FDA updates guidance on development of rare disease therapies

BioCentury

17 January 2019 - The FDA has revised its draft guidance for developing rare disease treatments to include the use of biomarkers as surrogate outcomes in trials to support an accelerated approval.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.

Additionally, the guidance said that early biomarkers of disease or effects of interventions could be used in adaptive seamless trial designs to help inform endpoint selection and achieve greater efficiency. Adaptive seamless trial designs may be especially useful when there are limited numbers of patients to study. The agency said sponsors should submit a thorough statistical plan with key features of the trial design to discuss with FDA before beginning the study.

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Michael Wonder

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Michael Wonder