February 22, 2018
Authored and Edited by Bonnie Fletcher Price, Ph.D.; Shana K. Cyr, Ph.D.
On February 15, 2018, FDA released draft guidance on Amyotrophic Lateral Sclerosis (ALS): Developing Drugs for Treatment. The guidance addresses the clinical development of drugs intended to treat the main neuromuscular aspects of ALS (i.e., muscle weakness and its direct consequences including shortened survival) rather than other ALS symptoms (e.g. muscle cramps, spasticity, sialorrhea, and pseudobulbar affect).
The guidance focuses on clinical drug development and trial design issues that are unique to the study of ALS. In particular, the guidance focuses on issues created by the highly variable course of ALS progression among patients.
Given the high variability among individual patients, FDA discourages the use of historically controlled trials, instead recommending randomized, placebo-controlled, double-blind, studies to demonstrate efficacy. Additionally, it recommends the assessment of various efficacy outcomes, such as effect on daily activity, in conjunction with assessment of survival benefit and need for full-time respiratory support.
FDA also recommends balancing treatment arms with respect to key prognostic factors to counter the high variance in survival time. Additionally, FDA recommends statistical analyses that combine survival and function into a single overall measure to prevent confounding of functional endpoints by data loss due to patient deaths.
Given the serious and life-threatening nature of ALS, the full array of typical clinical pharmacology studies may not be needed prior to approval. For example, carcinogenicity studies, and studies on the effects of renal or hepatic impairment may generally be deferred until after approval.
Readers are encouraged to read the draft guidance, also available on the FDA website. FDA requests that comments on the draft guidance be submitted by April 17, 2018.
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