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

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

Session #	Title	Presentations
1	Introduction and Motivations Co-chairs: Carl Peck, University of California, San Francisco; and Donald Rubin, Harvard University	Introduction.pdf 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.pdf Stephen Ruberg, Eli Lilly and Company   Fisher, Naymen and Bayes at FDA.pdf Donald Rubin, Harvard University   CDER and CBER Experience.pdf Lisa LaVange, US Food and Drug Administration   Bayesian Submissions to FDA and the Evidentiary Standard for Effectiveness: the CDER Experience.pdf 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; and John Scott, US Food and Drug Administration	Overview of DIA Bayesian Scientific Working Group.pdf Karen Price, Eli Lilly and Company   The Value of Bayesian Methods for Evidence-Based Medicine.pdf Steven Goodman, Stanford University   An Industry Perspective of the Value of Bayesian Methods.pdf David Ohlssen, Novartis   Regulatory Perspective of the Value of Bayesian Methods.pdf Telba Irony, US Food and Drug Administration
	Luncheon talk: Reverend Bayes Goes to Washington.pdf 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; and Lisa LaVange, US Food and Drug Administration	What a Bayesian Owes a Frequentist.pdf Frank Harrell, Vanderbilt University   The Role of Simulations for Bayesian Analyses and Regulatory Approval.pdf Scott Berry, Berry Consultants   Bayesian Methods in Regulatory Science: Identifying Patient Subgroups with Positive Treatment Effects.pdf Bradley Carlin, University of Minnesota   Panel_Discussion.pdf
4	Substantial Evidence through a Bayesian Lens Co-chairs: Stephen Ruberg, Eli Lilly and Company; and Gregory Campbell, Independent Consultant	Clinical Drug Development with a Bayesian Lens.pdf Stephen Ruberg, Eli Lilly and Company   Points to Consider Panel Discussion (no slides)

ACDRS Workshop Final Program 2016

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 and 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].