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Statement from Acting Commissioner of Food and Drugs Norman E. "Ned" Sharpless, M.D., Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research, and Janet Woodcock, M.D., director of the Center for Drug Evaluation and Research on final guidance to help advance development of novel treatments for ALS

SILVER SPRING, Md., Sept. 23, 2019 /PRNewswire/ -- Over the past few years we've witnessed new medical breakthroughs that are altering how diseases are treated in ways that seemed unimaginable just a decade ago. We recognize that scientific progress across different disease and therapeutic areas has been uneven, particularly lagging for complex neurological diseases, such as amyotrophic lateral sclerosis (ALS). In part, this is because the underlying causes of severe neurological diseases are often not well understood, contributing to the challenge in the development of drug and biological treatments.

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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. 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. We recognize the kind of strength it takes to battle ALS every day and the critical, unmet medical need that exists.

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 We appreciate the many thoughtful comments submitted to the docket for the draft guidance and other feedback we received.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.

Specific to clinical trial design, this final guidance provides clarity around some of the questions raised by stakeholders. It emphasizes that all patients in ALS trials should receive the best standard of care, and no patient should be denied effective therapies in order to be randomized to a placebo-only arm. While exposure to placebo may be the best way to determine the effectiveness of some products, the guidance also explains various strategies that can be applied to 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. As such, we stand ready to use the expedited development and approval programs available to help bring new treatments for ALS to patients as quickly as possible.

We also know that many patients with ALS are seeking access to investigational products for this devastating disease. The FDA remains committed to helping patients and health care professionals evaluate options for accessing investigational products, such as participating in a clinical trial, obtaining access to an unapproved, investigational product outside of a clinical trial through expanded access (compassionate use) or providing information for patients about the Right to Try Act. However, it's important to note that for a patient to receive a treatment under expanded access or Right to Try, the manufacturer or sponsor must be willing to provide it. Additionally, we encourage sponsors to offer access to investigational products after clinical trials are complete when continued access to a promising medicine would be appropriate under the expanded access program.

The FDA is generally prohibited by law from releasing confidential information about investigational products, so we ask that companies developing treatments for ALS provide information about the status of their research and information on availability of products under expanded access or Right to Try so that patients, caregivers and physicians can have informed discussions. For outcomes associated with use under expanded access, as explained in our expanded access guidance, FDA reviewers understand that assessment of any serious adverse event must consider the context in which the investigational drug or biologic is provided. We want to reassure sponsors that providing a drug under expanded access very rarely impacts development timelines.

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.

Media Inquiries: Sandy Walsh, 301-796-4669, sandy.walsh@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

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