FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

FDA Will Widen Expanded Access Tool to Cover Orphan Drugs

Nov. 9, 2017
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Writing in an FDA blog post, Commissioner Scott Gottlieb said the agency will broaden the scope of its Expanded Access Navigator tool — an information resource created by the Reagan-Udall Foundation to speed up pre-approval access to drugs.

Previously used only for cancer drugs, the expanded tool will cover drugs that treat orphan diseases, Gottlieb said.

Orphan diseases are ideal candidates for expanded access, as many patients don’t have access to FDA-approved drugs, he said. The traditional development process for such drugs can include numerous obstacles. For example, the diseases have patient populations small enough that recruiting clinical trial subjects is difficult. Similarly, patients with rare diseases have no single, consolidated place to find companies that offer expanded access drugs.

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