FDA Releases Guidelines on Effective ALS Treatment, Trial Design

The FDA has released updated guidelines surrounding the treatment of amyotrophic lateral sclerosis (ALS) in order to provide the industry with its current scientific thinking so that effective treatments with a favorable benefit to risk profile can be most efficiently developed, studied, and ultimately made available to patients. The report is the result of collaboration among experts across the agency and incorporates important input from patients, researchers, and advocates.

“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,” Norman E. Sharpless, MD, acting commissioner of the FDA, said in the prepared statement released today. “Staff from the FDA have been meeting regularly with members of the ALS community, including patients, their families and caregivers, and have heard their concerns loud and clear.”

The final guidance includes proposals to help advance the development of products for ALS patients, including recommendations on clinical trial design as well as ways to measure effectiveness. Additionally, the document recommends that researchers and companies interact with the FDA early in product development so that the agency can best advise on proposed development programs and the efficient design of trials to produce the data needed for FDA approval.

The guidance also clarifies around questions often posed by stakeholders, such as the use of placebo. Although the guideline indicates that the use of placebo may be the best way to determine the effectiveness of certain products, it also gives various strategies that can minimize unnecessary exposure to placebo and expedite trials, such as master protocols, adaptive designs, and enrichment strategies.

“ALS patients deserve effective treatments, just as patients with any other disease or condition do. The statutory standards for effectiveness apply to drugs and biologics for ALS just as the standards apply for all other drugs and biologics. However, the FDA has long stressed the appropriateness of exercising regulatory flexibility in applying the statutory standards to medical products for serious diseases with unmet medical needs, while preserving appropriate assurance that they are effective and have a favorable benefit to risk profile,” Dr Sharpless added.

The agency also asked that companies developing treatments for ALS provide information about the status of their research and information on availability of products under expanded access or the Right to Try Act so that patients, caregivers, and physicians can have informed discussions.

“Patients are at the forefront of everything we do. That’s why the aim of issuing this final guidance is to help foster the availability of treatments. The FDA remains deeply committed to supporting the ALS community in its efforts to fight the toll that this devastating disease takes on all those affected,” Dr Sharpless said.

Find the link to the full report on the FDA website.

Reference

• Statement on Final Guidelines to help Advance Development of Novel Treatments of ALS [press release]. FDA agency website. Published September 23, 2019. https://www.fda.gov/news-events/press-announcements/statement-final-guidance-help-advance-development-novel-treatments-als. Accessed September 23, 2019.