US FDA Reassures ALS Patients In Final Development Guidance

ALS advocates succeed in getting the FDA to soften its stance on use of randomized, placebo-controlled trials for drug development; final guidance also encourages patient-reported outcomes.

nerve cells, concept for neurodegenerative and neurological disease
The FDA encouraged development of endpoints that measure function in daily living in final ALS drug development guidance. • Source: Shutterstock

New ALS drugs should still use randomized, placebo-controlled trials to show efficacy, the US Food and Drug Administration says, but the agency clarified that it is not necessarily married to the traditional study design for the products.

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