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Evolution of FDA’s View of a Well-Designed Clinical Trial

1.85 credits


Full course description

The Georgia Clinical & Translational Science Alliance- Georgia CTSA and Southern California Clinical and Translational Science Institute -SC-CTSI have collaborated to provide free, high quality educational programs for clinical research professionals at novice to expert levels of experience. At the completion of each course or program, participants earn contact hours recognized by a certificate and/or badge.


An in-depth overview and history of the FDA will include the legal framework that governs the FDA’s regulatory oversight to ensure safe and effective medical products are approved and marketed.A comprehensive review of drug/product development from discovery phase to clinical trials and various pathways utilized by the FDA will be examined.


  1. Food and Drug Administration (FDA) Departments
  2. Center for Drug Evaluation and Research (CDER)
  3. Regulatory “Communication Tools”: Primary vs. Additional:
    • Primary Tools: Acts, Regulations, Code of Federal Regulations (CFR), Legal Precedents, Guidance
    • Additional Tools: Advisory Committees, Regulatory Decisions, Agency Initiatives, Regulatory Science/Research
  4. Good Guidance Practices Regulation
  5. The Process: Draft Regulations & Guidance
    • Public comment period
    • Regulation: FDA must address all comments
    • Guidance: FDA utilizes comments as advice
  6. Global Guidance: The International Conference on Harmonisation (ICH)
  7. The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human use ICH
    • Important ICH Guidelines pertaining to Clinical Trials: E3, E5, E6, E6, E9, E10, E17
    • Impact of how clinical trials can produce different data in different countries.
  8. FDA Administration Amendments Act (FDAAA) & Prescription Drug User Fee Act (PDUFA) IV
  9. FDA History:
    • Patent Medicine
    • The “Poison Squad” and Harvey W. Wiley, M.D.
  10. FDA History: The Law – Getting to Clinical Trials
    • 1906: Food and Drugs Act - Banned misbranding and adulterated products
    • 1938: The Federal Food, Drug, and Cosmetic (FDC)
    • 1962: Thalidomide & Kefauver-Harris Drug Amendments
  11. Francis Kelsey: FDA Reviewer
  12. Post 1962 Drug Amendments (1)
    • Conflict of interest concerns among FDA & agency/sponsor- FDA not allowed to endorse products
  13. Post 1962 Drug Amendments (2)
    • New U.S. drugs must be investigated under Investigational New Drug Application (IND)
  14. Post 1962 Drug Amendments (3)
    • Many drugs approved in 1938- 1962 had no proof efficacy resulting in reviewing of data.
    • Clinical Trial:
    • Definition
    • Clinical Trials in the 20th Century
      • Systematic schemes for treatment assignment
      • Treatment assignment by randomization
      • Multicenter trials- multiple investigators at different sites
  15. Why Evidence from Clinical Trials?
    • Best way to determine if intervention has proposed effect and see the risks
    • An individual’s reaction can still be different from the study
    • Example: The Cardiac Arrhythmia Suppression Trial (CAST)
  16. Drug Approval: Effectiveness
  17. Adequate and well-controlled multiples studies to approve effectiveness
    • Substantial Evidence- Adequate and Well-Controlled Studies
  18. Drug Approval: Effectiveness
  19. CFR: 314.126 Adequate and well-controlled studies
  20. “Gold Standard” for Approval of an NDA/BLA
    • Establishing a standard for uniformity
  21. Regulatory Science: The Confirmatory Trial
    • A confirmatory trial is an adequately controlled trial in which the hypotheses are stated in advance and evaluated
    • Trials can be expensive and resource-costly
    • Good documentation is a “simple” way to show this type of trial
  22. Evolving: The Evidence Document
  23. FDAMA (1997) and Clinical Investigations
    • Sec. 115. Clinical Investigations provision was clarified, and law was changed
  24. Clinical Trials Have Evolved Into—Big, Complex, Expensive Experiments
  25. NDA Submission Regulations
  26. Guideline for the Format and Content of the Clinical and Statistical Sections of New Drug Applications
  27. Prior to Electronic Submission – paper review of an NDA
  28. 21st Century Review of Drugs and Biologics
  29. Communication with Sponsors to Facilitate Drug Development & Review (Pre-PDUFA)
    • Rare Disease Program - conducting clinical trials with small, specific populations
  30. Dr. Woodcock’s Critical Path Initiative: 76 discreet projects
  31. These Are Exciting Times!
    • Oncology Center for Excellence
    • CDISC Data Standards
    • NIH FDA Protocol Template
  32. PDUFA VI: Complex Design Workshop
    • Possibility for sharing intellectual property and enriching the clinical trials of certain field/specific ailment
  33. Direct to Consumer Advertising


Steve Wilson, DrPH, MPH, received his doctorate in Biostatistics from the University of North Carolina, Chapel Hill in 1984 and worked for FDA’s Center for Drug Evaluation and Research for 30 years.His positions at FDA included mathematical statistician/reviewer, group leader, team leader, deputy division director and Acting Office Director for the Office of Business Process Support.Prior to his retirement from the PHS Commissioned Corps in 2017, he had served as the Director of the Division of Biometrics III in the Office of Biostatistics for more than 12 years.Before starting his FDA career, Dr. Wilson’s professional experience included positions with the East West Center (Honolulu), the Indonesian Central Bureau of Statistics (Jakarta), the University of North Carolina (Chapel Hill), the Federated States of Micronesia (Pohnpei) and the World Bank (Washington, DC).His professional interests and activities have been and continue to be focused on issues related to improvements in statistical thinking and decision-making, institutional development, clinical trials science and practice, data standards and the regulatory review of drug and biological therapies.Currently and throughout his career with the FDA, Dr. Wilson has actively supported a number of professional organizations dedicated to thinking about and improving statistical science, clinical trials and drug/biologics development, including: the Drug Information Association (DIA); the American Statistical Association (ASA); the Council for Biopharmaceutical Statisticians (CBS); the Society for Clinical Trials (SCT); the Society for Clinical Data Management (SCDM); the Clinical Data Interchange Standards Consortium (CDISC); and the Pharmaceutical Users Software Exchange (PhUSE).

Learner Level: Beginner/Intermediate/Advanced


This symposium is designed for Clinical Research Assistant/Associates or Clinical Research Coordinators in academia, clinics, hospitals, industry, or CRO with 1- 2 years of clinical research experience.Individuals may be new to clinical research with limited knowledge of clinical trial conduct, good clinical practices, regulations and common terminology associated with clinical research.

Contact Hours:

Emory Nursing Professional Development Center (ENPDC) is accredited as a provider of nursing continuing professional development by the American Credentialing Center’s Commission on Accreditation. Attendees to this CNE activity will be awarded 1.85 contact hours by ENPDC. No conflict of interest has been found with the speaker for this CNE activity nor with the members of the planning committee.

Need Help with Registration? Please contact us at or 404-727-9208.

Program Information: This is the second course in a five (5)-course program. To complete the entire program and earn a badge CLICK HERE.



Learner Disclosures:

Accreditation Status: Emory Nursing Professional Development Center (ENPDC) is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s (ANCC) Commission on Accreditation.

Financial Relationships: ENPDC has evaluated everyone who has the ability to control content of this activity (planning committee members, subject matter experts, presenters) and found that no relevant financial relationships exist.

Awarding Contact Hours: To obtain contact hours participants must:

·       Review the entire activity

·       Pass the quiz

·       Complete the evaluation at the end

Certificates will be distributed at the end of the evaluation.


Enduring Materials Dates:


·       Start Date: 4/22/2024

·       Expiration Date: 4/22/2027

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