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ULTRA Bill Introduced in the House Legislation Seeks to Permit Broader Use of Scientific Data to Support Surrogate Endpoints for 'Ultra Orphan' Drug Approvals

Posted 03 January 2012 | By

Two congressmen have introduced legislation that would amend the Food, Drug and Cosmetic Act to provide an easier, accelerated approval pathway for drugs for "Ultra Orphan" diseases, which affect fewer than 6,000 people.  In comparison, "Orphan Diseases" affect fewer than 200,000 individuals.

The bill, Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA) (H.R. 3737), would allow for surrogate endpoints that is "reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predict clinical benefits or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity."

As a result of the small patient population, conducting clinical trials in ultra-orphan populations is particularly difficult, which in turn affects the amount of study data available.  The act aims to partially offset this difficulty by banning the US Food and Drug Administration from requiring historical clinical data or clinical treatment data for approval if that data is not available.

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