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.

In our headlines segment, Wayne highlights these major developments:

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

In our Resource Links segment, we list key documents in our four core areas. To view these links, subscribe, or find out more information about our podcast, visit FDAWatch.net.

Order the Food & Drug Law Institute's A Practical Guide to FDA's Food and Drug Law and Regulation, Eighth Edition.

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Music by Dvir Silver from Pixabay.

Cautious Optimism Surrounding Rare Diseases and Orphan Drugs