Rare Daily Staff
A pair of attorneys specializing in regulatory law have authored a proposal that calls for 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, was expected to be presented September 13 at the EveryLife Foundation for Rare Diseases scientific workshop in Washington, D.C. Sasinowski is vice chairman of the EveryLife Foundation.
The proposal comes 35 years after the passage of the Orphan Drug Act, which provides incentives to make developing drugs for small numbers of patients financially viable. The authors note that the act has increased investment in the research and development of medical products to address rare diseases, but the increasingly demand on the FDA to conduct oversight and review applications for rare diseases has created unique regulatory challenges.
“There is no separate, lower, or lesser legal or regulatory standard for approval of orphan products, so researchers and product developers and FDA alike must confront these issues throughout all phases of development and employ creative approaches to product development and review must be employed,” the attorneys wrote in a blog post on their law firm’s website. “Given the unique challenges and, therefore, the unique expertise needed to advance the development and review of products for rare diseases, a Rare Disease COE would provide the necessary infrastructure to allow centers and offices across FDA to consistently and efficiently review novel products for these conditions.”
The proposal seeks to leverage authority given to the FDA under the 21st Century Cures Act to create centers of excellence and would follow 2017 establishment of an Oncology Center of Excellence that the authors say has resulted in the approval of dozens of new therapies.
They say the proposed Rare Disease Center of Excellence would be modeled on the Oncology Center of Excellence and established within the FDA’s Office of Medical Products and Tobacco. It would be charged with cross-center responsibilities.
The proposal calls for the creation of deputy directors for rare diseases within each review office and division across the agency’s centers for drug, biologics, and devices to build rare disease expertise across the agency’s offices.
It also calls for the establishment of a rare disease advisory committee made up of external rare disease experts who could advise the agency on issues of rare disease medical product development and review.
September 13, 2018
Photo Frank Sasinowski, director of Hyman, Phelps & McNamara and vice chairman of EveryLife Foundation.