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Wednesday, June 22 • 2:00pm - 3:00pm
#352: FDA Rare Disease Town Hall

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-629-L04-P ; CME 1.00; RN 1.00

Rare disease drug development continues to explode. As a result, development stratgies coupled with regulatory science and policy continues to evolve to keep up. Regulators from FDA will describe key trends and opportunities in this arena.

Learning Objectives

Discuss recent trends in the development and review of medical products for rare diseases; Identify new regulatory tools and guidance to support regulatory interactions around orphan drugs; Discuss how to apply unique strategies to overcome the unique challenges faced in clinical trials for rare conditions.


James Valentine, JD, MHS


Patrizia Cavazzoni, MD

Peter W. Marks, MD, PhD

Rare Disease Patient Community Perspective
Jennifer Farmer, MS

avatar for Patrizia Cavazzoni

Patrizia Cavazzoni

Director, Center for Drug Evaluation, FDA, United States
Dr. Cavazzoni received her medical degree at McGill University and completed a residency in Psychiatry and a fellowship in mood disorders at the University of Ottawa. She subsequently joined the faculty of medicine at the University of Ottawa as an assistant professor, where she was... Read More →
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →

Wednesday June 22, 2022 2:00pm - 3:00pm CDT
Room 187 McCormick Place 2301 South Indiana Avenue Gate 40 Chicago, IL 60616
  09: Regulatory, Forum |   05: Patient Engagement, Forum |   06: PreClinical Dev-EarlyPhaseCR, Forum