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Wednesday, June 21 • 2:00pm - 3:15pm
#344: Minding the Gaps: Using Totality of Evidence in the Clinical and CMC Contexts to Support Regulatory Approval

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Component Type: Session
Level: Intermediate
CE: CME 1.25; IACET 1.25; RN 1.25

Regulatory approval dossiers often lack full evidence in areas such as clinical or CMC. Sponsors and regulators can sometimes accommodate gaps in data by utilizing a totality of evidence approach. Examples of this include the use of totality of evidence in CMC packages for botanicals and clinical packages for accelerated or conditional approval of new active substances in general. This session will dive into the concept of totality of evidence and explore recent FDA and EMA approvals that relied on totality of evidence. Attendees will learn how to distinguish between valid and questionable uses of totality of evidence and how to apply totality of evidence in their development programs.

Learning Objectives

Identify how to use totality of evidence to support regulatory approval; Describe how to leverage regulatory flexibility in small or limited clinical programs; Develop strategies to overcome limitations in CMC packages, especially for botanical drug development.


Alexander Varond, JD


Short of Perfect - Approval With Data That's Less Than Optimal
Alexander Varond, JD

Clinical Evidence to Support Marketing Authorizations: Focus on Regulatory Approvals Based on a Single Pivotal Trial
Anne Vinther Morant, PhD, MSc

FDA’s Totality-of-Evidence Approach in Botanical New Drug Approval
Charles G. Wu, PhD

avatar for Alexander Varond

Alexander Varond

Associate, Goodwin Procter LLP
Alexander J. Varond works on drug development and medical devices, advertising and promotion, and enforcement issues. He counsels clients on drug development matters such as clinical trial design, REMS, orphan drug designation, formal dispute resolution, and patent and exclusivity... Read More →

avatar for Anne Morant

Anne Morant

Senior Specialist, Regulatory Science & Advocacy, H. Lundbeck A/S
Anne Vinther Morant is a Senior Specialist in Regulatory Science & Advocacy at H. Lundbeck A/S. Since 2008 she has worked in various functions within regulatory strategy and regulatory science with focus on development of medicines to treat psychiatric and neurologic diseases. Anne... Read More →
avatar for Charles Wu

Charles Wu

Expert Pharmacologist, OPQ, CDER, FDA
Dr. Charles Wu is an Expert Pharmacologist at Center for Drug Research and Evaluation of the FDA. He was trained in clinical medicine including the Traditional Chinese Medicine and earned his Ph.D. from University of Amsterdam. He has been working at the FDA since 2001 after a fellowship... Read More →

Wednesday June 21, 2017 2:00pm - 3:15pm CDT
S404a McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  05: Regulatory, Session