Comment from ALS Association
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Summary: The ALS Association supports the FDA's draft guidance on New Approach Methodologies (NAMs), praising its focus on human-relevant science and fit-for-purpose evaluation. They argue that these methodologies are critical for accelerating drug development for ALS by providing more accurate data than traditional animal models, and they urge the FDA to ensure these methods are clearly defined to facilitate both approval and patient access.
The ALS Association appreciates the opportunity to provide comments on the U.S. Food and Drug Administration’s (FDA’s) draft guidance, General Considerations for the Use of New Approach Methodologies (NAMs) in Drug Development. We commend FDA for taking this important step to modernize how emerging scientific methods are evaluated and incorporated into regulatory decision making.
Accelerating the development of effective therapies requires improving the quality, relevance, and speed of early development decisions. Drug development strategies that more closely model human biology, especially in the central nervous system and brain, may expedite development timelines, decrease the rate of attrition, and enhance the probability that therapies advancing to late-stage trials are likely to achieve significant clinical outcomes. FDA’s draft guidance on NAMs is therefore highly relevant to ALS and represents an important opportunity to align regulatory science with patient urgency.