The FDA finalized its guidance on developing drugs to treat amyotrophic lateral sclerosis (ALS) with the aim of fostering the availability of treatments for the complex neurological disease. "Despite years of research and the availability of some approved therapies, we know the lack of new treatments for ALS is deeply frustrating for patients and caregivers," the agency said. The guidance includes recommendations on clinical trial design and ways to measure effectiveness, answering some of the questions raised by stakeholders. While placebo controls may be the best way to determine the effectiveness of some products, the guidance emphasizes that no patients in ALS trials should be denied effective therapies so they can be randomized to a placebo-only arm. Instead, all patients in the trials should receive the best standard of care. The guidance explains various strategies – such as master protocols, adaptive designs and enrichment strategies – to minimize unnecessary exposure to placebo and expedite trials. Stressing its commitment to help patients with ALS access experimental treatment, the FDA asked companies developing ALS treatments to provide information about the status of their research and information on availability of their drug candidates through the agency's compassionate use or the Right to Try programs.
To minimize possible disruptions in the U.S. drug distribution supply chain, the FDA said it would delay, until Nov. 27, 2020, the requirement for wholesale distributors to verify a product identifier before further distributing returned drugs. The grace period is part of a final guidance the agency issued on its compliance policy for the wholesale distributor verification requirement for saleable returned drugs. The requirement is a provision of the Drug Supply Chain Security Act. Other requirements will still take effect Nov. 27, 2019, the agency said.