FDA issues final guidance for Industry on Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment

FDA issues final guidance for Industry on Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment

The FDA has a long-standing commitment to working with researchers, drug companies and the patient community to facilitate the development and approval of new products to treat ALS. To further support that effort, today the FDA issued a final guidance for industry, Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment. The final guidance provides industry with the FDA’s 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. It is the result of collaboration among experts across the agency and incorporates important input from patients, researchers and advocates. We appreciate the many thoughtful comments submitted to the docket for the draft guidance and other feedback we received. We carefully reviewed and considered all comments as we developed this final guidance.

The final guidance includes up-to-date information on our recommendations to help advance the development of products for ALS patients, including recommendations on clinical trial design and ways to measure effectiveness. We know that an important element of product development is working early and often with researchers and companies to help facilitate their clinical development programs. That’s why our final guidance recommends that researchers and companies interact with the FDA early in product development so that we can best advise on proposed development programs and the efficient design of trials to produce the data needed for FDA approval in order to get effective therapies to patients as efficiently as possible. Because this guidance presents non-binding recommendations for the FDA and sponsors, the FDA is open to considering alternative approaches to meeting our requirements for approval.

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. The FDA looks forward to continuing our dialogue with the ALS community, and is looking into how to proceed with additional meetings that will best facilitate this engagement.

To learn more, please visit: FDA Statement.