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DIA/FDA Biostatistics Industry and Regulator Forum


Session 4: Bridging the Gap: RWE and RCT

    Session Chair(s)
      Frank W. Rockhold, PhD, MSc

      Frank W. Rockhold, PhD, MSc

      • Professor of Biostatistics
      • Duke Clinical Research Institute, Duke University Medical Center, United States
      Mark S. Levenson, PhD

      Mark S. Levenson, PhD

      • Director, Division of Biometrics VII, Office of Biostatistics, CDER
      • FDA, United States
    There is currently a drive and energy behind the use of “big data” and “real world data”. There are three main motivations: Use of RWE data are viewed as 1) a quicker and less expensive approach to clinical development, 2) a way to answer questions of how treatments are or will be used in the real world, and 3) where randomized trials are not ethically possible. We will restrict the discussion to studies done post approval of the first indication as “RWE” is a non-sequitur for an unapproved drug. One of the current issues is the blurring in the use if RWE to answer scientific and medically important questions and if RWE is somehow distinct from the conduct randomized trials and only used to do observational studies. In this sense, the debate is often posed as “should I do a RWE study OR a clinical trial” or “how can I show that an Obs study will yield the same answer as a RCT”. The truth for drug development is the this should be an “and” discussion not an “or” discussion as both approaches can be valuable. In addition, should more care should be given not to avoid the concept of randomization too quickly in the use of RWE as there many techniques allow us to bridge that gap?
    Learning Objective : At the conclusion of this session, participants should be able to:
    • Recognize the circumstances when randomized trials and observational studies are appropriate to address clinical questions
    • Recognize the opportunities and challenges of pragmatic and clustered trials
    • Discuss the important issues when considering an observational study
    Speaker(s)
      Frank W. Rockhold, PhD, MSc

      Randomized Pragmatic Clinical Trials: A Potential Bridge Between ”RCTs” and “RWE” Studies?

      Frank W. Rockhold, PhD, MSc

      • Professor of Biostatistics
      • Duke Clinical Research Institute, Duke University Medical Center, United States
      Hrishikesh  Chakraborty

      Statistical Considerations for Pragmatic Cluster Randomized Trial Design

      Hrishikesh Chakraborty

      • Associate Director
      • DCRI, Duke University, United States
      Douglas E Faries, PhD

      Bridging the Gap: Better Use of RWE for Decision-Making from an Industry Perspective

      Douglas E Faries, PhD

      • Research Fellow
      • Eli Lilly and Company, United States
      Jessica  Franklin, PhD

      Using Healthcare Databases To Evaluate The Safety And Effectiveness Of Newly Marketed Medications

      Jessica Franklin, PhD

      • Assistant Professor
      • Brigham and Women's Hospital and Harvard Medical School, United States