Cautious Optimism Surrounding Rare Diseases and Orphan Drugs – FDA Watch
Wayne chats with Geoffrey Levitt, Head of Global Regulatory Law at argenx, and Frank Sasinowski, Director at Hyman, Phelps & McNamara. You’ll hear their thoughts on the recently introduced plausible mechanism framework for individualized therapies in ultra-rare diseases; where FDA is making meaningful progress for rare-disease sponsors and where gaps persist; the agency’s willingness to exercise regulatory flexibility in rare diseases; recent experiences in dealing with FDA on orphan drugs; how the agency has fared in translating concepts such as historical data, real-world evidence, and patient engagement into review decisions; and much more.
Headlines
FDA releases congressional justification for $7.2 billion FY 2027 budget
Pharma: FDA-backed proposals aim to entice pharma companies to test and produce drugs domestically
Devices: FDA launches READI-Home Innovation Challenge
Food: FDA seeking public input on potential market name change for 18 species of rockfish
Cosmetics: Processors must complete first biennial FDA facility registration renewals by July 1, as required under MoCRA
Resource Links
FDA launches new adverse event look
Pharma: FDA schedules public meeting for input on Commissioner’s National Priority Voucher pilot program
Devices: FDA issues final guidance on how to collect and submit patient preference information
About Our Guests
Frank Sasinowski
Frank J. Sasinowski, M.S., M.P.H., J.D., has helped secure FDA approval for hundreds of new drugs, including more than 100 new molecular entities, often for serious and rare diseases. Frank has been involved in more than 50% of all the drugs FDA approved by way of the accelerated approval pathway (outside of cancer). Frank is also involved in many cell and gene therapies and aided significantly on the first approved systemic gene therapy, Zolgensma, as well as the other “curative” gene therapy Lenmeldy. Finally, Frank has helped spearhead the implementation of the alternative effectiveness statutory standard including through his involvement in approvals of Duvyzat, Miplyffa, Skyclarys, Filsuvez and Vyjuvek.
Frank joined FDA in 1983 as regulatory counsel in the Center for Drugs and Biologics, where he was key to implementing both the 1983 Orphan Drug law and the 1984 Hatch-Waxman law. In 1987, he left the FDA as Deputy Director of health policy in Commissioner’s office and joined Hyman, Phelps & McNamara.
In its March 2012 issue, the Drug Information Journal published Frank’s seminal analysis on therapies for rare disorders: “Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders.” Other papers of his on Accelerated Approval and Orphan Drugs are regularly cited by FDA, industry and academia as well. Thanks to his extensive FDA experience, both from within the Agency & from a sponsor’s perspective, as well as his passionate advocacy for integrating the voice of the patient in developing medicines, Frank possesses a nuanced & deep understanding of the drug regulatory process.
Since 2014, Frank has been Adjunct Professor of Neurology at U. of Rochester School of Medicine. His work has been widely recognized. (e.g., Frank was asked by both political parties to testify at the May 2014 inaugural congressional hearing on the 21st Century Cures law). In May 2013, the National Organization for Rare Disorders (NORD) awarded Frank its first ever Lifetime Achievement Award. In 2000, Frank was elected to NORD’s Board of Directors, where he served as Chair and as Vice Chair & was on its Board until 2016. In 2017 Frank joined the Board of the Everylife Foundation for Rare Diseases where he currently is its Vice Chair. In October 2012, President Obama recognized Frank’s contributions to the President’s Council of Advisors on Science and Technology (PCAST) report, “Propelling Innovation in Drug Discovery, Development and Evaluation.” Frank is a founding Board member of the IndoUSrare patient organization, representing those in India and in the US with rare conditions. Frank has also served on the Board of Directors of the United States Pharmacopeia (USP) & the ARM Foundation and has served on the board of several biotechnology companies.
Geoffrey Levitt
Geoffrey Levitt is Head of Global Regulatory Law at argenx.
Geoff previously held senior legal and policy roles at DLA Piper, Pfizer Inc., and Wyeth Pharmaceuticals. He also chaired PhRMA’s Law Section Executive Committee and the Board of Directors of the Food and Drug Law Institute.
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