Making the Case for an FDA Rare Disease Center of Excellence

Making the Case for an FDA Rare Disease Center of Excellence

Two attorneys specializing in regulatory law recently proposed the creation of an FDA Rare Disease Center of Excellence to better allow the agency to address the challenges of advancing and reviewing rare disease products. The proposal, advanced by Hyman, Phelps & McNamara’s Director Frank Sasinowski and Associate James Valentine, also calls for the creation of deputy directors of rare disease at various FDA divisions and the creation of a rare disease advisory committee made up of external experts. We spoke to Valentine about the proposal, why these measures are needed, and the early response from one key figure at the FDA.


Betsey King, MEd

Senior Vice President, Client Services, Medistrava, an Inizio company

6 年

Thank you James for everything you do for patients!!!

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Gen Li, PhD, MBA

Founder & President, Phesi

6 年

While I applaud all the efforts from all parts of our industry on rare diseases, I don't think by adding another regulatory body is a positive move, neither to patients nor to the industry. The rationale of lacking expertise on rare diseases currently in FDA cannot be improved by adding a team of managers. Expertise on rare diseases, like those on all other diseases, should come from those experts in the front line in studying the disease and treating the patients. Objectively and dynamically identify and manage each pool of the related experts are now feasibly through revolutionary progresses on big data and artificial intelligence.

T Anthony Howell

Business Development Strategist & Closer - Solution Providers to Life Sciences

6 年

Definitely worth a listen and share...

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