1:32 PM
Sep 13, 2018
 |  BC Extra  |  Politics & Policy

Woodcock: single FDA center for rare diseases could be 'negative' to rare disease cause

FDA's Center for Drug Evaluation and Research (CDER) Director Janet Woodcock advised against a single FDA Center of Excellence (COE) for rare diseases. Woodcock made the remarks while speaking at the EveryLife Foundation for Rare Diseases scientific workshop on Thursday in Washington.

Her comments come after Hyman, Phelps & McNamara's Frank Sasinowski and James Valentine proposed creating a Rare Disease COE for greater consistency and efficiency in regulatory review of rare disease products (see "Proposal Seeks FDA Center for Rare Diseases").

Sasinowski is a director and Valentine is an associate at Hyman, Phelps & McNamara, a firm specializing in FDA law. Sasinowski is also vice chair of EveryLife Foundation's board.

Woodcock argued a single COE would be negative to the rare diseases cause, emphasizing the need for a multidisciplinary approach to rare disease product review.

Having a matrix structure where FDA could assemble teams from "clusters of expertise" might suit rare disease product review better, Woodcock said.

"For any given rare disease, if you have a rare disease staff, they’re not going to be experts in that disease area," she said. Rare disease experts will need to work with other clinical subspecialists, such as nephrologists or pediatricians, as well as experts in small clinical trials.

Woodcock rolled out a proposal to reorganize and update new drug review in a June 1 town hall meeting, and she said Thursday those changes will be "very positive" for rare diseases. The plan, subject to congressional approval, would see new disease-specific divisions, including one that would participate in multidisciplinary review for all rare diseases (see "Getting Flatter").

Sasinowski and Valentine told BioCentury Thursday that separate conversations with Woodcock led them to understand she's open to the two other parts of their proposal: establishing a Rare Disease Advisory Committee; and appointing deputy or associate directors for rare diseases within each review office across CDER, the Center for Devices and Radiological Health (CDRH) and the Center for Biologics Evaluation and Research (CBER).

"Dr. Woodcock's closing statement about needing further conversations on this signals openness to the concept as well," they added in an email.

They agree multidisciplinary review is important, and said their proposed model would enhance multidisciplinary review "by ensuring there is rare disease expertise on the review team."

Sasinowski presented his proposal Thursday in an afternoon session of the workshop.

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