit for the business over a specific time period. If there are multiple sources of revenue, these may be itemized for future comparisons over time. Early on in the business development, this may be generated monthly and eventually quarterly or annually. An income statement showing earnings before interest, taxes, depreciation, and amortization (EBITDA) acts as a frequent proxy for a cash flow statement.

A statement of cash flow projections shows what the company expects to bring in and how much it will be spending each month. This includes tracking the cash revenues and cash disbursements for the month, and reconciling these two against the opening balance from the previous month. It is important to demonstrate that the startup can adhere to a budget and not overspend consistently. Thus, conservative estimates are preferred; this will increase the level of expenses, but the company should be able to justify why those expenses are needed. Additionally, the statement should show how much working capital the startup needs to pay the bills early on, and how long it will take to have a positive cash flow (bringing in more money than the company is spending). By estimating conservatively, the team can ensure that the company will have enough financial support (i.e., runway) to achieve the milestones without falling short of cash and going bankrupt.

The balance sheet highlights any major working capital requirements and includes assets, liabilities, and equity. Rather than showing trends, the balance sheet reflects these as of a set date.

The additional components of the financial plan detail how the company expects to make money selling the product. These include:

the cost of the product, what factors go into the unit cost, and the plans for bringing these costs down when the company is at scale;

cost estimates for equipment, facilities, inventory, and day-to-day operations, including salaries;

the price the company expects to receive for the product and why it is achievable, especially in the context of reimbursement.

Due to the long process of taking a product to market in healthcare businesses, there should be a section in the financial plan about the capital required for the various regulatory milestones. As outlined in the operating plan, all product development, technical, and regulatory milestones would come with an estimated budget and timeframe for completion. Included in this is the cost of the Food and Drug Administration’s drug review process—a major consideration in the financial projections for life science startups. From preclinical research to the Investigational New Drug application, to clinical trials, to the New Drug Application submission and review, this process is expensive and takes many years to complete. How far along a product is in this process will factor greatly into how much money will be necessary to complete Phase 1, 2, and/or 3 trials, as well as the subsequent regulatory requirements.

Clinical trials can incur substantial costs beyond distributing the study drug/device and the associated study procedures. Site costs, fees for storage, technology solutions, and safety monitoring, core lab fees, and study staff salaries—for scientists, physicians, project managers, data managers, research coordinators, biostatisticians, and site management, including regulatory visits and investigator meetings—all must be budgeted into the cost of each phase, and the timeline to complete the data collection should be considered. In addition, up to 30% of any grant funding may need to go toward administrative overhead to carry out the study, if implemented at an academic medical center. In some cases, academic entrepreneurs can establish sponsored research agreements that allow components of the preclinical or clinical research to occur at their university (see chapters on “Post Alliance Agreements and Sponsored Research Agreements” and “ Understanding Conflict of Interest for Academic Entrepreneurs ”). As mentioned earlier, many of these projections may be pro forma because they will rely on the achievement of other milestones prior to implementation.

H) Risks/Anticipated Problems

All business plans should include a section on anticipated risks/problems and potential alternative strategies. This can demonstrate to investors that the entrepreneur has thought through potential challenges and has plans to either prevent them from occurring, or backup plans to mitigate the consequences. Generally speaking, a balanced approach is helpful here—not hiding or obfuscating major challenges, especially those that have befallen other companies, but at the same time not overwhelming the reader with negativity. At the very least, this can be a thought exercise for the startup and may identify issues that had not previously been considered. While a business plan is not a legal document or binding contract, intentional distortion of facts can come back to haunt a company.

Business Plans for Clinical or Operational Programs

There will be many situations where an idea is not yet at the stage of becoming a company, but requires investment to drive growth or sustain operations. Examples include developing a new clinical program or service (e.g., a novel surveillance protocol postsurgical intervention that will result in diagnostic testing revenue) or expanding operations (e.g., building a new facility to treat patients who have an eating disorder). These situations also call for business plans in order to garner support and investment—in this case, the investment would be coming internally from the health system or institution rather than outside investors.

The key components of these business plans are executive summary, background, proposal description, market and competitive analyses, operating plan, metrics for success, financial projections, and potential risks. Many of the same concepts from earlier apply, but the key differences include: 1) the business plan should specify the dollar amount of the resources needed to make the plan operational; and 2) the proposal description, operating plan, financial projections, and risks should take into account the impact of the plan on the existing operations of the system.

As mentioned earlier, most health systems will have internal strategy and finance teams, which are helpful resources for developing business plans and should be consulted early on to help with the financial projections as well as the anticipated system impacts of the plan. The remainder of this section outlines each part of the business plan and focuses on the key differences from a startup business plan.

Executive summary: In addition to the points covered in the startup executive summary, the executive summary for a new program/service/facility should include the “ask”—what resources (e.g., capital, new full-time employees, or other operating expenses) are being requested to support the new program. The executive summary should also state the expected financial return on the investment from the perspective of the institution (usually in terms of annual steady-state contribution margin or total incremental contribution margin over a period of time, usually seven years).

Background: This section should describe the current state of operations. If the authors are proposing a new clinical program, for example, they should describe the patient population being addressed, how they are currently being served, and the current volumes. It should also describe the limitations of the current state, the unmet need, and what factors necessitate a new solution.

Proposal description: The authors should describe what new program/service/facility is being proposed, and how it addresses the current challenges. This section should also describe how the proposal will lead to growth—will the program reach a new patient population not previously served? Have a greater geographic reach? Result in greater utilization of other services at the institution? This is also an appropriate place to describe other benefits of the plan, including improved quality, safety, patient experience, efficient resource utilization, etc. It is important for this section to align with the institution’s priorities.

Market and competitive analyses: This section should look very much the same as described above in the startup business plan.

Operating plan: This section should build on the proposal description and go into more technical detail. Details should include how the program will be staffed, what type of services will be delivered, where they will be delivered, hours of operation, pricing and reimbursement, and what the impact will be on downstream services and the rest of the institution. Thus, it requires a detailed understanding of how the institution currently operates, so the new proposal can realistically be integrated without excessive disruptions to operant workflow.

Metrics for success: The authors should list two–three measurable metrics that will show the success of the program. These can include volume, financial, patient/staff satisfaction, or other related metrics. Ideally, there should be targets for each metric (e.g., “increase volume by 10% above baseline by year 3”). For plans where measurable impact will not be realized until several years out, milestone-based targets are also acceptable as near-term goals (e.g., “achieve regulatory approval by X date”). It is preferable that these metrics be ones that the institution already monitors, thus enhancing alignment with current priorities and facilitating the ability to add this new project. In some cases, though, it may be necessary to develop new metrics, which should be done in consultation with the institution’s leadership.

Financial projections: This section should include all components outlined above, with the exception of a balance sheet, since the plan is only describing a subset of operations within a larger system. For the pro forma income statement, it is useful to show multiple views: a base case view (if applicable), which shows current operations of the program/facility; an incremental view, which just shows the incremental revenue and expenses associated with the proposed plan; and a strategic view, which is the addition of the base case and incremental. It is also highly recommended to have a separate section outlining the assumptions used to develop the financial projections (e.g., data source, patient population, growth projections, operational start date and ramp-up speed). As these business plans are usually in the context of a large academic institution, it is helpful for planning purposes for the financial statements to specify the organizational entity to which the projected revenues and expenses will accrue (e.g., Hospital vs. Practice Plan vs. Research).

Risks and mitigation: This section should look very much the same as described above in the startup business plan. In addition, the plan should consider potential risks/negative impacts to other parts of the institution. For example, if the plan proposes to build a new facility in a suburban location, will it potentially cannibalize volume from the main location, and if so, what is the plan to mitigate revenue loss?

The same tips on business plan writing apply here. Since clinical and operational programs need to function within the environment of an existing system, socializing the plan early and often is especially critical for gaining support, both for funding the plan and for implementing it. In some cases, this may involve obtaining regulatory approval from internal institutional entities, such as the institutional review board (IRB), legal counsel, or information services (especially if information protected under the Health Insurance Portability and Accountability Act (HIPAA) is involved). Factoring time for these approvals is crucial.

External Resources

In academic entrepreneurship, there are a host of extra challenges that must be considered, including who owns the intellectual property, technology transfer agreements, regulatory procedures, and conflict of interest, among others (Kolchinsky) . At the same time, being part of an academic institution provides many resources not available to non-academic entrepreneurs, including business planning, legal, and funding support. Networking and taking advantage of other schools within the academic system who have experts in these fields will improve the entrepreneur’s knowledge, especially when entrepreneurship is not one’s first career or when this is one’s first experience with developing a product. This can be particularly valuable when estimating costs related to preclinical and clinical research. Many academic centers have clinical research offices that have pre-populated budget templates, which can help to ensure that all aspects of the clinical trial are adequately budgeted. Most academic systems also have corporate Strategy and Finance teams that can help with business analyses as well, such as market/competitive dynamics and projecting future demand (whether that be in terms of patient volume, demand for the product, etc.). In addition, university libraries may provide online access to publications and market research related to the product and the industry. For specific tasks, such as preparing an exit strategy that involves an initial public offering (IPO), extensive external legal and financial consultations are generally required.

Tips on Business Plan Writing

Remember that a business plan is dynamic, changing with the growth of the company and as the company pivots due to unexpected challenges or market trends. The plan should be comprehensive, and key points, data, and strategies should be consistent throughout. Be mindful of including important details without going into excessive minutiae. Lastly, this is a professional document, and it can be the first and lasting impression of the company on potential investors, so appearance matters. To this end, recruit trusted editorial support to make sure the plan is consistent from beginning to end and to check for correct spelling, grammar, and formatting.

Writing a business plan is a process. It requires brainstorming, researching, prewriting, drafting, revising, and editing. The business plan is not only an internal company tool for thinking about the future, it is also about presenting the company’s idea, solution, marketing strategy, financial projections, and long-term vision to prospective investors. It is an introspective exercise that each executive team should utilize to help identify misconceptions before they become costly, to organize and streamline team members’ efforts, to focus on the big picture rather than getting caught up in short-term actions, and to establish performance standards for the company (Valentin) . It is important that all key stakeholders participate in the development and writing of the business plan. As the money at risk increases, the benefits of having a well-developed business plan increase as well.

U.S. Small Business Administration .

BPlans , produced by Palo Alto Software, is a free resource for new entrepreneurs with tools, webinars, and templates for writing a business plan.

Business Plan for Scientists .

Business Plans Handbook : Craft, D. Business Plans Handbook, vol. 42, pp. vii–viii. Gale, 2018. Gale Virtual Reference Library .

The contents of this chapter represent the opinions of the chapter authors and editors. The contents should not be construed as legal advice. The contents do not necessarily represent the official views of any affiliated organizations, partner organizations, or sponsors. For programs or organizations mentioned in this chapter, the authors encourage the reader to directly contact the relevant organization for additional information.

Great article, thank you!

Thanks, that's exactly what I was looking for! I read an article about tech company business plan , everything is quite detailed. But I needed a narrower specialty. Your post is just right!

Author Linda Miller and her colleague Brendon Ardietta recently conducted a CHOP workshop on articulating your idea as a “triple win” in healthcare: value for patients, clinicians/researchers, and financers (e.g. CHOP, grant funders, VCs). See video and handouts

• Content Index

• BUSINESS IDEAS
• HOW TO START
• Multi-generational Business Planning
• Services By Stunners
• CALCULATORS

Clinical research organizations (CROs) play an important role in the design and execution of clinical studies, which are required to bring innovative medications and medical devices to market. A contract research organization (CRO) is a corporation that provides assistance and services to pharmaceutical, biotechnology, and medical device companies during the clinical trial process. The growing complexity of clinical trials has increased demand for CRO services in the US market, as corporations seek to outsource portions of the trial process to experienced specialists.

The purpose of this blog post is to provide a thorough guide on how to start a Clinical Research Organization, including step-by-step instructions and success suggestions in the clinical research sector. At the end of this essay, readers should have a firm grasp on the procedures required to launch their own CRO and position themselves for success in this expanding market.

Table of Contents

How to Start a Clinical Research Organization – Step by Step Guide

Step 1: overview of the market.

To establish a successful clinical research organization (CRO), it is necessary to first learn about the clinical research sector in the United States. The clinical research sector in the United States is a highly regulated and complicated market, with clinical research undertaken across a wide range of therapeutic fields. Clinical research of various forms is undertaken in the United States, including Phase I-IV clinical trials, observational studies, and registries. Clinical trials are the most common sort of clinical research, in which a novel medicine or medical technology is tested on human participants to establish its safety and efficacy.

Clinical research in the United States has a lengthy history, reaching back to the early twentieth century. With the establishment of the Food and Drug Administration (FDA) in 1938 and the implementation of the Clinical Laboratory Improvement Amendments (CLIA) in 1988, the business has become increasingly complicated and heavily regulated throughout time.

CROs have played a significant role in the clinical research business, supporting and assisting pharmaceutical, biotechnology, and medical device companies during the clinical trial process. Services such as study design, patient recruiting, data collecting and management, and regulatory support are included. Entrepreneurs wishing to create their own CRO can position themselves for success by studying the clinical research business in the United States and the function of CROs within it.

Step 2: Prepare Business Plan

A robust business plan is essential for launching a successful clinical research enterprise (CRO). A business plan serves as a road map for the company, ensuring that all parts of the firm are examined and planned for. An overview of the sector, target market analysis, marketing and sales strategies, financial projections, finance requirements, and operational details are all included in a well-written business plan. It also describes the company’s vision, mission, and values, as well as its unique selling proposition (USP), which differentiates it from other CROs in the market.

Startup Stunners has been providing high-quality business plan writing services for years, and we’re ready to assist you in developing a complete, effective strategy that will move your company ahead. Our team of professionals is committed to assisting you in achieving your company objectives and obtaining finance from banks, grants, or other sources. We’re here to help you succeed whether you’re a newbie, entrepreneur, or small company owner. Don’t put it off any longer; visit startupstunners.com/contact-us/ today and let us lead you to success!

Step 3: Establishing Clinical Trial Services

After you’ve gained a thorough understanding of the clinical research industry and the value of a business strategy, the next stage in launching a successful CRO is to establish clinical trial services. Clinical trial services are the foundation of a CRO because they are the core offering to pharmaceutical, biotechnology, and medical device businesses.

A CRO’s clinical trial services can vary depending on the company’s size and specialism. CROs commonly provide services such as study design, site selection, patient recruitment, data collection and management, and regulatory support. It is critical to have a skilled and experienced team on hand to provide these services, as they necessitate expertise and attention to detail to ensure that the clinical trial runs smoothly and produces accurate and reliable results.

A CRO must have a reputation for providing high-quality clinical trial services in order to form partnerships with pharmaceutical companies. Building a strong network of contacts within the industry, attending conferences and networking events, and showcasing previous successes to potential clients are all essential. A CRO can also distinguish itself by providing unique or specialized services that set it apart from competitors. A CRO, for example, may be an expert in a specific therapeutic area or provide cutting-edge technology solutions for data management and analysis.

Step 4: Develop Standard Operating Procedures (SOPs)

Developing Standard Operating Procedures (SOPs) is an essential step in launching a successful CRO. SOPs are written instructions that explain how to complete a task or activity in a consistent and uniform manner. They are significant because they ensure that all parts of the company are carried out in a methodical and structured manner, which is critical for ensuring the quality of clinical trial services supplied by the CRO.

A title page, purpose, scope, definitions, duties, procedures, and reference papers are all common components of SOPs. These parts contribute to the SOP being thorough and covering all aspects of the task or activity described. It is critical to include individuals who are skilled and knowledgeable in the area being documented while writing SOPs. This ensures that the SOP is correct and reflects the best practices and procedures for that particular task or activity.

Employee training on SOPs is critical to ensuring that they are followed consistently and accurately. Employees that have received SOP training understand the value of adhering to defined protocols and are better suited to do their duties. This training can take a variety of forms, including classroom instruction, hands-on instruction, and refresher courses. Furthermore, SOPs must be updated on a regular basis to reflect changes in rules, industry standards, or best practices.

SOPs ensure that all elements of the business are carried out in an uniform and organized manner, which is essential for ensuring the quality of clinical trial services supplied. A CRO can establish a strong basis for its operations and position itself for success in the clinical research sector by including experienced personnel in the formulation of SOPs, offering extensive training to staff, and constantly updating SOPs.

Step 5: Securing Funds

Obtaining money is a critical step in launching a CRO. CROs can obtain financing from a variety of sources, including:

• Equity financing: In exchange for finance, equity financing entails selling ownership shares in the Company to investors. This can be accomplished with the help of private equity firms, venture capitalists, or angel investors. Equity finance can provide large capital, but it also dilutes the company’s control.
• Debt financing: Debt financing is borrowing money from lenders such as banks or financial institutions and repaying it over time with interest. This can take the shape of a loan, credit line, or credit card. Debt financing can provide instant cash flow, but it also involves interest repayment, which can be a substantial burden for a fledgling business.
• Government funding: CROs can obtain government support in the form of grants, loans, or tax credits. These funding are often allocated to certain research fields or sectors and may impose stringent documentation and reporting requirements.
• Crowdfunding: Crowdfunding is the practice of soliciting small sums of money from a large number of individuals, generally through internet platforms. This can be an excellent alternative for CROs wishing to generate small sums of money or establish a network of supporters.
• Strategic alliances: Strategic alliances involve collaborating with other businesses or organizations to exchange resources, knowledge, and finance. This can be an excellent alternative for CROs wanting to secure finance by leveraging current ties and networks.

Step 6: Tips for Success in the clinical research industry

To compete in the clinical research sector, CROs must have a solid foundation, cultivate strong client relationships, and stay current on industry rules and best practices. Here are some pointers for achieving success in the clinical research industry:

• Concentrate on quality: CROs must build a reputation for offering top-notch services because quality is crucial in the clinical research sector. This includes making certain that all areas of the firm are standardized and organized, and that workers are well-trained and experienced.
• Establish good client relationships: Establishing strong client relationships is crucial for success in the clinical research sector. Listening to clients’ wants and concerns, being responsive and open, and following through on promises are all part of this. CROs may establish a solid reputation and position themselves for long-term success by fostering customer trust and loyalty.
• Maintain compliance with legislation: Because the clinical research sector is extensively regulated, CROs must maintain compliance with all relevant regulations and guidelines. This includes investing in regular personnel training and education as well as being up to date on changes in rules and best practices.
• Use technology to increase efficiency and quality: Technology is becoming increasingly crucial in the clinical research sector, and CROs that can use it to improve efficiency and quality will have a competitive advantage. Investing in technology solutions such as electronic data capture systems and clinical trial administration software is part of this.
• Concentrate on innovation: The clinical research market is continuously changing, and CROs who can innovate and adapt to changes will be well-positioned for success. This entails investing in R&D, being current on emerging trends and technology, and being ready to take prudent risks.

Step 7: Networking and partnership formation

Networking and forming alliances are critical for success in the clinical research sector. Here are some pointers for good networking and partnership formation:

• Attend industry conferences and events: Attending industry conferences and events is a terrific method to meet possible partners and collaborators. These gatherings allow attendees to network with industry experts, learn about new trends and technology, and form new business partnerships.
• Join industry groups and organizations: Another excellent strategy to network and form partnerships is to join industry associations and organizations. These associations and organizations give members access to industry experts and leaders, as well as educational and networking opportunities.
• Use social media: Social media sites such as LinkedIn and Twitter allow you to interact with industry people and develop new connections. CROs can establish new alliances and expand their brand by distributing thought leadership content and communicating with other industry professionals.
• Be aggressive in your approach: Creating alliances necessitates proactive outreach. CROs should identify suitable partners and approach them with a clear value proposition and a plan for how the collaboration may benefit both parties.
• Emphasize collaboration: Relationships thrive when they are collaborative and mutually beneficial. CROs should focus on developing partnerships based on shared values and goals that exploit both parties’ strengths.
• Follow up and keep in touch: Successful collaborations include continual contact and follow-up. CROs should make an attempt to maintain contact with partners and collaborators, as well as to cultivate these connections over time.

Step 8: Staying up-to-date with industry trends

Keeping current with industry developments is critical for clinical research success. Here are some of the reasons:

• Competitiveness: The clinical research sector is extremely competitive, and staying current on industry developments can provide CROs with a competitive advantage. CROs may provide better services and solutions to their clients by understanding the latest technology, methodologies, and approaches.
• Compliance: Because the clinical research sector is highly regulated, maintaining current on industry trends is crucial for assuring compliance. CROs may ensure that they are functioning in a compliant and ethical manner by remaining up to date on the current legislation and guidelines.
• Innovation: Because the clinical research market is always changing, being current on industry trends is critical for innovation. CROs can produce new services and solutions that match the changing needs of their clients by understanding the newest research and development trends.
• Reputation: Keeping up with market changes can help CROs establish themselves as industry leaders. CROs may demonstrate their expertise and establish themselves as trusted partners for their clients by releasing thought leadership content and engaging in industry events and forums.
• Efficiency: Keeping current on industry developments can also help CROs run more efficiently. CROs can enhance their bottom line and become more competitive by implementing new technology and practices that streamline their processes and cut costs.

Step 9: Technology

There are various technologies that can be used in clinical research, each with its own set of advantages. Here are a couple such examples:

• EDC Systems: EDC systems are software applications designed to gather, handle, and store clinical trial data. EDC systems can increase data quality and accuracy by streamlining data gathering and management procedures, reducing mistakes and inconsistencies, and streamlining data collection and management processes.
• Clinical Trial Management Systems (CTMS): CTMS are software tools developed to manage clinical trial operational features such as patient enrollment, study progress, and trial finances. CTMS can increase research efficiency, save expenses, and improve trial management overall.
• Electronic Patient-Reported Outcomes (ePRO) Systems: ePRO systems are software applications that enable patients to electronically complete study-related questionnaires and assessments, typically using a mobile device or PC. ePRO systems can increase patient compliance and engagement while decreasing data entry errors and improving data accuracy.
• Wearable Devices: Smartwatches and fitness trackers, for example, can be used to collect patient health data such as heart rate and activity levels. This information can be utilized to track patient health and the efficacy of treatments. Wearable technology can help boost patient involvement and compliance.
• Artificial Intelligence (AI): Artificial intelligence (AI) technologies such as machine learning and natural language processing can be used to examine enormous datasets and detect patterns and trends. AI can assist researchers in discovering new treatments and therapies, increasing patient recruitment and retention, and improving overall trial design and management.

To summarize, establishing a clinical research organization can be a difficult yet rewarding endeavor. It is critical to have a good business plan in place, to form relationships with pharmaceutical companies, to develop complete SOPs, to acquire funding, and to stay current on industry developments and innovations. CROs may provide high-quality clinical trial services and contribute to the advancement of medical research by following these steps and harnessing the latest technologies. With rising demand for CRO services in the US market, there has never been a better moment to establish a clinical research company.

Frequently Asked Questions

What is the definition of a clinical research organization (cro).

A clinical research organization (CRO) is a corporation that provides clinical trial support services to the pharmaceutical, biotechnology, and medical device sectors.

Why are contract research organizations (CROs) significant in clinical research?

Clinical research organizations (CROs) are significant in clinical research because they provide the infrastructure, resources, and knowledge needed to perform clinical trials rapidly and successfully. CROs also help to reduce the risks involved with clinical trials and assure regulatory compliance.

What forms of clinical research are carried out in the United States?

Clinical research in the United States includes drug trials, device trials, observational studies, and epidemiological research.

What exactly are normal operating procedures?

SOPs are written documents that outline the procedures, processes, and rules that a corporation uses to maintain consistency and quality in its operations.

How may CROs obtain funding?

CROs can obtain money in a variety of ways, including venture capital, private equity, grants, loans, and collaborations with pharmaceutical corporations.

How are clinical research technologies used?

Electronic data capture (EDC) systems, clinical trial management systems (CTMS), electronic patient-reported outcomes (ePRO) systems, wearable devices, and artificial intelligence are all extensively utilized in clinical research (AI).

What are some pointers for achieving success in the clinical research industry?

Building a professional and experienced staff, creating good partnerships with pharmaceutical companies, remaining up to speed with industry changes and technologies, and maintaining a focus on quality and compliance are some guidelines for success in the clinical research sector.

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How to start a clinical research site

31st Jul 2018

Establishing a new research site can be exciting but challenging – to be successful there needs to be a lot of preparation and hard work

Starting a clinical research site is an exciting opportunity for the ambitious research professional looking to progress from employee to site ownership, or the medical entrepreneur looking to expand a portfolio with a lucrative investment. Clinical research provides financial and altruistic appeal: a successful site will conduct a diversity of studies with commensurate compensation. The data generated (from trials conducted) could possibly contribute to a regulatory agency submission for new drug approval. At the very least, a successful site will impact the drug development process.

Beyond the altruism is the enormity of work involved in establishing an investigative site. From funding to infrastructure, staffing to strategic connections, it takes business and clinical acumen to start and a salesperson’s mentality to sustain a successful study site.

Appropriate funding is integral to starting and continuing the site momentum until new trials can sustain the effort. The investigational site founders must know the amount needed to start the site, and how long the funding will cover all costs until xxx deadline and the need to re-fund or re-evaluate the effort.

Some prospective site owners self-fund-with personal savings or business loans, while others align with investors or physician groups looking to expand into clinical research. Gualberto Perez MD, founder and president at Research GCP, and a successful research site owner, has participated in several successful financial strategies for site start up. Perez states that “it all depends on the size of your start up. When I first started many years ago (25 approximately) we commenced in a big way with a full-blown phase I unit— multiple private investors; we later borrowed from a bank”.

Ten years later Perez opened his late-phase site GCP Research with his own funding. He grew it organically in a financially graded fashion, expanding to other services and embarking into outpatient phase I work. His success is due to his expertise, and the incredible work ethic needed to start an investigational site.

The right location is dependent on the site model and overall objective. An ambitious early phase site may need a larger facility to accommodate specific serial testing (blood, respiratory, vitals) and visit duration (overnight and or extended patient stays), while a medical office/clinic would accommodate the needs of an investigational site conducting simpler late-phase trials. The basic location requirements are examination room/bed, locked area for refrigerated or ambient study drug storage, secure storage for supplies/records, staff and monitor work areas, a room for specific activities (if needed) such as blood draws/electrocardiogram/pulmonary function testing and a lab area for blood/urine processing, (centrifuges) storage/shipment (freezers). There are additional lab certificate/waiver requirements dependent on the type and extent of lab testing to be completed at the site.

Establishing infrastructure with standardised clinical research process (informed consent, safety reporting, deviation reporting, study drug administration/destruction, staff on boarding and training, etc.) and staff job descriptions will promote consistency and transparency with job performance. Centralised filing of staff licensure and training certificates (for such things as good clinical practice, equipment, and computer systems/study devices) to correspond with the delegation of authority log demonstrates due diligence and appropriate task delegation.

Patient recruitment is integral to successful study conduct and sponsor consideration for repeat business. For a dedicated research site, affiliation with hospitals, clinics and/or physician practices is critical to identify and enroll patients, as well as recruitment staff to develop tools/tactics to advertise the studies. Alternatively, some investigators house the research department within their physical medical practice and may have the opportunity to review their practice database for potential patients.

Hiring staff

The ideal functional team includes the experienced physician investigator, study nurse/coordinator/data manager (to divide clinical and administrative, scheduling and data management duties), experienced finance/legal/business personnel to handle contracts, budgets, and business strategy to optimise site growth, and key individuals with the network/relationships to attract investigational studies.

Role is also driven by site size and business model. A study coordinator at a smaller physician practice will likely wear many hats and complete study duties as well as contracts, regulatory and recruitment. A larger research site will have separate personnel for clinical, data management, legal and administrative responsibilities, as well as dedicated business development staff to cultivate relationships.

The smart administrator or investigator will develop connections with local imaging, ophthalmology, spirometry, e.g. medical offices that perform ancillary testing, so that if a study presents with specific safety testing requirements for study drug, the ancillary facilities are already vetted and capabilities confirmed. This strengthens a site’s credibility and helps diversity their therapeutic portfolio.

Dan Sfera, CEO of DSCS CRO and “The Clinical Trials Guru” podcast host, as well as a successful investigative site owner, feels that adaptability and salesmanship are two critical traits for successful site owners. Sfera says that the effort should not be underestimated when starting an investigational site.

“If there are any important lessons that I’ve learned to own a clinical research site, it is the fact that there are almost no rules or guidelines or standards for how to structure your business. When starting out, it could be very challenging and overwhelming for somebody coming from a structured environment, such as a CRA or a study coordinator, to go out on his/her own and become a site owner. At the end of the day, the most important concept that you need to understand and master is the art of salesmanship. You must understand that from the very minute you start your site as a business owner, you are a sales person first. It does not matter what your background is. It does not matter what you’ve done in the past. You have to master sales.”

Sfera also gives two pivotal examples of adaptability as a site owner; “the ability to adapt. I talk about this all the time. Your first principal investigator will probably leave you at some point. The studies that you specialise in at some point will go through a downcycle. The patients that used to get referred to you will at some point stop. The same thing goes for your employee’s retention.”

Whatever the motivation, or business model pursued, an individual with the tenacity, experience and work ethic has the potential to build a successful research site that will benefit society and the employees vested to make it successful.

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Write Your Research Plan

In this part, we give you detailed information about writing an effective Research Plan. We start with the importance and parameters of significance and innovation.

We then discuss how to focus the Research Plan, relying on the iterative process described in the Iterative Approach to Application Planning Checklist shown at Draft Specific Aims  and give you advice for filling out the forms.

You'll also learn the importance of having a well-organized, visually appealing application that avoids common missteps and the importance of preparing your just-in-time information early.

While this document is geared toward the basic research project grant, the R01, much of it is useful for other grant types.

Table of Contents

Research plan overview and your approach, craft a title, explain your aims, research strategy instructions, advice for a successful research strategy, graphics and video, significance, innovation, and approach, tracking for your budget, preliminary studies or progress report, referencing publications, review and finalize your research plan, abstract and narrative.

Your application's Research Plan has two sections:

• Specific Aims —a one-page statement of your objectives for the project.
• Research Strategy —a description of the rationale for your research and your experiments in 12 pages for an R01.

In your Specific Aims, you note the significance and innovation of your research; then list your two to three concrete objectives, your aims.

Your Research Strategy is the nuts and bolts of your application, where you describe your research rationale and the experiments you will conduct to accomplish each aim. Though how you organize it is largely up to you, NIH expects you to follow these guidelines.

• Organize using bold headers or an outline or numbering system—or both—that you use consistently throughout.
• Start each section with the appropriate header: Significance, Innovation, or Approach.
• Organize the Approach section around your Specific Aims.

Format of Your Research Plan

To write the Research Plan, you don't need the application forms. Write the text in your word processor, turn it into a PDF file, and upload it into the application form when it's final.

Because NIH may return your application if it doesn't meet all requirements, be sure to follow the rules for font, page limits, and more. Read the instructions at NIH’s Format Attachments .

For an R01, the Research Strategy can be up to 12 pages, plus one page for Specific Aims. Don't pad other sections with information that belongs in the Research Plan. NIH is on the lookout and may return your application to you if you try to evade page limits.

Follow Examples

As you read this page, look at our Sample Applications and More  to see some of the different strategies successful PIs use to create an outstanding Research Plan.

Keeping It All In Sync

Writing in a logical sequence will save you time.

Information you put in the Research Plan affects just about every other application part. You'll need to keep everything in sync as your plans evolve during the writing phase.

It's best to consider your writing as an iterative process. As you develop and finalize your experiments, you will go back and check other parts of the application to make sure everything is in sync: the "who, what, when, where, and how (much money)" as well as look again at the scope of your plans.

In that vein, writing in a logical sequence is a good approach that will save you time. We suggest proceeding in the following order:

• Create a provisional title.
• Write a draft of your Specific Aims.
• Start with your Significance and Innovation sections.
• Then draft the Approach section considering the personnel and skills you'll need for each step.
• Evaluate your Specific Aims and methods in light of your expected budget (for a new PI, it should be modest, probably under the $250,000 for NIH's modular budget).
• As you design experiments, reevaluate your hypothesis, aims, and title to make sure they still reflect your plans.
• Prepare your Abstract (a summary of your Specific Aims).
• Complete the other forms.

Even the smaller sections of your application need to be well-organized and readable so reviewers can readily grasp the information. If writing is not your forte, get help.

To view writing strategies for successful applications, see our Sample Applications and More . There are many ways to create a great application, so explore your options.

Within the character limit, include the important information to distinguish your project within the research area, your project's goals, and the research problem.

Giving your project a title at the outset can help you stay focused and avoid a meandering Research Plan. So you may want to launch your writing by creating a well-defined title.

NIH gives you a 200 character limit, but don’t feel obliged to use all of that allotment. Instead, we advise you to keep the title as succinct as possible while including the important information to distinguish your project within the research area. Make your title reflect your project's goals, the problem your project addresses, and possibly your approach to studying it. Make your title specific: saying you are studying lymphocyte trafficking is not informative enough.

For examples of strong titles, see our Sample Applications and More .

After you write a preliminary title, check that

• My title is specific, indicating at least the research area and the goals of my project.
• It is 200 characters or less.
• I use as simple language as possible.
• I state the research problem and, possibly, my approach to studying it.
• I use a different title for each of my applications. (Note: there are exceptions, for example, for a renewal—see Apply for Renewal  for details.)
• My title has appropriate keywords.

Later you may want to change your initial title. That's fine—at this point, it's just an aid to keep your plans focused.

Since all your reviewers read your Specific Aims, you want to excite them about your project.

If testing your hypothesis is the destination for your research, your Research Plan is the map that takes you there.

You'll start by writing the smaller part, the Specific Aims. Think of the one-page Specific Aims as a capsule of your Research Plan. Since all your reviewers read your Specific Aims, you want to excite them about your project.

For more on crafting your Specific Aims, see Draft Specific Aims .

Write a Narrative

Use at least half the page to provide the rationale and significance of your planned research. A good way to start is with a sentence that states your project's goals.

For the rest of the narrative, you will describe the significance of your research, and give your rationale for choosing the project. In some cases, you may want to explain why you did not take an alternative route.

Then, briefly describe your aims, and show how they build on your preliminary studies and your previous research. State your hypothesis.

If it is likely your application will be reviewed by a study section with broad expertise, summarize the status of research in your field and explain how your project fits in.

In the narrative part of the Specific Aims of many outstanding applications, people also used their aims to

• State the technologies they plan to use.
• Note their expertise to do a specific task or that of collaborators.
• Describe past accomplishments related to the project.
• Describe preliminary studies and new and highly relevant findings in the field.
• Explain their area's biology.
• Show how the aims relate to one another.
• Describe expected outcomes for each aim.
• Explain how they plan to interpret data from the aim’s efforts.
• Describe how to address potential pitfalls with contingency plans.

Depending on your situation, decide which items are important for you. For example, a new investigator would likely want to highlight preliminary data and qualifications to do the work.

Many people use bold or italics to emphasize items they want to bring to the reviewers' attention, such as the hypothesis or rationale.

Detail Your Aims

After the narrative, enter your aims as bold bullets, or stand-alone or run-on headers.

• State your plans using strong verbs like identify, define, quantify, establish, determine.
• Describe each aim in one to three sentences.
• Consider adding bullets under each aim to refine your objectives.

How focused should your aims be? Look at the example below.

Spot the Sample

Read the Specific Aims of the Application from Drs. Li and Samulski , "Enhance AAV Liver Transduction with Capsid Immune Evasion."

• Aim 1. Study the effect of adeno-associated virus (AAV) empty particles on AAV capsid antigen cross-presentation in vivo .
• Aim 2. Investigate AAV capsid antigen presentation following administration of AAV mutants and/or proteasome inhibitors for enhanced liver transduction in vivo .
• Aim 3. Isolate AAV chimeric capsids with human hepatocyte tropism and the capacity for cytotoxic T lymphocytes (CTL) evasion.

After finishing the draft Specific Aims, check that

• I keep to the one-page limit.
• Each of my two or three aims is a narrowly focused, concrete objective I can achieve during the grant.
• They give a clear picture of how my project can generate knowledge that may improve human health.
• They show my project's importance to science, how it addresses a critical research opportunity that can move my field forward.
• My text states how my work is innovative.
• I describe the biology to the extent needed for my reviewers.
• I give a rationale for choosing the topic and approach.
• I tie the project to my preliminary data and other new findings in the field.
• I explicitly state my hypothesis and why testing it is important.
• My aims can test my hypothesis and are logical.
• I can design and lead the execution of two or three sets of experiments that will strive to accomplish each aim.
• As much as possible, I use language that an educated person without expertise can understand.
• My text has bullets, bolding, or headers so reviewers can easily spot my aims (and other key items).

For each element listed above, analyze your text and revise it until your Specific Aims hit all the key points you'd like to make.

After the list of aims, some people add a closing paragraph, emphasizing the significance of the work, their collaborators, or whatever else they want to focus reviewers' attention on.

Your Research Strategy is the bigger part of your application's Research Plan (the other part is the Specific Aims—discussed above.)

The Research Strategy is the nuts and bolts of your application, describing the rationale for your research and the experiments you will do to accomplish each aim. It is structured as follows:

• Significance
• You can either include this information as a subsection of Approach or integrate it into any or all of the three main sections.
• If you do the latter, be sure to mark the information clearly, for example, with a bold subhead.
• Possible other sections, for example, human subjects, vertebrate animals, select agents, and others (these do not count toward the page limit).

Though how you organize your application is largely up to you, NIH does want you to follow these guidelines:

• Add bold headers or an outlining or numbering system—or both—that you use consistently throughout.
• Start each of the Research Strategy's sections with a header: Significance, Innovation, and Approach.

For an R01, the Research Strategy is limited to 12 pages for the three main sections and the preliminary studies only. Other items are not included in the page limit.

Find instructions for R01s in the SF 424 Application Guide—go to NIH's SF 424 (R&R) Application and Electronic Submission Information for the generic SF 424 Application Guide or find it in your notice of funding opportunity (NOFO).

For most applications, you need to address Rigor and Reproducibility by describing the experimental design and methods you propose and how they will achieve robust and unbiased results. The requirement applies to research grant, career development, fellowship, and training applications.

If you're responding to an institute-specific program announcement (PA) (not a parent program announcement) or a request for applications (RFA), check the NIH Guide notice, which has additional information you need. Should it differ from the NOFO, go with the NIH Guide .

Also note that your application must meet the initiative's objectives and special requirements. NIAID program staff will check your application, and if it is not responsive to the announcement, your application will be returned to you without a review.

When writing your Research Strategy, your goal is to present a well-organized, visually appealing, and readable description of your proposed project. That means your writing should be streamlined and organized so your reviewers can readily grasp the information. If writing is not your forte, get help.

There are many ways to create an outstanding Research Plan, so explore your options.

What Success Looks Like

Your application's Research Plan is the map that shows your reviewers how you plan to test your hypothesis.

It not only lays out your experiments and expected outcomes, but must also convince your reviewers of your likely success by allaying any doubts that may cross their minds that you will be able to conduct the research.

Notice in the sample applications how the writing keeps reviewers' eyes on the ball by bringing them back to the main points the PIs want to make. Write yourself an insurance policy against human fallibility: if it's a key point, repeat it, then repeat it again.

The Big Three

So as you write, put the big picture squarely in your sights. When reviewers read your application, they'll look for the answers to three basic questions:

• Can your research move your field forward?
• Is the field important—will progress make a difference to human health?
• Can you and your team carry out the work?

Add Emphasis

Savvy PIs create opportunities to drive their main points home. They don't stop at the Significance section to emphasize their project's importance, and they look beyond their biosketches to highlight their team's expertise.

Don't take a chance your reviewer will gloss over that one critical sentence buried somewhere in your Research Strategy or elsewhere. Write yourself an insurance policy against human fallibility: if it's a key point, repeat it, then repeat it again.

Add more emphasis by putting the text in bold, or bold italics (in the modern age, we skip underlining—it's for typewriters).

Here are more strategies from our successful PIs:

• While describing a method in the Approach section, they state their or collaborators' experience with it.
• They point out that they have access to a necessary piece of equipment.
• When explaining their field and the status of current research, they weave in their own work and their preliminary data.
• They delve into the biology of the area to make sure reviewers will grasp the importance of their research and understand their field and how their work fits into it.

You can see many of these principles at work in the Approach section of the Application from Dr. William Faubion , "Inflammatory cascades disrupt Treg function through epigenetic mechanisms."

• Reviewers felt that the experiments described for Aim 1 will yield clear results.
• The plans to translate those findings to gene targets of relevance are well outlined and focused.
• He ties his proposed experiments to the larger picture, including past research and strong preliminary data for the current application. 

Anticipate Reviewer Questions

Our applicants not only wrote with their reviewers in mind they seemed to anticipate their questions. You may think: how can I anticipate all the questions people may have? Of course you can't, but there are some basic items (in addition to the "big three" listed above) that will surely be on your reviewers' minds:

• Will the investigators be able to get the work done within the project period, or is the proposed work over ambitious?
• Did the PI describe potential pitfalls and possible alternatives?
• Will the experiments generate meaningful data?
• Could the resulting data prove the hypothesis?
• Are others already doing the work, or has it been already completed?

Address these questions; then spend time thinking about more potential issues specific to you and your research—and address those too.

For applications, a picture can truly be worth a thousand words. Graphics can illustrate complex information in a small space and add visual interest to your application.

Look at our sample applications to see how the investigators included schematics, tables, illustrations, graphs, and other types of graphics to enhance their applications.

Consider adding a timetable or flowchart to illustrate your experimental plan, including decision trees with alternative experimental pathways to help your reviewers understand your plans.

Plan Ahead for Video

If you plan to send one or more videos, you'll need to meet certain standards and include key information in your Research Strategy now.

To present some concepts or demonstrations, video may enhance your application beyond what graphics alone can achieve. However, you can't count on all reviewers being able to see or hear video, so you'll want to be strategic in how you incorporate it into your application.

Be reviewer-friendly. Help your cause by taking the following steps:

• Caption any narration in the video.
• Choose evocative still images from your video to accompany your summary.
• Write your summary of the video carefully so the text would make sense even without the video.

In addition to those considerations, create your videos to fit NIH’s technical requirements. Learn more in the SF 424 Form Instructions .

Next, as you write your Research Strategy, include key images from the video and a brief description.

Then, state in your cover letter that you plan to send video later. (Don't attach your files to the application.)

After you apply and get assignment information from the Commons, ask your assigned scientific review officer (SRO) how your business official should send the files. Your video files are due at least one month before the peer review meeting.

Know Your Audience's Perspective

The primary audience for your application is your peer review group. Learn how to write for the reviewers who are experts in your field and those who are experts in other fields by reading Know Your Audience .

Be Organized: A B C or 1 2 3?

In the top-notch applications we reviewed, organization ruled but followed few rules. While you want to be organized, how you go about it is up to you.

Nevertheless, here are some principles to follow:

• Start each of the Research Strategy's sections with a header: Significance, Innovation, and Approach—this you must do.

The Research Strategy's page limit—12 for R01s—is for the three main parts: Significance, Innovation, and Approach and your preliminary studies (or a progress report if you're renewing your grant). Other sections, for example, research animals or select agents, do not have a page limit.

Although you will emphasize your project's significance throughout the application, the Significance section should give the most details. Don't skimp—the farther removed your reviewers are from your field, the more information you'll need to provide on basic biology, importance of the area, research opportunities, and new findings.

When you describe your project's significance, put it in the context of 1) the state of your field, 2) your long-term research plans, and 3) your preliminary data.

In our Sample Applications , you can see that both investigators and reviewers made a case for the importance of the research to improving human health as well as to the scientific field.

Look at the Significance section of the Application from Dr. Mengxi Jiang , "Intersection of polyomavirus infection and host cellular responses," to see how these elements combine to make a strong case for significance.

• Dr. Jiang starts with a summary of the field of polyomavirus research, identifying critical knowledge gaps in the field.
• The application ties the lab's previous discoveries and new research plans to filling those gaps, establishing the significance with context.
• Note the use of formatting, whitespace, and sectioning to highlight key points and make it easier for reviewers to read the text.

After conveying the significance of the research in several parts of the application, check that

• In the Significance section, I describe the importance of my hypothesis to the field (especially if my reviewers are not in it) and human disease.
• I also point out the project's significance throughout the application.
• The application shows that I am aware of opportunities, gaps, roadblocks, and research underway in my field.
• I state how my research will advance my field, highlighting knowledge gaps and showing how my project fills one or more of them.
• Based on my scan of the review committee roster, I determine whether I cannot assume my reviewers will know my field and provide some information on basic biology, the importance of the area, knowledge gaps, and new findings.

If you are either a new PI or entering a new area: be cautious about seeming too innovative. Not only is innovation just one of five review criteria, but there might be a paradigm shift in your area of science. A reviewer may take a challenge to the status quo as a challenge to his or her world view.

When you look at our sample applications, you see that both the new and experienced investigators are not generally shifting paradigms. They are using new approaches or models, working in new areas, or testing innovative ideas.

After finishing the draft innovation section, check that

• I show how my proposed research is new and unique, e.g., explores new scientific avenues, has a novel hypothesis, will create new knowledge.
• Most likely, I explain how my project's research can refine, improve, or propose a new application of an existing concept or method.
• Make a very strong case for challenging the existing paradigm.
• Have data to support the innovative approach.
• Have strong evidence that I can do the work.

In your Approach, you spell out a few sets of experiments to address each aim. As we noted above, it's a good idea to restate the key points you've made about your project's significance, its place in your field, and your long-term goals.

You're probably wondering how much detail to include.

If you look at our sample applications as a guide, you can see very different approaches. Though people generally used less detail than you'd see in a scientific paper, they do include some experimental detail.

Expect your assigned reviewers to scrutinize your approach: they will want to know what you plan to do and how you plan to do it.

NIH data show that of the peer review criteria, approach has the highest correlation with the overall impact score.

Look at the Application from Dr. Mengxi Jiang , "Intersection of polyomavirus infection and host cellular responses," to see how a new investigator handled the Approach section.

For an example of an experienced investigator's well-received Approach section, see the Application from Dr. William Faubion , "Inflammatory cascades disrupt Treg function through epigenetic mechanisms."

Especially if you are a new investigator, you need enough detail to convince reviewers that you understand what you are undertaking and can handle the method.

• Cite a publication that shows you can handle the method where you can, but give more details if you and your team don't have a proven record using the method—and state explicitly why you think you will succeed.
• If space is short, you could also focus on experiments that highlight your expertise or are especially interesting. For experiments that are pedestrian or contracted out, just list the method.

Be sure to lay out a plan for alternative experiments and approaches in case you get negative or surprising results. Show reviewers you have a plan for spending the four or five years you will be funded no matter where the experiments lead.

See the Application from Drs. Li and Samulski , "Enhance AAV Liver Transduction with Capsid Immune Evasion," for a strong Approach section covering potential. As an example, see section C.1.3.'s alternative approaches.

Here are some pointers for organizing your Approach:

• Enter a bold header for each Specific Aim.
• Under each aim, describe the first set of experiments.
• If you get result X, you will follow pathway X; if you get result Y, you will follow pathway Y.
• Consider illustrating this with a flowchart.

Trim the fat—omit all information not needed to make your case. If you try to wow reviewers with your knowledge, they'll find flaws and penalize you heavily. Don't give them ammunition by including anything you don't need.

As you design your experiments, keep a running tab of the following essential data on a separate piece of paper:

• Who. A list of people who will help you for your Key Personnel section later.
• What. A list of equipment and supplies for the experiments you plan.
• Time. Notes on how long each step takes. Timing directly affects your budget as well as how many Specific Aims you can realistically achieve.

Jotting this information down will help you Create a Budget and complete other sections later.

After finishing a draft Approach section, check that

• I include enough background and preliminary data to give reviewers the context and significance of my plans.
• They can test the hypothesis (or hypotheses).
• I show alternative experiments and approaches in case I get negative or surprising results.
• My experiments can yield meaningful data to test my hypothesis (or hypotheses).
• As a new investigator, I include enough detail to convince reviewers I understand and can handle a method. I reviewed the sample applications to see how much detail to use.
• If I or my team has experience with a method, I cite it; otherwise I include enough details to convince reviewers we can handle it.
• I describe the results I anticipate and their implications.
• I omit all information not needed to state my case.
• I keep track of and explain who will do what, what they will do, when and where they will do it, how long it will take, and how much money it will cost.
• My timeline shows when I expect to complete my aims.

If you are applying for a new application, include preliminary studies; for a renewal or a revision (a competing supplement to an existing grant), prepare a progress report instead.

Describing Preliminary Studies

Your preliminary studies show that you can handle the methods and interpret results. Here's where you build reviewer confidence that you are headed in the right direction by pursuing research that builds on your accomplishments.

Reviewers use your preliminary studies together with the biosketches to assess the investigator review criterion, which reflects the competence of the research team.

Give alternative interpretations to your data to show reviewers you've thought through problems in-depth and are prepared to meet future challenges. If you don't do this, the reviewers will!

Though you may include other people's publications, focus on your preliminary data or unpublished data from your lab and the labs of your team members as much as you can.

As we noted above, you can put your preliminary data anywhere in the Research Strategy that you feel is appropriate, but just make sure your reviewers will be able to distinguish it. Alternatively, you can create a separate section with its own header.

Including a Progress Report

If you are applying for a renewal or a revision (a competing supplement to an existing grant), prepare a progress report instead of preliminary studies.

Create a header so your program officer can easily find it and include the following information:

• Project period beginning and end dates.
• Summary of the importance of your findings in relation to your Specific Aims.
• Account of published and unpublished results, highlighting your progress toward achieving your Specific Aims.

Note: if you submit a renewal application before the due date of your progress report, you do not need to submit a separate progress report for your grant. However, you will need to submit it, if your renewal is not funded.

After finishing the draft, check that

• I interpret my preliminary results critically.
• There is enough information to show I know what I'm talking about.
• If my project is complex, I give more preliminary studies.
• I show how my previous experience prepared me for the new project.
• It's clear which data are mine and which are not.

References show your breadth of knowledge of the field. If you leave out an important work, reviewers may assume you're not aware of it.

Throughout your application, you will reference all relevant publications for the concepts underlying your research and your methods.

Read more about your Bibliography and References Cited at Add a Bibliography and Appendix .

• Throughout my application I cite the literature thoroughly but not excessively, adding citations for all references important to my work.
• I cite all papers important to my field, including those from potential reviewers.
• I include fewer than 100 citations (if possible).
• My Bibliography and References Cited form lists all my references.
• I refer to unpublished work, including information I learned through personal contacts.
• If I do not describe a method, I add a reference to the literature.

Look over what you've written with a critical eye of a reviewer to identify potential questions or weak spots.

Enlist others to do that too—they can look at your application with a fresh eye. Include people who aren't familiar with your research to make sure you can get your point across to someone outside your field.

As you finalize the details of your Research Strategy, you will also need to return to your Specific Aims to see if you must revise. See Draft Specific Aims .

After you finish your Research Plan, you are ready to write your Abstract (called Project Summary/Abstract) and Project Narrative, which are attachments to the Other Project Information form.

These sections may be small, but they're important.

• All your peer reviewers read your Abstract and narrative.
• Staff and automated systems in NIH's Center for Scientific Review use them to decide where to assign your application, even if you requested an institute and study section.
• They show the importance and health relevance of your research to members of the public and Congress who are interested in what NIH is funding with taxpayer dollars.

Be sure to omit confidential or proprietary information in these sections! When your application is funded, NIH enters your title and Abstract in the public RePORTER database.

Think brief and simple: to the extent that you can, write these sections in lay language, and include appropriate keywords, e.g., immunotherapy, genetic risk factors.

As NIH referral officers use these parts to direct your application to an institute for possible funding, your description can influence the choice they make.

Write a succinct summary of your project that both a scientist and a lay person can understand (to the extent that you can).

• Use your Specific Aims as a template—shorten it and simplify the language.
• In the first sentence, state the significance of your research to your field and relevance to NIAID's mission: to better understand, treat, and prevent infectious, immunologic, and allergic diseases.
• Next state your hypothesis and the innovative potential of your research.
• Then list and briefly describe your Specific Aims and long-term objectives.

In your Project Narrative, you have only a few sentences to drive home your project's potential to improve public health.

Check out these effective Abstracts and Narratives from our R01  Sample Applications :

• Application from Dr. Mengxi Jiang , "Intersection of polyomavirus infection and host cellular responses"
• Application from Dr. William Faubion , "Inflammatory cascades disrupt Treg function through epigenetic mechanisms"
• My Project Summary/Abstract and Project Narrative (and title) are accessible to a broad audience.
• They describe the significance of my research to my field and state my hypothesis, my aims, and the innovative potential of my research.
• My narrative describes my project's potential to improve public health.
• I do not include any confidential or proprietary information.
• I do not use graphs or images.
• My Abstract has keywords that are appropriate and distinct enough to avoid confusion with other terms.
• My title is specific and informative.

Previous Step

Have questions.

A program officer in your area of science can give you application advice, NIAID's perspective on your research, and confirmation that your proposed research fits within NIAID’s mission.

Find contacts and instructions at When to Contact an NIAID Program Officer .

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A 3-Step Plan to Set Up Your Clinical Trial Site for Success in the New Year

What makes up a clinical trial infrastructure?

What are adaptive infrastructure decisions shown to help recruit difficult patient populations, what factors contribute to a successful clinical monitoring team, what pre-planning strategies are necessary to bring cohesiveness to the clinical monitoring and site management teams, what factors are important to improve the experience of a clinical trial participant, why is the industry experiencing a shift to more patient-centric practices in clinical trial settings, references:.

Ever heard the term fail-forward? Many accounts of failed clinical trials have been reported – some of which resulted in costly delays and unsatisfactory trial conclusions. More importantly, those failures provided valuable lessons to those in the industry. We curated a set of best practices on how to set up your clinical trial site for success in the new year. We assembled a bold, high-impact process to help you navigate the clinical trial process with minimal hiccups.

Each step addresses areas prone to problems and benefits from early mitigation planning. By focusing upon the 3-step plan listed below, you are giving your clinical trial site the best opportunity to achieve activation status without delays. Starting the clinical trial with a solid foothold on the process helps steer the trial process through to successful completion.

Our 3-step plan details the rigors of setting up infrastructure, team development, and patient-centric modalities. We paired these pain points with valuable take-away prompts to assist clinical monitoring teams get a head-start.

1. Infrastructure: Decision-Making Based on the Needs and Perspective of the Clinical Participant

• Information Systems
• Regulatory Considerations
• Data Management
• Funding and Working Capital
• Incentives and Financial Rewards
• Specimen and Materials Management
• Safety Monitoring Planning
• Clinical Monitoring Plan
• Mitigation Strategies and Risk Assessments
• Site Planning
• Data Collection

Each clinical trial presents unique challenges and needs that may make it difficult to stay on target. The principal investigator must work diligently to establish a plan to meet or exceed clinical participation and recruitment goals. This is in addition to securing funding, potential site selection, and much more.

Experts suggest that investing in digital solutions and patient-centric infrastructure is an avenue that may help boost clinical trial participation rates. For example, an NIH discussion paper highlighted the barriers affecting participation. They estimated that despite the advances in precision therapeutics for oncological-based studies, less than 3% of adult cancer patients have participated in clinical trials. [ 1 ]

DCTs or Decentralized Clinical Trials

DCTs offer a maximum amount of flexibility for the patient. The COVID-19 pandemic illustrated to the research community that virtual trial settings can be viable with the right provisions in place and adequate planning. Exploring the possibility of introducing digital connective devices may assist in the clinical trial monitoring process of data collection. [ 2 ]

Opt-Ins for EHR (Electronic Health Record) Integration

So, here is the thing – EHR is not always a perfect antidote to simplifying data collection processes. However, we are rapidly approaching that point in the digital learning curve where next-gen technological solutions are inevitably going to be mainstream. Early-adaptation may be worth exploring.[ 2 ]

Health Literacy and Personalized Patient Education

This is the one area where technology cannot replace human connectivity. In situations where patients are potentially compromised, the capacity to learn and make quality decisions is dramatically lessened. Patient advocacy is important in clinical research. Expanding the clinical trial platform to include health literacy may yield high dividends when it comes to patient recruitment strategies.

Takeaway Prompts:

• Data collection and organization are fundamental; however, funding is a common barrier to establishing an EHR system. Determine the best allocation for expenses and, if integrating patient files into a non-established EHR system, explore digital alternatives. The key is streamlining the way you collect patient data and accessibility.
• Explore opportunities to deliver real-time, in-person patient education. This can be best achieved on a personal and one-on-one basis. Taking the extra mile to hire third-party patient advocates who specialize in any cultural aspects, language, and other personal barriers to understanding complex medical terminology. As the world is tuning in to the Internet of Things (IOT), people are self-educating and making assumptions based on popular misconceptions. Dispelling these misconceptions through literature, phone calls, or other means is difficult and can risk unsatisfactory recruitment and retention quotas. Principal investigators must be aware that survey responses affirm that clinical trial participants desire compassion, convenience, personalized attention, and transparency.

2. On-Boarding and Team Development: Tools for Optimizing Clinical Trial Site Management

• Goal Setting and Recognition
• Attention to Detail
• Communication
• Team Chemistry
• Knowledge and Experience
• Willingness and Coachability

Clinical Trial Schedule of Assessments or SOA

A clinical trial schedule of assessments is essentially the roadmap for all intended activities related to the trial. It should align with regulatory, and compliance needs and address all facets of third-party collaborations. Like the site plan, a clinical trial schedule applies actions toward safety and risk assessments in addition to details surrounding clinical trial site management, such as team schedules, location alternatives, and multiple-site initiation. [ 3 ]

Simulation of Clinical Trial Events

Orchestrating a trial-run or a mock-clinical trial participant visit can expose vulnerabilities within your site plan. Studies demonstrate through mock site visits key insights such as underdeveloped staffers, inadequate supplies, and logistical concerns. Clinical trial participants highlighted examples of communication gaps and cultural and racial under-representations. Consequently, as a matter of preparedness, mock clinical trial runs are considered a best practice. [ 4 ]

Clinical Monitoring Team Training

Clinical team training is essential for a smooth operation. The job of the principal investigator is to make sure each team member is solid with respect to their job function, emergencies, specimen, and data collection.

Takeaway Prompts

• Determining pain points to be included within the SOA falls under the responsibility of the Principal Investigator. Utilizing the SOA will help maintain organization and lessen the amount of stress involved as you navigate through the checklists.
• Team training is one of the more important facets of a well-executed operation. Finding ways to keep your clinical team on point and up to speed will be aided by effective communication and follow-up. (Never assume your staff members are up-to-date on regulatory changes or protocol deviations.

3. Boost Clinical Trial Participation with Thoughtful Site Design: Amenities, Wearables, Convenience, and Support

• Accessibility
• Safety Considerations
• Personal Risk and Fear Support
• Patient-Centric Clinical Design and Logistics

Determining how to select and set up a clinical site is not a size-fit approach. Industry experts suggest that Principal Investigators who take dogmatic stances in how they set up their clinical sites may be missing the mark when it comes to optimizing patient experience and outcomes.

If Covid-19 taught us anything, it is that sometimes gold can be found in a more non-linear adaptive design strategy. [ 5 ] Studies indicate that the lack of CTP (clinical trial participant) engagement and participation results in low recruitment numbers and problems in retention. [ 6 ]

This translates to thoughtful considerations towards every aspect of the clinical trial from parking to laboratory tests. Understanding that participant compliance is heavily associated with patient experience is an inconvenient truth many clinical trial teams are now coming to grips with. Because non-compliance has such strong implications for the study, Principal investigators are tasked with the incredibly difficult job of incorporating patient and budget-friendly solutions into the site plan. [ 7 ]

Healthcare in general is undergoing a massive shift towards improving the overall patient experience. In recent years, precision medicine has gained momentum. We have learned that by empowering patients in their own healthcare decisions, we also improve safety, adherence, and clinical outcomes. [ 8 , 9 ]

The best takeaways can be best articulated in a series of next-action responses to help you determine apply your knowledge in a way that will unlock the untapped solutions.

• Do you have a measure of flexibility included in your site plan? If so, what is it? Does it need refining? And if not, your next action is to include a response action for different scenarios in your site mockup visits.
• Examine your budget allowances. The principal investigator’s next action response should be tailored towards accounting for any unplanned expenses and gaps that may arise OR if there are opportunities for funding enhancements on the part of clinical sponsors. Preplanning will bring clarity to these decisions.
• Wearables and Digital Solutions: Next action responses should be research, implementation, and accuracy testing and reporting schematics.

Understandably, setting up a clinical trial can be daunting for principal investigators and their respective teams. Furthermore, it is clear how important the patient perspective is to the process. Finding avenues that work for the clinical study you are involved in might be akin to a creative endeavor where non-traditional approaches may work best.

It is also noteworthy to emphasize that digital solutions are expanding the potential for clinical trials globally. Clinical trial site management is entering an exciting new phase of innovation making it easier to explore what is possible within the virtual trial landscape. With this, we wish you success in the New Year!

1. National Academies of Sciences, Engineering, and Medicine (NASEM). 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop . Washington, DC: The National Academies Press.

2. Marra, Caroline, Jacqueline L. Chen, Andreas Coravos, and Ariel D. Stern. 2020. “ Quantifying the use of connected digital products in clinical research. ” npj Digital Medicine 3:50.

3. Lam, S. Sam, Alan J. Kivitz, Doug McKinnell, M. Edward Pierson, and Faye S. O’Brien. 2017. “ Simulating clinical trial visits yields patient insights into study design and recruitment. ” Patient Preference and Adherence 11:1295-07.

4. Lam, S. Sam, Alan J. Kivitz, Doug McKinnell, M. Edward Pierson, and Faye S. O’Brien. 2017. “ Simulating clinical trial visits yields patient insights into study design and recruitment. ” Patient Preference and Adherence 11:1295-07.

5. Breckenridge, Alasdair, Jeffrey K. Aronson, Terrence F. Blaschke, Dan Hartman, Carl C. Peck, and Bernard Vrijens. 2017. “ Poor medication adherence in clinical trials: consequences and solutions. ” Nature Reviews Drug Discovery 16(3):149-50.

6. Ashish Kadam, Rashmi, Sanghratna Umakant Borde, Sapna Amol Madas, Sundeep Santosh Salvi, and Sneha Saurabh Limaye. 2016. “ Challenges in recruitment and retention of clinical trial subjects. ”  Perspectives in Clinical Research  7(3):137-43.

7. Paul, Varun, Lori Ferranti, and David M. Dilts. 2022. “ Using a clinical trial schedule of assessments (SOA) as a quality improvement tool. ” Journal of Clinical Oncology 40(16_suppl):e18638.

8. Lam, S. Sam, Alan J. Kivitz, Doug McKinnell, M. Edward Pierson, and Faye S. O’Brien. 2017. “ Simulating clinical trial visits yields patient insights into study design and recruitment. ” Patient Preference and Adherence 11:1295-07.

9. Schindler, Thomas M., Frank Grieger, Anna Zak, Ramona Rorig, Kavya Chowdary Konka, Anna Ellsworth, Christopher Pfitzer, Keir Hodge, and Christine Crandall. 2020. “ Patient preferences when searching for clinical trials and adherence of study records to ClinicalTrials.gov guidance in key registry data fields. ” PloS One 15(5):e0233294.

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How to Start a Clinical Research Business: A Comprehensive Guide

Do you dream of starting your own clinical research business, but don’t know where to begin? Fear not, my friend! Starting any business can be a daunting task, but with the right information and guidance, it can also be a rewarding and satisfying journey. In this article, we will explore the steps you need to take to start your own clinical research business.

First things first, you need to have a solid plan. This means conducting research, understanding your target market, and creating a business plan that outlines your goals and objectives. Once you have a clear plan in place, the next step is to acquire the necessary knowledge and expertise in the clinical research field. This may involve continuing education, networking, and gaining experience through internships or working for other companies within the industry.

Finally, it’s time to take action and start building your business. This involves setting up your physical space, creating a website and marketing materials, and developing relationships with industry contacts and potential clients. Remember, starting any business requires hard work, dedication, and a willingness to adapt to changing circumstances. With a solid plan, the right skills, and the determination to succeed, you too can start your own successful clinical research business. Conducting a Market Analysis

Before starting a clinical research business, it is crucial to conduct a thorough market analysis. This involves researching the market and competitors to identify opportunities and potential challenges that may affect the success of your business.

• Research the target market: You need to identify the demographics, behaviors, and needs of the target market. Understanding the needs of your prospective patients and service providers will enable you to customize your services to meet those needs, thus increasing your chances of success.
• Determine the current trends and demands: Identifying the current trends and demands in the market will enable you to develop services that meet those needs.
• Identify potential competitors: Research your competitors to determine their strengths, weaknesses, and pricing strategies. This information will help you position your services competitively.

To conduct a more in-depth analysis, it’s recommended to create SWOT analysis. This technique will help you identify the strengths, weaknesses, opportunities, and threats that affect your business.

Your potential business partners and investors will also want to see the results of your market analysis, so invest time and resources into this aspect of your business planning.

Developing a Business Plan

Developing a business plan is essential for starting a clinical research business. The business plan outlines the company’s goals, strategies, and financial projections, and helps investors and stakeholders understand the nature and potential of the business.

A well-crafted business plan should include the following:

• Executive summary – a concise overview of the business and its objectives.
• Company description – a detailed description of the business, including its history, mission, and services.
• Market analysis – a study of the target market, competitors, and industry trends.
• Services and products – a breakdown of the products and services offered by the business.
• Marketing and sales strategies – a plan for promoting and selling the business’s products and services.
• Management structure – an overview of the management and organizational structure of the business.
• Financial projections – a detailed financial analysis of the business, including revenue, expenses, and profit projections.
• Appendix – supporting documents and information, such as resumes, contracts, and market research data.

In addition to these elements, the business plan should also consider potential risks and contingencies, such as changes in regulations or unexpected market shifts. It should also include a timeline for achieving goals and milestones, as well as a plan for adapting and revising the plan as necessary.

Developing a business plan is a crucial step in starting a successful clinical research business. A comprehensive and well-researched plan can help attract investors and stakeholders, guide decision making, and ensure the long-term success of the business.

Overall, a well-developed business plan is an essential tool for starting and maintaining a thriving clinical research business.

Legal Considerations

Starting a clinical research business involves complying with legal requirements. Understanding the legal considerations is crucial to avoid legal liabilities and ensure the safety of participants in your clinical trials. Below are some of the important legal considerations:

• Good Clinical Practice (GCP) regulations: GCP regulations are a set of international ethical and scientific quality standards for designing, conducting, recording, and reporting clinical research involving human subjects. Compliance with GCP regulations ensures that the rights, safety, and well-being of clinical trial participants are protected. It is important to ensure that your clinical research business complies with GCP regulations.
• Obtaining regulatory approvals: Before starting any clinical trial, regulatory approvals must be obtained from the relevant authorities in your country. Regulatory approvals include approval from the ethics committee or institutional review board (IRB) and approval from the regulatory agency responsible for the oversight of clinical trials (such as the FDA in the United States). Failure to obtain regulatory approvals can result in legal liabilities, fines, and even criminal charges.
• Intellectual property protection: Protecting intellectual property (IP) is crucial in any business, including clinical research. The IP in clinical research includes patents, trademarks, copyrights, and trade secrets. Protecting your IP ensures that your business maintains its competitive edge and gains recognition in the market. You can protect your IP by obtaining patents and trademarks, registering copyrights, and maintaining confidentiality agreements and nondisclosure agreements (NDAs) with employees and stakeholders.

It is important to consult with legal experts to ensure compliance with all legal requirements when starting a clinical research business. Additionally, you should keep yourself updated with the changing legal landscape relating to clinical research, by subscribing to relevant publications and attending conferences and seminars.

Below is a table showing some of the legal documents that are required for conducting clinical trials:

Understanding the legal considerations when starting a clinical research business is essential for the success of your business. By complying with the legal requirements, you can ensure the safety of your participants, protect your IP, and avoid legal liabilities.

Regulatory Compliance

When starting a clinical research business, regulatory compliance is crucial. All clinical research must adhere to regulations and guidelines set by governing bodies such as the Food and Drug Administration (FDA), the International Conference on Harmonisation (ICH), and the Institutional Review Board (IRB). Failure to comply with these regulations can lead to legal action, loss of credibility, and harm to study participants.

• Develop a thorough understanding of all regulatory requirements and guidelines before beginning any clinical research.
• Ensure that all study documents and procedures adhere to these regulations and guidelines.
• Implement quality assurance processes to ensure ongoing compliance throughout the study.

It is important to note that regulatory requirements and guidelines can vary depending on the type of research being conducted, such as studies involving drugs or medical devices. Consulting with regulatory experts and seeking advice from governing bodies can help ensure compliance and reduce the risk of non-compliance.

IRB Approval

The Institutional Review Board (IRB) plays an integral role in the clinical research process. All studies involving human subjects must be reviewed and approved by an IRB to ensure that the study is ethical and that the rights and welfare of participants are protected.

When starting a clinical research business, it is important to have a thorough understanding of the IRB approval process. This includes:

• Preparing and submitting a comprehensive study protocol to the IRB for review
• Addressing any concerns or questions raised by the IRB during the review process
• Following all IRB requirements and guidelines during the study

Data Management

Data management is a critical component of clinical research. Proper management and documentation of study data ensures accuracy, security, and compliance with regulatory requirements. This includes:

• Establishing data management processes and protocols before beginning the study
• Using secure and reliable data management systems that meet regulatory requirements
• Ensuring all study data is properly documented and stored, including informed consent forms and case report forms

By implementing best practices for data management, clinical research businesses can ensure regulatory compliance and produce high-quality data for analysis and reporting.

Staffing and Recruitment

One of the most crucial aspects of starting a clinical research business is staffing and recruitment. Human resources are the backbone of any organization, and it is imperative to have the right team in place. Here are some essential steps to follow when staffing and recruiting for your clinical research business:

• Define the job description – Before starting the recruitment process, create a clear job description that outlines the responsibilities, duties, and requirements of the position. Make sure the job description is specific and detailed to attract the right candidates.
• Create a competitive compensation package – Talented individuals are in high demand, and it is essential to offer a competitive compensation package that includes health benefits, retirement plans, bonuses, and other incentives.
• Utilize online job postings – Use online job boards such as LinkedIn, Indeed, and Glassdoor to find potential candidates. These platforms have a vast pool of job seekers and allow you to filter candidates based on education, experience, and location.

Another effective way to recruit talent is through referrals. Ask your industry peers, colleagues, and friends if they know anyone who fits the job description. Offering a referral bonus can encourage individuals to refer qualified candidates to your organization.

Once you have received applications, the next step is to conduct a thorough screening process. This may include reviewing resumes and cover letters, conducting phone interviews, and scheduling in-person interviews. During the interview process, assess the candidate’s fit for the role and company culture, their problem-solving abilities, and communication skills.

Finally, after selecting the right candidate, it is essential to provide adequate training to ensure they are equipped to handle the job and have a clear understanding of the company’s goals and objectives.

By following these steps and putting the right recruitment and staffing strategies in place, your clinical research business can attract and retain top talent, leading to a successful and thriving business.

Identifying Funding Opportunities

Identifying funding opportunities is one of the most important steps to establish a successful clinical research business. Funding opportunities help researchers support their projects and contribute to expanding scientific knowledge. Here are some techniques to help in identifying funding opportunities:

• Search for federal funding options – The federal government provides funding for a wide range of research projects. The National Institutes of Health (NIH) is the largest provider of biomedical research funding in the United States. Grants.gov is another online platform where you can find a listing of federal grants available for different categories.
• Explore private foundations – Private foundations also provide funding for clinical research projects. Examples include the Bill and Melinda Gates Foundation, the Susan G. Komen Foundation, and the American Cancer Society. These foundations usually have specific areas of interest and eligibility criteria for applicants.
• Network with industry partners – Networking with industry partners is another valuable strategy for identifying funding opportunities. Industry partners often invest in research projects that align with their business strategies. Collaboration with industry partners can also offer researchers access to valuable resources and expertise.

Once funding opportunities have been identified, it is crucial to follow the guidelines carefully and ensure that the application meets the eligibility criteria. Additionally, researchers should ensure that they have available resources and equipment to carry out their project if funding is awarded.

Establishing Partnerships and Collaborations

Starting a clinical research business requires a substantial amount of investment, both in terms of finances and time. Establishing partnerships and collaborations with other businesses can be advantageous for startups to share responsibilities and resources. Here are some ways to establish partnerships and collaborations:

• Identify Potential Partners: Before establishing partnerships, it is essential to identify potential partners, such as pharmaceutical companies, contract research organizations, and academic institutions, among others.
• Assess Their Capabilities: Evaluating the capabilities of potential partners is vital to avoid any conflicts that may arise during the partnership. Consider their expertise, experience, financial standing, and reputation.
• Outline Terms and Conditions: After identifying and evaluating partners, draw up an agreement that outlines the terms and conditions of the partnership. Such agreements should include the scope of work, responsibilities, financial contributions, and timelines.

Formal partnerships and collaborations can take different forms, such as joint ventures, strategic alliances, and licensing agreements. These collaborations can foster research and development, knowledge sharing, and the pooling of financial resources to achieve common goals. For example, an academic institution may partner with a clinical research organization to conduct a clinical trial on a new drug or therapy, and share the results with the pharmaceutical industry.

Table 1 below shows some benefits of establishing partnerships and collaborations:

Establishing partnerships and collaborations is an effective way to grow a clinical research business. It enables startups to share risks, costs, and resources, and leverage the expertise and knowledge of other businesses. However, partnerships and collaborations require effective communication, clear agreements, and mutual trust to achieve the goals of all parties involved.

Developing Standard Operating Procedures (SOPs)

Standard Operating Procedures (SOPs) are comprehensive documents that outline how a specific task or process should be executed. SOPs are critical for clinical research as they ensure consistency, accuracy, and compliance with regulatory standards. Here are 8 steps to developing effective SOPs for your clinical research business:

• Identify the task: Determine which tasks require an SOP and prioritize them.
• Involve the experts: Invite personnel with the necessary expertise to draft the SOP.
• Gather references: Collect relevant regulations, guidelines, and research papers to support the SOP.
• Define the scope: Outline the objective, purpose, and intended audience of the SOP.
• Develop the format: Decide on the structure, style, and format of the SOP, ensuring it is user-friendly and easy to update.
• Write the content: Draft the SOP in a clear and concise language, specifying the steps, responsibilities, timelines, and possible risks involved.
• Validate the SOP: Review and revise the SOP with all stakeholders to ensure it reflects their feedback and is compliant with regulations.
• Implement and train: Communicate the SOP to everyone involved in the task and provide the necessary training to ensure they understand and follow it.

Following these steps will result in the development of effective SOPs that are essential for the smooth and compliant execution of clinical research tasks.

Moreover, SOPs should be periodically reviewed, updated, and revised to ensure they remain relevant and compliant with changing regulations and guidelines. A documented process should also be in place for revisions and updates to maintain compliance throughout the clinical research process.

Implementing effective SOPs is critical to the success and reputation of your clinical research business. It demonstrates your commitment to quality, ethics, and regulatory compliance, and sets a foundation for the growth and sustainability of your business.

Implementing Quality Assurance and Quality Control Measures

Implementing quality assurance and quality control measures is an essential step in starting a clinical research business. Quality assurance (QA) involves ensuring that all processes and procedures are conducted in a systematic, standardized, and consistent manner. Quality control (QC) involves monitoring and verifying that the processes and procedures are implemented correctly and producing accurate and reliable outcomes.

• Developing Standard Operating Procedures (SOPs) – SOPs are a set of written instructions that outline the steps for carrying out a specific task. Developing SOPs for all clinical research activities ensures that all staff is aware of the processes and procedures that need to be followed, and they are implemented consistently.
• Training Staff – Staff needs to be appropriately trained to understand and implement the SOPs. Training should be provided to both new and existing employees to ensure that they are up-to-date with the latest procedures and policies.
• Assigning Roles and Responsibilities – A clear delineation of roles and responsibilities ensures that all staff knows their responsibilities and the accountability for specific tasks.

Another critical aspect is monitoring and verifying that the processes and procedures are correct and producing accurate outcomes. This process is known as Quality Control (QC). To implement QC effectively, you need to establish a Quality control unit (QCU), responsible for assessing quality control measures. The QCU follows the following steps to ensure the integrity and accuracy of research data:

• Implementing Data Cleaning Procedures – Data cleaning is the process of identifying and correcting errors in collected data. A well-designed data cleaning protocol ensures the accuracy and integrity of the data for analysis.
• Performing Site Visits – Site visits ensure that data is being collected and recorded accurately and consistently. They also help to monitor study compliance and detect problems early.
• Adverse Event Reporting and Monitoring – Adverse events are unexpected or unfavorable outcomes that may occur during a clinical trial or research study. A system must be in place to report and monitor adverse events to minimize the risk of harm to patients.

Overall, implementing Quality Assurance and Quality Control measures is essential to ensure that clinical research activities are conducted appropriately, accurately, and ethically. A poorly designed and executed study not only compromises patient safety but also impacts the reliability of the data and the credibility of the research.

Therefore, a well-conducted study ensures that data collected is accurate and useful, leading to reliable results and conclusions. Implementing QA/QC measures are the key to achieving these objectives.

Identifying Potential Clients and Developing Marketing Strategies

In the clinical research business, identifying potential clients is a crucial first step in developing effective marketing strategies. Here are ten essential steps to identify potential clients:

• 1. Identify your target market: Knowing your target market helps you focus your marketing efforts on specific clients who require your services.
• 2. Conduct market research: Research your potential clients to find out their challenges, needs, pain points, and how your services can solve their problems.
• 3. Know your competitors: Identify your competitors and what distinguishes your services, and how you can offer value-added services to your clients.
• 4. Build a brand: Develop a brand that distinguishes you from your competitors and aligns with the needs of your target market.
• 5. Attend conferences and events: Attend conferences and events relevant to your target market to network and identify potential clients.
• 6. Leverage social media: Use social media platforms to publish relevant content, engage with your audience, and drive traffic to your website.
• 7. Build a website: A well-designed website that highlights your services and positions you as a thought leader in the industry helps you attract potential clients.
• 8. Use email marketing: Build an email list of potential clients and use email campaigns to inform them about your services and promotions.
• 9. Develop partnerships: Build strategic partnerships with other businesses in the industry to increase your reach and identify potential clients.
• 10. Offer exceptional customer service: Delivering exceptional customer service helps you retain clients, get referrals, and build a positive reputation in the industry.

Once you have identified potential clients, the next step is to develop marketing strategies that resonate with them. These strategies should communicate how your services solve their challenges, and how your company distinguishes itself from competitors. Here are a few proven marketing strategies:

• Create a blog: Publish educational and informative content that establishes you as a thought leader and gives you credibility with potential clients.
• Offer case studies: Case studies showcase your experience in solving real problems for clients and validate your services to potential clients.
• Run paid ads: Paid ads on social media platforms and search engines help you increase your reach and attract potential clients.

Remember, developing effective marketing strategies requires a deep understanding of your potential clients and how you can help them solve their problems. Stay committed to understanding your target audience, and your efforts will pay off in the form of new clients and revenue growth.

Identifying potential clients and developing marketing strategies can be a complex process that requires constant refinement. However, by staying committed to understanding your target audience and delivering exceptional services, you can establish a reputation as a reliable provider of clinical research services and drive growth for your business.

Frequently Asked Questions – Starting a Clinical Research Business

1. what qualifications do i need to start a clinical research business.

To get started in this field, you will typically need at least a bachelor’s degree in a related field such as biology, chemistry, or healthcare administration. Having additional certifications or previous experience in clinical research can also be very helpful.

2. What kind of funding is required to start a clinical research business?

Starting a clinical research business can be quite expensive, given the need for specialized equipment and software. Some common funding options include angel investors, venture capitalists, and government grants.

3. How do I go about finding potential clients for my clinical research business?

Potential clients typically include pharmaceutical companies, medical device manufacturers, and academic research institutions. You can start by networking with professionals in these fields or attending industry conferences and events.

4. What are some common challenges I may face when starting my own clinical research business?

Some common challenges include the need for a lot of funding upfront, navigating complex regulations and guidelines, and finding and retaining top talent. It’s important to plan for these challenges and develop strategies to overcome them.

5. How can I ensure that my clinical research business is operating ethically and within all regulatory guidelines?

It’s important to stay up-to-date on all relevant regulations and guidelines, and to work with experienced consultants or advisors who can help ensure that your business is operating ethically and within the law.

6. What are some key factors that will determine the success of my clinical research business?

Key factors include your ability to attract and retain high-quality talent, develop strong relationships with clients, stay on top of the latest advances and trends in the field, and successfully manage your finances.

7. How long does it typically take for a clinical research business to become profitable?

This can vary widely depending on a variety of factors, including the services you offer, the size of your client base, and your overall financial management. In general, it can take several years to become profitable in this field.

Closing Thoughts

We hope this guide has been helpful in getting you started on the path to starting your own clinical research business! Remember to stay committed, plan carefully, and seek out the support and resources you need along the way. Thanks for reading, and we hope to see you back here soon!

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Business continuity plan for clinical trial sites: why electronic source matters during covid-19.

The impact of COVID-19 on clinical trials has likely become obvious for your site during this phase of the pandemic. Sites are struggling with screening holds, limited staff, an influx of sponsor requests while staff is not on-site, the challenge of retaining enrolled subjects, and more. It is now more important than ever to implement a business continuity plan for reopening your clinical research site. Be prepared for potential new waves of COVID-19 that could result in individual staff members being quarantined or the site having to take precautionary measures. This means that on short notice the site must be in a position to limit patient-staff interactions, and ensure that the PI and Sponsor representatives have remote access to patient data.

Before joining  Clinical Research IO , I was a manager at a women’s health site. I oversaw the selection and implementation of the CRIO eSource – eRegulatory – CTMS system at my site. I knew this was the right decision because it allowed us to control quality and improve efficiency. But when the pandemic hit and we had to modify our procedures, I really  knew that we had made the right decision. Unlike many sites I knew, we were able to adapt very quickly to the new circumstances, maintaining our productivity while enabling remote monitoring for our sponsors.

With CRIO, the system fully integrated all of our modules into one log-in: Scheduling,  eSource ,  eRegulatory , Finance, and Recruitment. We could collect data electronically during the visit and then review and query that data remotely and in real-time. We could give read-only access as needed, and restrict access to different modules to different users. We could give our monitors instant access only to the studies they were assigned to and view and respond to their queries remotely. These capabilities became life-savers when we had to institute work-from-home and prohibit monitors from visiting our site.

Even though parts of our country are opening up again, most epidemiologists are forecasting future waves of the pandemic, which could necessitate many of the same measures sites have recently undertaken – work-from-home, virtual visits, restricted monitor travel, etc. Having CRIO implemented at your site will allow you to adapt instantly to these changes.

Here are the things we were able to do with CRIO during the pandemic:

1. We easily minimized the number of research site staff who had to be onsite:

• Coordinators took separate shifts that did not overlap. When one person was in the office seeing patients, other personnel could be at home, doing EDC entry, answering queries, processing regulatory documents or documenting phone call visits to patients.
• Our investigators could stay home and enter progress notes, conduct phone visits and record the source in real-time, and sign off on labs and visit notes.
• Our finance person could track receivables in real-time, as the data was being completed, and send invoices in a timely manner so as to maintain cash flow. We could see in an instant which invoices and visits were overdue on payment, and take appropriate follow up.

If you’d like to learn more about how CRIO-enabled clinical trial sites are allowing their staff to work remotely, schedule a demo with our team to learn more about how CRIO’s CTMS.

2. CRCs, Lab staff, and Investigators had access to the subject chart at the same time.

PIs could write a progress note immediately after seeing a patient while the CRC finished the visit. Lab techs were able to document blood draw times while the coordinator uploaded relevant visit files.

3. We were able to give our CRAs remote monitoring access at any time.

Our CRAs could view our data and leave their queries behind, and we could respond to and close out those queries from home. By the way, because of the built-in edit checks, we had a lot fewer queries to address, and saved a lot of time not having to scan and email source in.

4. We were able to adapt our eSource on the fly.

This way we could complete certain procedures on-site and certain procedures, and/or visits remotely. We were able to implement virtual visits and at-home deliveries of IP by creating appropriate procedures, adding them to our library, then adding them as needed to the appropriate studies.

5. We used CRIO’s automated text reminder feature to add COVID screening questions…

And a reminder to patients to wear a mask to their visit!

6. We could build and add a COVID-19 screening questionnaire per CDC guidelines (temperature check, etc.) to the beginning of each study visit.

Using that screening questionnaire, we could then generate an invoiceable charge to the sponsor, if that’s what our clinical trial agreement allowed.

No one could have predicted that we would be dealing with something as disruptive and devastating as COVID-19. Having CRIO really allowed us to adapt our processes while maintaining data quality and PI oversight. Based on this experience, I became such a strong advocate of the CRIO system that I joined their team – my job is to help other sites implement the system and gain the efficiencies I experienced firsthand.

If you’d like to learn more, you can talk directly to our team here .

21 CFR Part 11 Regulation Compliance Update

Compliance Update:  The 21 CFR Part 11 Regulation is a cornerstone of conducting clinical trials in today’s world.  The release of the regulation 1997 established guidelines for the use of electronic records and electronic signatures in FDA-regulated industries and had a significant impact on the pharmaceutical and medical device industries. The FDA began working on...

The Current State of Clinical Trials in Ukraine—A Conversation with Dr. Roman Fishchuk

Meet Dr. Roman Fishchuk, an esteemed otorhinolaryngologist (ENT) whose journey in the healthcare field has been marked by a commitment to providing medical care and innovative treatment options through clinical trials in his native Ukraine. Having graduated from Ivano-Frankivsk National Medical University, Dr. Fishchuk specialized in otolaryngology and completed a Master’s degree at the University...

Transitioning to CRIO? How to Get Your Data Ready for Migration

You have just signed on to use CRIO and you are excited to get started. Even if you are moving primarily from paper, you may have data that you want to migrate into CRIO rather than having to re-enter it. For many of our new clients, they have a wealth of information available in spreadsheets...

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Clinical Trials and Clinical Research: A Comprehensive Review

Venkataramana kandi.

1 Clinical Microbiology, Prathima Institute of Medical Sciences, Karimnagar, IND

Sabitha Vadakedath

2 Biochemistry, Prathima Institute of Medical Sciences, Karimnagar, IND

Clinical research is an alternative terminology used to describe medical research. Clinical research involves people, and it is generally carried out to evaluate the efficacy of a therapeutic drug, a medical/surgical procedure, or a device as a part of treatment and patient management. Moreover, any research that evaluates the aspects of a disease like the symptoms, risk factors, and pathophysiology, among others may be termed clinical research. However, clinical trials are those studies that assess the potential of a therapeutic drug/device in the management, control, and prevention of disease. In view of the increasing incidences of both communicable and non-communicable diseases, and especially after the effects that Coronavirus Disease-19 (COVID-19) had on public health worldwide, the emphasis on clinical research assumes extremely essential. The knowledge of clinical research will facilitate the discovery of drugs, devices, and vaccines, thereby improving preparedness during public health emergencies. Therefore, in this review, we comprehensively describe the critical elements of clinical research that include clinical trial phases, types, and designs of clinical trials, operations of trial, audit, and management, and ethical concerns.

Introduction and background

A clinical trial is a systematic process that is intended to find out the safety and efficacy of a drug/device in treating/preventing/diagnosing a disease or a medical condition [ 1 , 2 ]. Clinical trial includes various phases that include phase 0 (micro-dosing studies), phase 1, phase 2, phase 3, and phase 4 [ 3 ]. Phase 0 and phase 2 are called exploratory trial phases, phase 1 is termed the non-therapeutic phase, phase 3 is known as the therapeutic confirmatory phase, and phase 4 is called the post-approval or the post-marketing surveillance phase. Phase 0, also called the micro-dosing phase, was previously done in animals but now it is carried out in human volunteers to understand the dose tolerability (pharmacokinetics) before being administered as a part of the phase 1 trial among healthy individuals. The details of the clinical trial phases are shown in Table ​ Table1 1 .

This table has been created by the authors.

MTD: maximum tolerated dose; SAD: single ascending dose; MAD: multiple ascending doses; NDA: new drug application; FDA: food and drug administration

Clinical research design has two major types that include non-interventional/observational and interventional/experimental studies. The non-interventional studies may have a comparator group (analytical studies like case-control and cohort studies), or without it (descriptive study). The experimental studies may be either randomized or non-randomized. Clinical trial designs are of several types that include parallel design, crossover design, factorial design, randomized withdrawal approach, adaptive design, superiority design, and non-inferiority design. The advantages and disadvantages of clinical trial designs are depicted in Table ​ Table2 2 .

There are different types of clinical trials that include those which are conducted for treatment, prevention, early detection/screening, and diagnosis. These studies address the activities of an investigational drug on a disease and its outcomes [ 4 ]. They assess whether the drug is able to prevent the disease/condition, the ability of a device to detect/screen the disease, and the efficacy of a medical test to diagnose the disease/condition. The pictorial representation of a disease diagnosis, treatment, and prevention is depicted in Figure ​ Figure1 1 .

This figure has been created by the authors.

The clinical trial designs could be improvised to make sure that the study's validity is maintained/retained. The adaptive designs facilitate researchers to improvise during the clinical trial without interfering with the integrity and validity of the results. Moreover, it allows flexibility during the conduction of trials and the collection of data. Despite these advantages, adaptive designs have not been universally accepted among clinical researchers. This could be attributed to the low familiarity of such designs in the research community. The adaptive designs have been applied during various phases of clinical trials and for different clinical conditions [ 5 , 6 ]. The adaptive designs applied during different phases are depicted in Figure ​ Figure2 2 .

The Bayesian adaptive trial design has gained popularity, especially during the Coronavirus Disease-19 (COVID-19) pandemic. Such designs could operate under a single master protocol. It operates as a platform trial wherein multiple treatments can be tested on different patient groups suffering from disease [ 7 ].

In this review, we comprehensively discuss the essential elements of clinical research that include the principles of clinical research, planning clinical trials, practical aspects of clinical trial operations, essentials of clinical trial applications, monitoring, and audit, clinical trial data analysis, regulatory audits, and project management, clinical trial operations at the investigation site, the essentials of clinical trial experiments involving epidemiological, and genetic studies, and ethical considerations in clinical research/trials.

A clinical trial involves the study of the effect of an investigational drug/any other intervention in a defined population/participant. The clinical research includes a treatment group and a placebo wherein each group is evaluated for the efficacy of the intervention (improved/not improved) [ 8 ].

Clinical trials are broadly classified into controlled and uncontrolled trials. The uncontrolled trials are potentially biased, and the results of such research are not considered as equally as the controlled studies. Randomized controlled trials (RCTs) are considered the most effective clinical trials wherein the bias is minimized, and the results are considered reliable. There are different types of randomizations and each one has clearly defined functions as elaborated in Table ​ Table3 3 .

Principles of clinical trial/research

Clinical trials or clinical research are conducted to improve the understanding of the unknown, test a hypothesis, and perform public health-related research [ 2 , 3 ]. This is majorly carried out by collecting the data and analyzing it to derive conclusions. There are various types of clinical trials that are majorly grouped as analytical, observational, and experimental research. Clinical research can also be classified into non-directed data capture, directed data capture, and drug trials. Clinical research could be prospective or retrospective. It may also be a case-control study or a cohort study. Clinical trials may be initiated to find treatment, prevent, observe, and diagnose a disease or a medical condition.

Among the various types of clinical research, observational research using a cross-sectional study design is the most frequently performed clinical research. This type of research is undertaken to analyze the presence or absence of a disease/condition, potential risk factors, and prevalence and incidence rates in a defined population. Clinical trials may be therapeutic or non-therapeutic type depending on the type of intervention. The therapeutic type of clinical trial uses a drug that may be beneficial to the patient. Whereas in a non-therapeutic clinical trial, the participant does not benefit from the drug. The non-therapeutic trials provide additional knowledge of the drug for future improvements. Different terminologies of clinical trials are delineated in Table ​ Table4 4 .

In view of the increased cost of the drug discovery process, developing, and low-income countries depend on the production of generic drugs. The generic drugs are similar in composition to the patented/branded drug. Once the patent period is expired generic drugs can be manufactured which have a similar quality, strength, and safety as the patented drug [ 9 ]. The regulatory requirements and the drug production process are almost the same for the branded and the generic drug according to the Food and Drug Administration (FDA), United States of America (USA).

The bioequivalence (BE) studies review the absorption, distribution, metabolism, and excretion (ADME) of the generic drug. These studies compare the concentration of the drug at the desired location in the human body, called the peak concentration of the drug (Cmax). The extent of absorption of the drug is measured using the area under the receiver operating characteristic curve (AUC), wherein the generic drug is supposed to demonstrate similar ADME activities as the branded drug. The BE studies may be undertaken in vitro (fasting, non-fasting, sprinkled fasting) or in vivo studies (clinical, bioanalytical, and statistical) [ 9 ].

Planning clinical trial/research

The clinical trial process involves protocol development, designing a case record/report form (CRF), and functioning of institutional review boards (IRBs). It also includes data management and the monitoring of clinical trial site activities. The CRF is the most significant document in a clinical study. It contains the information collected by the investigator about each subject participating in a clinical study/trial. According to the International Council for Harmonisation (ICH), the CRF can be printed, optical, or an electronic document that is used to record the safety and efficacy of the pharmaceutical drug/product in the test subjects. This information is intended for the sponsor who initiates the clinical study [ 10 ].

The CRF is designed as per the protocol and later it is thoroughly reviewed for its correctness (appropriate and structured questions) and finalized. The CRF then proceeds toward the print taking the language of the participating subjects into consideration. Once the CRF is printed, it is distributed to the investigation sites where it is filled with the details of the participating subjects by the investigator/nurse/subject/guardian of the subject/technician/consultant/monitors/pharmacist/pharmacokinetics/contract house staff. The filled CRFs are checked for their completeness and transported to the sponsor [ 11 ].

Effective planning and implementation of a clinical study/trial will influence its success. The clinical study majorly includes the collection and distribution of the trial data, which is done by the clinical data management section. The project manager is crucial to effectively plan, organize, and use the best processes to control and monitor the clinical study [ 10 , 11 ].

The clinical study is conducted by a sponsor or a clinical research organization (CRO). A perfect protocol, time limits, and regulatory requirements assume significance while planning a clinical trial. What, when, how, and who are clearly planned before the initiation of a study trial. Regular review of the project using the bar and Gantt charts, and maintaining the timelines assume increased significance for success with the product (study report, statistical report, database) [ 10 , 11 ].

The steps critical to planning a clinical trial include the idea, review of the available literature, identifying a problem, formulating the hypothesis, writing a synopsis, identifying the investigators, writing a protocol, finding a source of funding, designing a patient consent form, forming ethics boards, identifying an organization, preparing manuals for procedures, quality assurance, investigator training and initiation of the trial by recruiting the participants [ 10 ].

The two most important points to consider before the initiation of the clinical trial include whether there is a need for a clinical trial, if there is a need, then one must make sure that the study design and methodology are strong for the results to be reliable to the people [ 11 ].

For clinical research to envisage high-quality results, the study design, implementation of the study, quality assurance in data collection, and alleviation of bias and confounding factors must be robust [ 12 ]. Another important aspect of conducting a clinical trial is improved management of various elements of clinical research that include human and financial resources. The role of a trial manager to make a successful clinical trial was previously reported. The trial manager could play a key role in planning, coordinating, and successfully executing the trial. Some qualities of a trial manager include better communication and motivation, leadership, and strategic, tactical, and operational skills [ 13 ].

Practical aspects of a clinical trial operations

There are different types of clinical research. Research in the development of a novel drug could be initiated by nationally funded research, industry-sponsored research, and clinical research initiated by individuals/investigators. According to the documents 21 code of federal regulations (CFR) 312.3 and ICH E-6 Good Clinical Practice (GCP) 1.54, an investigator is an individual who initiates and conducts clinical research [ 14 ]. The investigator plan, design, conduct, monitor, manage data, compile reports, and supervise research-related regulatory and ethical issues. To manage a successful clinical trial project, it is essential for an investigator to give the letter of intent, write a proposal, set a timeline, develop a protocol and related documents like the case record forms, define the budget, and identify the funding sources.

Other major steps of clinical research include the approval of IRBs, conduction and supervision of the research, data review, and analysis. Successful clinical research includes various essential elements like a letter of intent which is the evidence that supports the interest of the researcher to conduct drug research, timeline, funding source, supplier, and participant characters.

Quality assurance, according to the ICH and GCP guidelines, is necessary to be implemented during clinical research to generate quality and accurate data. Each element of the clinical research must have been carried out according to the standard operating procedure (SOP), which is written/determined before the initiation of the study and during the preparation of the protocol [ 15 ].

The audit team (quality assurance group) is instrumental in determining the authenticity of the clinical research. The audit, according to the ICH and GCP, is an independent and external team that examines the process (recording the CRF, analysis of data, and interpretation of data) of clinical research. The quality assurance personnel are adequately trained, become trainers if needed, should be good communicators, and must handle any kind of situation. The audits can be at the investigator sites evaluating the CRF data, the protocol, and the personnel involved in clinical research (source data verification, monitors) [ 16 ].

Clinical trial operations are governed by legal and regulatory requirements, based on GCPs, and the application of science, technology, and interpersonal skills [ 17 ]. Clinical trial operations are complex, time and resource-specific that requires extensive planning and coordination, especially for the research which is conducted at multiple trial centers [ 18 ].

Recruiting the clinical trial participants/subjects is the most significant aspect of clinical trial operations. Previous research had noted that most clinical trials do not meet the participant numbers as decided in the protocol. Therefore, it is important to identify the potential barriers to patient recruitment [ 19 ].

Most clinical trials demand huge costs, increased timelines, and resources. Randomized clinical trial studies from Switzerland were analyzed for their costs which revealed approximately 72000 USD for a clinical trial to be completed. This study emphasized the need for increased transparency with respect to the costs associated with the clinical trial and improved collaboration between collaborators and stakeholders [ 20 ].

Clinical trial applications, monitoring, and audit

Among the most significant aspects of a clinical trial is the audit. An audit is a systematic process of evaluating the clinical trial operations at the site. The audit ensures that the clinical trial process is conducted according to the protocol, and predefined quality system procedures, following GCP guidelines, and according to the requirements of regulatory authorities [ 21 ].

The auditors are supposed to be independent and work without the involvement of the sponsors, CROs, or personnel at the trial site. The auditors ensure that the trial is conducted by designated professionally qualified, adequately trained personnel, with predefined responsibilities. The auditors also ensure the validity of the investigational drug, and the composition, and functioning of institutional review/ethics committees. The availability and correctness of the documents like the investigational broacher, informed consent forms, CRFs, approval letters of the regulatory authorities, and accreditation of the trial labs/sites [ 21 ].

The data management systems, the data collection software, data backup, recovery, and contingency plans, alternative data recording methods, security of the data, personnel training in data entry, and the statistical methods used to analyze the results of the trial are other important responsibilities of the auditor [ 21 , 22 ].

According to the ICH-GCP Sec 1.29 guidelines the inspection may be described as an act by the regulatory authorities to conduct an official review of the clinical trial-related documents, personnel (sponsor, investigator), and the trial site [ 21 , 22 ]. The summary report of the observations of the inspectors is performed using various forms as listed in Table ​ Table5 5 .

FDA: Food and Drug Administration; IND: investigational new drug; NDA: new drug application; IRB: institutional review board; CFR: code of federal regulations

Because protecting data integrity, the rights, safety, and well-being of the study participants are more significant while conducting a clinical trial, regular monitoring and audit of the process appear crucial. Also, the quality of the clinical trial greatly depends on the approach of the trial personnel which includes the sponsors and investigators [ 21 ].

The responsibility of monitoring lies in different hands, and it depends on the clinical trial site. When the trial is initiated by a pharmaceutical industry, the responsibility of trial monitoring depends on the company or the sponsor, and when the trial is conducted by an academic organization, the responsibility lies with the principal investigator [ 21 ].

An audit is a process conducted by an independent body to ensure the quality of the study. Basically, an audit is a quality assurance process that determines if a study is carried out by following the SPOs, in compliance with the GCPs recommended by regulatory bodies like the ICH, FDA, and other local bodies [ 21 ].

An audit is performed to review all the available documents related to the IRB approval, investigational drug, and the documents related to the patient care/case record forms. Other documents that are audited include the protocol (date, sign, treatment, compliance), informed consent form, treatment response/outcome, toxic response/adverse event recording, and the accuracy of data entry [ 22 ].

Clinical trial data analysis, regulatory audits, and project management

The essential elements of clinical trial management systems (CDMS) include the management of the study, the site, staff, subject, contracts, data, and document management, patient diary integration, medical coding, monitoring, adverse event reporting, supplier management, lab data, external interfaces, and randomization. The CDMS involves setting a defined start and finishing time, defining study objectives, setting enrolment and termination criteria, commenting, and managing the study design [ 23 ].

Among the various key application areas of clinical trial systems, the data analysis assumes increased significance. The clinical trial data collected at the site in the form of case record form is stored in the CDMS ensuring the errors with respect to the double data entry are minimized.

Clinical trial data management uses medical coding, which uses terminologies with respect to the medications and adverse events/serious adverse events that need to be entered into the CDMS. The project undertaken to conduct the clinical trial must be predetermined with timelines and milestones. Timelines are usually set for the preparation of protocol, designing the CRF, planning the project, identifying the first subject, and timelines for recording the patient’s data for the first visit.

The timelines also are set for the last subject to be recruited in the study, the CRF of the last subject, and the locked period after the last subject entry. The planning of the project also includes the modes of collection of the data, the methods of the transport of the CRFs, patient diaries, and records of severe adverse events, to the central data management sites (fax, scan, courier, etc.) [ 24 ].

The preparation of SOPs and the type and timing of the quality control (QC) procedures are also included in the project planning before the start of a clinical study. Review (budget, resources, quality of process, assessment), measure (turnaround times, training issues), and control (CRF collection and delivery, incentives, revising the process) are the three important aspects of the implementation of a clinical research project.

In view of the increasing complexity related to the conduct of clinical trials, it is important to perform a clinical quality assurance (CQA) audit. The CQA audit process consists of a detailed plan for conducting audits, points of improvement, generating meaningful audit results, verifying SOP, and regulatory compliance, and promoting improvement in clinical trial research [ 25 ]. All the components of a CQA audit are delineated in Table ​ Table6 6 .

CRF: case report form; CSR: clinical study report; IC: informed consent; PV: pharmacovigilance; SAE: serious adverse event

Clinical trial operations at the investigator's site

The selection of an investigation site is important before starting a clinical trial. It is essential that the individuals recruited for the study meet the inclusion criteria of the trial, and the investigator's and patient's willingness to accept the protocol design and the timelines set by the regulatory authorities including the IRBs.

Before conducting clinical research, it is important for an investigator to agree to the terms and conditions of the agreement and maintain the confidentiality of the protocol. Evaluation of the protocol for the feasibility of its practices with respect to the resources, infrastructure, qualified and trained personnel available, availability of the study subjects, and benefit to the institution and the investigator is done by the sponsor during the site selection visit.

The standards of a clinical research trial are ensured by the Council for International Organizations of Medical Sciences (CIOMS), National Bioethics Advisory Commission (NBAC), United Nations Programme on Human Immunodeficiency Virus/Acquired Immunodeficiency Syndrome (HIV/AIDS) (UNAIDS), and World Medical Association (WMA) [ 26 ].

Recommendations for conducting clinical research based on the WMA support the slogan that says, “The health of my patient will be my first consideration.” According to the International Code of Medical Ethics (ICME), no human should be physically or mentally harmed during the clinical trial, and the study should be conducted in the best interest of the person [ 26 ].

Basic principles recommended by the Helsinki declaration include the conduction of clinical research only after the prior proof of the safety of the drug in animal and lab experiments. The clinical trials must be performed by scientifically, and medically qualified and well-trained personnel. Also, it is important to analyze the benefit of research over harm to the participants before initiating the drug trials.

The doctors may prescribe a drug to alleviate the suffering of the patient, save the patient from death, and gain additional knowledge of the drug only after obtaining informed consent. Under the equipoise principle, the investigators must be able to justify the treatment provided as a part of the clinical trial, wherein the patient in the placebo arm may be harmed due to the unavailability of the therapeutic/trial drug.

Clinical trial operations greatly depend on the environmental conditions and geographical attributes of the trial site. It may influence the costs and targets defined by the project before the initiation. It was noted that one-fourth of the clinical trial project proposals/applications submit critical data on the investigational drug from outside the country. Also, it was noted that almost 35% of delays in clinical trials owing to patient recruitment with one-third of studies enrolling only 5% of the participants [ 27 ].

It was suggested that clinical trial feasibility assessment in a defined geographical region may be undertaken for improved chances of success. Points to be considered under the feasibility assessment program include if the disease under the study is related to the population of the geographical region, appropriateness of the study design, patient, and comparator group, visit intervals, potential regulatory and ethical challenges, and commitments of the study partners, CROs in respective countries (multi-centric studies) [ 27 ].

Feasibility assessments may be undertaken at the program level (ethics, regulatory, and medical preparedness), study level (clinical, regulatory, technical, and operational aspects), and at the investigation site (investigational drug, competency of personnel, participant recruitment, and retention, quality systems, and infrastructural aspects) [ 27 ].

Clinical trials: true experiments

In accordance with the revised schedule "Y" of the Drugs and Cosmetics Act (DCA) (2005), a drug trial may be defined as a systematic study of a novel drug component. The clinical trials aim to evaluate the pharmacodynamic, and pharmacokinetic properties including ADME, efficacy, and safety of new drugs.

According to the drug and cosmetic rules (DCR), 1945, a new chemical entity (NCE) may be defined as a novel drug approved for a disease/condition, in a specified route, and at a particular dosage. It also may be a new drug combination, of previously approved drugs.

A clinical trial may be performed in three types; one that is done to find the efficacy of an NCE, a comparison study of two drugs against a medical condition, and the clinical research of approved drugs on a disease/condition. Also, studies of the bioavailability and BE studies of the generic drugs, and the drugs already approved in other countries are done to establish the efficacy of new drugs [ 28 ].

Apart from the discovery of a novel drug, clinical trials are also conducted to approve novel medical devices for public use. A medical device is defined as any instrument, apparatus, appliance, software, and any other material used for diagnostic/therapeutic purposes. The medical devices may be divided into three classes wherein class I uses general controls; class II uses general and special controls, and class III uses general, special controls, and premarket approvals [ 28 ].

The premarket approval applications ensure the safety and effectiveness, and confirmation of the activities from bench to animal to human clinical studies. The FDA approval for investigational device exemption (IDE) for a device not approved for a new indication/disease/condition. There are two types of IDE studies that include the feasibility study (basic safety and potential effectiveness) and the pivotal study (trial endpoints, randomization, monitoring, and statistical analysis plan) [ 28 ].

As evidenced by the available literature, there are two types of research that include observational and experimental research. Experimental research is alternatively known as the true type of research wherein the research is conducted by the intervention of a new drug/device/method (educational research). Most true experiments use randomized control trials that remove bias and neutralize the confounding variables that may interfere with the results of research [ 28 ].

The variables that may interfere with the study results are independent variables also called prediction variables (the intervention), dependent variables (the outcome), and extraneous variables (other confounding factors that could influence the outside). True experiments have three basic elements that include manipulation (that influence independent variables), control (over extraneous influencers), and randomization (unbiased grouping) [ 29 ].

Experiments can also be grouped as true, quasi-experimental, and non-experimental studies depending on the presence of specific characteristic features. True experiments have all three elements of study design (manipulation, control, randomization), and prospective, and have great scientific validity. Quasi-experiments generally have two elements of design (manipulation and control), are prospective, and have moderate scientific validity. The non-experimental studies lack manipulation, control, and randomization, are generally retrospective, and have low scientific validity [ 29 ].

Clinical trials: epidemiological and human genetics study

Epidemiological studies are intended to control health issues by understanding the distribution, determinants, incidence, prevalence, and impact on health among a defined population. Such studies are attempted to perceive the status of infectious diseases as well as non-communicable diseases [ 30 ].

Experimental studies are of two types that include observational (cross-sectional studies (surveys), case-control studies, and cohort studies) and experimental studies (randomized control studies) [ 3 , 31 ]. Such research may pose challenges related to ethics in relation to the social and cultural milieu.

Biomedical research related to human genetics and transplantation research poses an increased threat to ethical concerns, especially after the success of the human genome project (HGP) in the year 2000. The benefits of human genetic studies are innumerable that include the identification of genetic diseases, in vitro fertilization, and regeneration therapy. Research related to human genetics poses ethical, legal, and social issues (ELSI) that need to be appropriately addressed. Most importantly, these genetic research studies use advanced technologies which should be equally available to both economically well-placed and financially deprived people [ 32 ].

Gene therapy and genetic manipulations may potentially precipitate conflict of interest among the family members. The research on genetics may be of various types that include pedigree studies (identifying abnormal gene carriers), genetic screening (for diseases that may be heritable by the children), gene therapeutics (gene replacement therapy, gene construct administration), HGP (sequencing the whole human genome/deoxyribonucleic acid (DNA) fingerprinting), and DNA, cell-line banking/repository [ 33 ]. The biobanks are established to collect and store human tissue samples like umbilical tissue, cord blood, and others [ 34 ].

Epidemiological studies on genetics are attempts to understand the prevalence of diseases that may be transmitted among families. The classical epidemiological studies may include single case observations (one individual), case series (< 10 individuals), ecological studies (population/large group of people), cross-sectional studies (defined number of individuals), case-control studies (defined number of individuals), cohort (defined number of individuals), and interventional studies (defined number of individuals) [ 35 ].

Genetic studies are of different types that include familial aggregation (case-parent, case-parent-grandparent), heritability (study of twins), segregation (pedigree study), linkage study (case-control), association, linkage, disequilibrium, cohort case-only studies (related case-control, unrelated case-control, exposure, non-exposure group, case group), cross-sectional studies, association cohort (related case-control, familial cohort), and experimental retrospective cohort (clinical trial, exposure, and non-exposure group) [ 35 ].

Ethics and concerns in clinical trial/research

Because clinical research involves animals and human participants, adhering to ethics and ethical practices assumes increased significance [ 36 ]. In view of the unethical research conducted on war soldiers after the Second World War, the Nuremberg code was introduced in 1947, which promulgated rules for permissible medical experiments on humans. The Nuremberg code suggests that informed consent is mandatory for all the participants in a clinical trial, and the study subjects must be made aware of the nature, duration, and purpose of the study, and potential health hazards (foreseen and unforeseen). The study subjects should have the liberty to withdraw at any time during the trial and to choose a physician upon medical emergency. The other essential principles of clinical research involving human subjects as suggested by the Nuremberg code included benefit to the society, justification of study as noted by the results of the drug experiments on animals, avoiding even minimal suffering to the study participants, and making sure that the participants don’t have life risk, humanity first, improved medical facilities for participants, and suitably qualified investigators [ 37 ].

During the 18th world medical assembly meeting in the year 1964, in Helsinki, Finland, ethical principles for doctors practicing research were proposed. Declaration of Helsinki, as it is known made sure that the interests and concerns of the human participants will always prevail over the interests of the society. Later in 1974, the National Research Act was proposed which made sure that the research proposals are thoroughly screened by the Institutional ethics/Review Board. In 1979, the April 18th Belmont report was proposed by the national commission for the protection of human rights during biomedical and behavioral research. The Belmont report proposed three core principles during research involving human participants that include respect for persons, beneficence, and justice. The ICH laid down GCP guidelines [ 38 ]. These guidelines are universally followed throughout the world during the conduction of clinical research involving human participants.

ICH was first founded in 1991, in Brussels, under the umbrella of the USA, Japan, and European countries. The ICH conference is conducted once every two years with the participation from the member countries, observers from the regulatory agencies, like the World Health Organization (WHO), European Free Trade Association (EFTA), and the Canadian Health Protection Branch, and other interested stakeholders from the academia and the industry. The expert working groups of the ICH ensure the quality, efficacy, and safety of the medicinal product (drug/device). Despite the availability of the Nuremberg code, the Belmont Report, and the ICH-GCP guidelines, in the year 1982, International Ethical Guidelines for Biomedical Research Involving Human Subjects was proposed by the CIOMS in association with WHO [ 39 ]. The CIOMS protects the rights of the vulnerable population, and ensures ethical practices during clinical research, especially in underdeveloped countries [ 40 ]. In India, the ethical principles for biomedical research involving human subjects were introduced by the Indian Council of Medical Research (ICMR) in the year 2000 and were later amended in the year 2006 [ 41 ]. Clinical trial approvals can only be done by the IRB approved by the Drug Controller General of India (DGCI) as proposed in the year 2013 [ 42 ].

Current perspectives and future implications

A recent study attempted to evaluate the efficacy of adaptive clinical trials in predicting the success of a clinical trial drug that entered phase 3 and minimizing the time and cost of drug development. This study highlighted the drawbacks of such clinical trial designs that include the possibility of type 1 (false positive) and type 2 (false negative) errors [ 43 ].

The usefulness of animal studies during the preclinical phases of a clinical trial was evaluated in a previous study which concluded that animal studies may not completely guarantee the safety of the investigational drug. This is noted by the fact that many drugs which passed toxicity tests in animals produced adverse reactions in humans [ 44 ].

The significance of BE studies to compare branded and generic drugs was reported previously. The pharmacokinetic BE studies of Amoxycillin comparing branded and generic drugs were carried out among a group of healthy participants. The study results have demonstrated that the generic drug had lower Cmax as compared to the branded drug [ 45 ].

To establish the BE of the generic drugs, randomized crossover trials are carried out to assess the Cmax and the AUC. The ratio of each pharmacokinetic characteristic must match the ratio of AUC and/or Cmax, 1:1=1 for a generic drug to be considered as a bioequivalent to a branded drug [ 46 ].

Although the generic drug development is comparatively more beneficial than the branded drugs, synthesis of extended-release formulations of the generic drug appears to be complex. Since the extended-release formulations remain for longer periods in the stomach, they may be influenced by gastric acidity and interact with the food. A recent study suggested the use of bio-relevant dissolution tests to increase the successful production of generic extended-release drug formulations [ 47 ].

Although RCTs are considered the best designs, which rule out bias and the data/results obtained from such clinical research are the most reliable, RCTs may be plagued by miscalculation of the treatment outcomes/bias, problems of cointerventions, and contaminations [ 48 ].

The perception of healthcare providers regarding branded drugs and their view about the generic equivalents was recently analyzed and reported. It was noted that such a perception may be attributed to the flexible regulatory requirements for the approval of a generic drug as compared to a branded drug. Also, could be because a switch from a branded drug to a generic drug in patients may precipitate adverse events as evidenced by previous reports [ 49 ].

Because the vulnerable population like drug/alcohol addicts, mentally challenged people, children, geriatric age people, military persons, ethnic minorities, people suffering from incurable diseases, students, employees, and pregnant women cannot make decisions with respect to participating in a clinical trial, ethical concerns, and legal issues may prop up, that may be appropriately addressed before drug trials which include such groups [ 50 ].

Conclusions

Clinical research and clinical trials are important from the public health perspective. Clinical research facilitates scientists, public health administrations, and people to increase their understanding and improve preparedness with reference to the diseases prevalent in different geographical regions of the world. Moreover, clinical research helps in mitigating health-related problems as evidenced by the current Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2) pandemic and other emerging and re-emerging microbial infections. Clinical trials are crucial to the development of drugs, devices, and vaccines. Therefore, scientists are required to be up to date with the process and procedures of clinical research and trials as discussed comprehensively in this review.

The content published in Cureus is the result of clinical experience and/or research by independent individuals or organizations. Cureus is not responsible for the scientific accuracy or reliability of data or conclusions published herein. All content published within Cureus is intended only for educational, research and reference purposes. Additionally, articles published within Cureus should not be deemed a suitable substitute for the advice of a qualified health care professional. Do not disregard or avoid professional medical advice due to content published within Cureus.

The authors have declared that no competing interests exist.

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