FDA Updates Rare Disease Guidance for Drug Developers

January 17, 2019

Rare Daily Staff

The U.S. Food and Drug Administration issued updated guidance for the development of therapeutics to treat rare diseases.

The draft guidance revises the one the agency issued on rare disease drug development in August 2015.

It includes changes to the section on natural history studies, issues for evaluation and validation of surrogate biomarkers, and  information on external controls and early randomization.

“Although the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs, these issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, natural history data, and drug development experience,” the agency said.


January 17, 2019

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