March Event: A Window into the Changing Landscape of FDA Drug Effectiveness Standards

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A Window into the Changing Landscape of FDA Drug Effectiveness Standards


Program Speaker:

Frank Sasinowski

Director at Hyman, Phelps & McNamara, P.C.


Wednesday, March 17, 2021



FLYER

Event Agenda:

Date: Wednesday, 17 March 2021

Time*:

6:00 – 6:05 PM Welcome and Announcements

6:05 – 7:05 PM Program Presentation

7:05 – 7:30 PM Q & A *Times are approximate.


Program Topic:


Almost 60 years have passed since the requirements for efficacy were first added to the Federal Food, Drug, and Cosmetic Act with the Kefauver-Harris Amendments in 1962. Following that landmark addition to require evidence of efficacy for drug approval and marketing, what constitutes substantial evidence of effectiveness has continually evolved. These changes have been driven by numerous forces such as new statutory revisions, long overdue incorporation of the patient voice in regulatory decision-making, responses to the unique opportunities of drug discovery in the 21st Century as well as the hurdles of rare diseases and the need to quickly manage public health emergencies like Covid. FDA response to these forces comes in cycles, whereby every 20-25 years the Agency revisits and updates the drug effectiveness standards. Frank Sasinowski will draw upon his nearly 40 years of drug development experience to illuminate how the FDA appears to be on the cusp of another major revision. His talk will point to some of this history, examine the current signals of change coming from the FDA, and, most importantly, illuminate these signals by looking to his personal and recent experiences assisting pharmaceutical clients across the full spectrum of diseases and drug modalities.


Speaker Biography:

Frank J. Sasinowski, M.S., M.P.H., J.D., 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. Frank was involved in 6 of the most recent 8 drugs FDA approved by way of its accelerated approval process. He has helped secure FDA approval for hundreds of new drugs, including more than 100 new molecular entities, often for serious and rare diseases by assisting sponsors and patient organizations in developing new medicines. Frank is involved in many cell and gene therapies and aided significantly on the first approved systemic gene therapy, Zolgensma.


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 an Adjunct Professor of Neurology at the University Of Rochester School Of Medicine. His work has been widely recognized by industry and political leaders. For example, Frank was asked by both political parties to testify at the May 2014 inaugural congressional hearing on the 21st Century Cures law. In August 2018 Frank was appointed to the Board of Directors for the Alliance for Regenerative Medicine (ARM) Foundation for Cell and Gene Medicine. 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) and has served on the board of several biotechnology companies.


Location:

Not applicable - This is an online event

Online registration (ends 16Mar2021): $10 SDRAN Member

$10 Non-Member

For Questions Email: programs.committee@sdran.org