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How to Succeed in Orphan Drug Regulatory Affairs
Session Chair(s)
Tim Cote, MD, MPH
Founder and Chief Executive Officer
Only Orphans Cote, United States
In this session, orphan drug thought leaders will share their insights about how to succeed in orphan regulatory affairs. Orphan drug development begins with designation, and we will explain what does not work when submitting to the Office of Orphan Products Development. Moving from designation to the review division process, we will provide a quantitative analysis of the special treatment afforded orphan drugs by the FDA’s review divisions. We will also include a European perspective on the orphan drug endeavor, reviewing the unique European approach to the way orphan drugs become licensed in the European community. We will present a picture of orphan drug regulatory success drawn from thousands of regulatory actions from their collective experience.
Learning Objective : Identify the leading reasons why orphan designation applications fail; Discuss key strategies to prevent orphan designation application failure; Discuss the premise of the question "What is the disease?" and how that applies to orphan drug designation; Describe the unique way in which regulators use the word subset when deciding whether an orphan designation application will or will not yield a designation award; Discuss nuances of the European orphan drug landscape.
Speaker(s)
Why Orphan Drug Designation Applications Fail
Tim Cote, MD, MPH
Only Orphans Cote, United States
Founder and Chief Executive Officer
Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: An Update to the 2012 Seminal Analysis
James Valentine, JD, MHS
Hyman, Phelps & McNamara, PC, United States
Director
European Perspective
Christopher J Holloway, PhD
ERA Consulting Group, Germany
Group Director, Regulatory Affairs and CSO
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