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DIA/FDA Biostatistics Industry and Regulator Forum

This event is now offered in a new entirely virtual format.

Session 5: The Role of Pragmatic Trials for Regulatory Problems

Session Chair(s)

Brenda  Crowe, PhD

Brenda Crowe, PhD

  • Senior Research Advisor, Global Statistical Sciences
  • Eli Lilly and Company, United States
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, OT, CDER
  • FDA, United States
Regulators and industry are actively exploring the use of real world evidence (RWE) to address important regulatory problems. Randomized pragmatic trials provide a promising source of RWE. This session considers the role of pragmatic trials for regulatory problems and focuses on the clinical and statistical objectives of pragmatic trials and their appropriateness for regulatory problems.
Learning Objective :

At the conclusion of this session, participants should be able to:

  • Describe what is a pragmatic trial
  • Explain various possible objectives of pragmatic trials
  • Understand the various estimands to achieve the objectives


Miguel  Hernan, DrPH, MD

Per-Protocol Analyses of Pragmatic Trials

Miguel Hernan, DrPH, MD

  • Professor
  • Department of Epidemiology, Harvard T.H. Chan School of Public Health, United States
Janet Turk Wittes, PhD

ITT Analyses of Pragmatic Trials: Is the 21st Century Different from the Past?

Janet Turk Wittes, PhD

  • President
  • Statistics Collaborative Inc., United States
Peter P. Stein, MD


Peter P. Stein, MD

  • Director, Office of New Drugs, CDER
  • FDA, United States

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Additional Information

Biostatistics Industry Resource Kit