Orphan Drugs: Latest Regulatory, Compliance and Legal Developments – Webinar Recording/Transcript
What if you could get orphan drug-related regulatory and compliance clarity directly from the FDA?
You can, with this presentation.
Brian Malkin is a partner at McDermott, Will & Emery who spent time in the Office of the Commissioner and the Center for Drug Evaluation and Research (CDER) at the FDA. Along with Aaron Friedman, senior regulatory counsel in the FDA’s Office of Orphan Products Development (OOPD), and Karin Hoelzer, director of policy and regulatory affairs at the National Organization for Rare Disorders® (NORD®), he will discuss the recent decisions and litigation related to orphan drugs, ensuring you understand the implications and the FDA’s challenges.
The presentation team will reveal perspectives from a cross-section of current and former FDA regulators, FDA litigators and major orphan drug trade organizations.
You’ll discover how the FDA has resolved some issues, is seeking legislative fixes for others (such as those in the Catalyst decision and the Avadel litigation) and what additional changes the agency might make (including the recommendations in the Government Accountability Office’s 2018 report).
They’ll also show you the potential pathways forward for greater regulatory transparency, accountability and predictability.
All so you’re more secure in how to proceed with your orphan products.
- Why orphan drug development — and transparent, consistent, predictable decisionmaking by the regulators — helps the industry, affected patients and their healthcare providers
- 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), newer rulings on being considered an orphan drug based on lack of ability to make a profit on the drug (is this still viable?), and orphan drug labeling considerations for same disease/condition or use/indication
- How the FDA interprets statutes and promulgates regulations, and what happens when federal courts/appeals courts determine the agency did not interpret correctly
- 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?
- Legislative or other changes that may be contemplated or recommended for orphan products
If you’re selling, developing or considering developing orphan products or products with potential orphan indications, you need the information these FDAers and rare disease advocates have to share!
Meet Your Presenters
Brian Malkin, a partner at McDermott, Will & Emery, counsels pharmaceutical and biologic clients on FDA regulatory matters and intellectual property law, with an emphasis on patent litigation. Mr. Malkin’s regulatory experience includes all types of FDA-regulated products: drugs and biologics (including animal drugs and biologics), medical devices, cannabis, foods and dietary supplements, cosmetics and tobacco products. He is a key advisor to pharmaceutical and biologic clients in the premarket; regulatory review; and marketing, enforcement and lifecycle management phases of product development. Mr. Malkin has more than 20 years of FDA and intellectual property law experience, including time spent in the Office of the Commissioner and CDER.
Dr. Karin Hoelzer 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 pertain to the Food and Drug Administration (FDA) and National Institutes of Health (NIH). In prior roles, she served as research scientist at Cornell University, risk analyst and modeler at the FDA, as senior officer for health programs at The Pew Charitable Trusts and most recently as senior director for public health data analytics at Maximus Inc. She holds a D.V.M. from the University of Veterinary Medicine, Hanover Germany and a Ph.D. in Comparative Biomedical Sciences from Cornell University. In addition, she is a Registered Patent agent and serves as co-editor in chief for Elsevier and as executive director for the District of Columbia Veterinary Medical Association.
Aaron Friedman is the senior regulatory counsel in the FDA’s Office of Orphan Products Development (OOPD). He 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 Mr. 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.
Who Will Benefit
- Regulatory affairs
- Government affairs
- Regulatory counsel or in-house counsel and outside counsel
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Oct. 4, 2022