Advocates Press For US FDA Rare Disease Center Of Excellence
Attorneys' proposal aims to better connect dispersed rare disease expertise within FDA's drug, biologic, and device centers through more leadership, new advisory committee.
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Number one ask is for congressional pressure to centralize rare disease expertise at the agency, but advocates also are preparing legislation that would mandate the new center.
FDA's Peter Stein calls idea "useful"; rare disease experts would temporarily serve on standing advisory committees when needed.
Common system throughout FDA also could facilitate cross-center product reviews; Sentinel program is template for the approach.