Orphan Drug Designation: Benefits and Challenges
Note: This publication will be available in PDF format by Nov. 30, 2022.
Success with the FDA’s Orphan Drug Program is possible … if you know its ins and outs.
Orphan Drug Designation: Benefits and Challenges will explain everything you need.
This management report provides an insider’s view of the program designed to encourage you to research and develop treatments for rare diseases, making the investment of time and resources worth your while.
Aaron Friedman — senior regulatory counsel in the FDA’s Office of Orphan Products Development (OOPD) — provides clarity on the program’s benefits (e.g., seven-year exclusivity, user fee exemptions, tax credits), the process for applying for designation, the information you need to make a case for OOPD support and the categories of support available.
You’ll get Mr. Friedman’s explanation for the rationale and regulatory development of the program and understand specific concepts like “clinically superior” and “same drug.”
Together with Brian Malkin — a partner at McDermott, Will & Emery who spent time in the FDA’s Office of the Commissioner and the Center for Drug Evaluation (CDER) — you’ll get takeaways from the legal challenges the program has faced.
Contributor Karin Hoelzer, National Organization for Rare Disorders (NORD) policy and regulatory affairs director, explains the value of the FDA’s accelerated approval pathway and the uses of real-world evidence (RWE) in rare-disease drug development.
Management Report Takeaways:
- Recent regulatory decisions and legislative changes regarding orphan drug exclusivity, e.g., when a plausible theory of clinical superiority must be demonstrated to obtain orphan drug exclusivity for a “same” drug (same active moiety/ingredient and orphan indication)
- Rulings on being considered an orphan drug based on lack of ability to make a profit on the drug
- Labeling considerations for same disease/condition or use/indication
- Successes of the Orphan Drug Program
- Specific terms of exclusivity
- Why designation does not equal FDA approval of an orphan drug
- The Government Accountability Office’s (and others’) recommendations to OOPD to improve decisionmaking and whether such recommendations have been implemented and, if so, to what effect?
The FDA’s Orphan Drug Program holds great promise for your rare disease treatments — if you know how to navigate it. Orphan Drug Designation: Benefits and Challenges will ensure you do.
About the Contributors
Brian Malkin is a partner at McDermott, Will & Emery. He counsels biopharma companies on FDA regulatory matters and intellectual property law, with an emphasis on patent litigation. Mr. Malkin has more than 20 years of FDA and intellectual property law experience, including time spent in the Office of the Commissioner and the Center for Drug Evaluation at the FDA.
Aaron Friedman is the senior regulatory counsel in the FDA’s Office of Orphan Products Development (OOPD). Friedman has worked in OOPD since 2016 on legal and policy matters related to the Orphan Drug Designation Program, the Rare Pediatric Disease Priority Review Voucher Program and OOPD’s grants programs. Much of Friedman’s work has focused on orphan drug exclusivity issues, including complex exclusivity matters involved in litigation and citizen petitions. Prior to joining OOPD, he worked in the FDA’s Office of Regulatory Affairs and the Center for Tobacco Products.
Karin Hoelzer, DVM serves as director of policy and regulatory affairs for the National Organization for Rare Disorders (NORD). In this role, she is responsible for NORD’s rare disease policy on issues pertaining to the FDA and the National Institutes of Health. In prior roles, she served as research scientist at Cornell University, risk analyst and modeler at the FDA, senior officer for health programs at The Pew Charitable Trusts and most recently as senior director for public health data analytics at Maximus Inc.
Who Will Benefit
- Regulatory affairs/compliance personnel
- R&D personnel
- Marketing/promotion personnel
- Legal affairs professionals
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Karin Hoelzer, DVM
Nov. 30, 2023
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