4:17 PM
Sep 12, 2018
 |  BC Extra  |  Politics & Policy

Proposal seeks FDA center for rare diseases

An FDA Rare Disease Center of Excellence (COE) could bring greater consistency and efficiency to regulatory review for rare disease products, according to a proposal from Hyman, Phelps & McNamara's Frank Sasinowski and James Valentine.

Sasinowski is a director and Valentine is an associate at Hyman, Phelps & McNamara, a firm specializing in FDA law.

The co-authors envision a Rare Disease COE with a similar structure and function as FDA's Oncology Center of Excellence, the agency's first COE that was launched in January 2017.

Increased investment in rare disease R&D following the Orphan Drug Act's 1983 approval has led to "unique regulatory challenges" for FDA in providing regulatory oversight and in conducting review of marketing applications, Sasinowski and Valentine wrote in a separate blog post Wednesday.

Small sample sizes, limited natural history data and little precedent for biomarkers or clinical outcome assessments all contribute to challenges for sponsors and regulators, Valentine told BioCentury.

"Without any changes, the expertise in rare disease product development and review will continue to be disparate from division-to-division or office-to-office," Valentine told BioCentury -- an unfortunate outcome given the great deal of rare disease expertise at FDA, he added.

In their proposed model, the Rare Disease COE would sit within FDA's Office of Medical Products and Tobacco and harmonize regulatory approaches specific to rare diseases, as well as coordinate cross-center rare disease-focused meetings, among other tasks. FDA would appoint a deputy or associate director for rare diseases within each review office across the Center for Drug Evaluation and Research (CDER), the Center for Devices and Radiological Health (CDRH) and the Center for Biologics Evaluation and Research (CBER). It would also establish a Rare Disease Advisory Committee to advise on product development and review issues.

Sasinowski will present the proposed model Thursday at the EveryLife Foundation for Rare Diseases scientific workshop in Washington. He is vice chair of the foundation's board.

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