Workshop: Substantial Evidence in 21st Century Regulatory Science: Borrowing Strength from Accumulating Data

This Special ACDRS Workshop opens discussion on deriving substantial evidence of effectiveness based on a high probability of effectiveness

Details

About

The statutory requirement for substantial evidence of effectiveness of a new drug has generally been interpreted to require demonstration of a low probability (p<0.05), assuming non-effectiveness, based on data from two phase III clinical trials. This Special ACDRS Workshop opens discussion on deriving substantial evidence of effectiveness based on a high probability of effectiveness, utilizing evidence from all reliable sources of effectiveness data.

Program

Ses­sionTitlePresentations
1

Introduction and Motivations

Co-chairs:

  • Carl Peck, University of California, San Francisco
  • Donald Rubin, Harvard University
  • Introduction by Carl Peck, University of California, San Francisco
  • Keynote Address (no slides)
    Janet Woodcock, US Food and Drug Administration
  • Bayesian Methods, Regulatory Decisions, and the Evidence Development Landscape
    Mark McClellan, Duke University
  • Tutorial Frequentist Bayesian - Statistical Frameworks for Substantial Evidence 
    Stephen Ruberg, Eli Lilly and Company
  • Fisher, Naymen and Bayes at FDA 
    Donald Rubin, Harvard University
  • CDER and CBER Experience 
    Lisa LaVange, US Food and Drug Administration
  • Bayesian Submissions to FDA and the Evidentiary Standard for Effectiveness: the CDER Experience  
    Gregory Campbell, Independent Consultant
2

Stakeholder Perspectives on the Use of Bayesian Methods in Drug Development and Regulatory Science (DD&R)

Co-chairs:

  • Karen Price, Eli Lilly and Company
  • John Scott, US Food and Drug Administration
  • Overview of DIA Bayesian Scientific Working Group 
    Karen Price, Eli Lilly and Company
  • The Value of Bayesian Methods for Evidence-Based Medicine
    Steven Goodman, Stanford University
  • An Industry Perspective of the Value of Bayesian Methods 
    David Ohlssen, Novartis
  • Regulatory Perspective of the Value of Bayesian Methods 
    Telba Irony, US Food and Drug Administration
 

Luncheon talk

Reverend Bayes Goes to Washington

Sharon Bertsch McGrayne, Author of “The Theory that Would Not Die, How Bayes’ Rule Cracked the Enigma Code, Hunted Down Russian Submarines & Emerged Triumphant from Two Centuries of Controversy”

3

Opportunities to Advance the Use of Bayesian Methods for DD&R

Co-chairs:

  • Karen Price, Eli Lilly and Company
  • Lisa LaVange, US Food and Drug Administration
  • What a Bayesian Owes a Frequentist 
    Frank Harrell, Vanderbilt University
  • The Role of Simulations for Bayesian Analyses and Regulatory Approval 
    Scott Berry, Berry Consultants
  • Bayesian Methods in Regulatory Science: Identifying Patient Subgroups with Positive Treatment Effects
    Bradley Carlin, University of Minnesota
  • Panel Discussion
4

Substantial Evidence through a Bayesian Lens

Co-chairs:

  • Stephen Ruberg, Eli Lilly and Company
  • Gregory Campbell, Independent Consultant
  • Clinical Drug Development with a Bayesian Lens 
    Stephen Ruberg, Eli Lilly and Company
  • Points to Consider Panel Discussion (no slides)

Speakers and panelists

  • Scott Berry, PhD, Berry Consultants
  • Gregory Campbell, PhD, Consultant
  • Bradley Carlin, PhD, University of Minnesota
  • David W. Feigal Jr., MD, MPH, NDA Partners
  • Steven Goodman, MD, MHS, PhD, Stanford University
  • Frank E. Harrell Jr., PhD, Vanderbilt University
  • Telba Irony, PhD, Food and Drug Administration
  • Lisa LaVange, PhD, Food and Drug Administration
  • Mark McClellan, MD PhD, Duke University
  • Sharon Bertsch McGrayne, Author of “The Theory that Would Not Die”
  • David Ohlssen, PhD, Novartis
  • Carl Peck, MD, UCSF and NDA Partners
  • Karen Price, PhD, Eli Lilly and Company
  • Stephen Ruberg, PhD, Eli Lilly and Company
  • Donald Rubin, PhD, Harvard University
  • John Scott, PhD, Food and Drug Administration
  • Robert Temple, MD, Food and Drug Administration
  • Janet Woodcock, MD, Food and Drug Administration

Planning committee

  • Co-Chairs:
    • Carl Peck, MD, UCSF and NDA Partners
    • Stephen Ruberg, PhD, Eli Lilly and Company
  • Gregory Campbell, PhD, Consultant
  • Lisa LaVange, PhD, Food and Drug Administration
  • Karen Price, PhD, Eli Lilly and Company
  • Donald Rubin, PhD, Harvard University
  • John Scott, PhD, Food and Drug Administration

Questions?

Please contact [email protected]

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