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US FDA Reassures ALS Patients In Final Development Guidance

 
• By Derrick Gingery

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.

In final guidance on development of drugs for amyotrophic lateral sclerosis (ALS), the agency included much more patient-focused language in describing study designs, likely in part to avoid recruitment...

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• By Eliza Slawther

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• By Eliza Slawther

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• By Sarah Karlin-Smith

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• By Michael McCaughan

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