Regulatory Science Virtual Symposium: “Study Design for Clinical Trials: Types and Trends” Session 4: Types and Trends (2023)

Course Syllabus/Topics

  1. Thoughts on trends - Regulatory Perspective
    1. Clinical trial diversity
    2. Medical device trials
    3. Clinical/statistical issues-Surrogate endpoints for accelerated approval
  2. CT Diversity Plans
    1. Food and Drug Omnibus Reform Act (FDORA) additions:
      1. Subsection 505(z)
        1. Requires sponsors of any phase 3 or other pivotal drug study to submit diversity action plans
      2. Subsection 520(g)(9)
        1. Must submit a diversity action plan for sponsors of device trials that require an Investigational Drug Exception (IDE)
    2. Recommended Content
      1. Overview of disease/condition
      2. Scope of medical product development
      3. Goals for enrollment of under-represented racial and ethnic populations
      4. Specific plan to enroll and retain underrepresented diverse participants
      5. Status of meeting enrollment goals (if applicable)
    3. CTs for Medical Devices
      1. Upon a search of interventional trials in the United States in, there were over 20,000 medical device trials
    4. Digital health
      1. Mobile Health
      2. Health information technology (IT)
      3. Wearable devices
      4. Telehealth/telemedicine & personalized medicine
      5. Clinical decisions support software
      6. AI/ML
      7. Computing platforms, software, etc.
      8. Companion diagnostics
    5. Center for Devices and Radiological Health (CDRH) Guidance re: Software
      1. Addresses the types of software
      2. Four criteria
    6. Surrogate Endpoints
      1. An endpoint that is a substitute, or “surrogate”, for the outcome you want to study
      2. Use of surrogate endpoints instead of clinical outcomes in some clinical trials
      3. Clinical outcomes directly measure whether people in a trial feel or function better or live longer
    7. Questions/comments?


Accompanying text created by Roxy Terteryan, RKS Project Administrator, SC CTSI ( and Cyan Tan, Student Worker


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