ARTICLE | Politics & Policy
FDA updates guidance on development of rare disease therapies
January 16, 2019 10:17 PM UTC
FDA has revised its draft guidance for developing rare disease treatments to include the use of biomarkers as surrogate endpoints in trials to support an accelerated approval.
Although the guidance notes that currently, clinical outcomes are the usual endpoints for adequate and well-controlled trials, sponsors may consider using biomarkers as surrogate endpoints. Sponsors would be required to first demonstrate analytical and clinical validation of the biomarker test...