Thanks to resources made possible by the ALS Ice Bucket Challenge, The ALS Association has launched a groundbreaking initiative to create a patient-focused drug development guidance that is designed to accelerate the development of therapies to slow or stop amyotrophic lateral sclerosis (ALS). The guidance, which will be submitted to the Food and Drug Administration (FDA) this spring, will serve as a roadmap to help the pharmaceutical industry navigate the development process and provide the FDA with an ALS community-centered view of how it should approach therapies for ALS. The goals are to make the drug development process, including clinical trials, more efficient, predictable, faster, and more effective at assessing drug efficacy. This will speed access, reduce costs, help ensure resources are most effectively utilized, and incentivize industry to enter the ALS market and develop new treatments for ALS. The guidance initiative also includes a parallel effort to update ALS clinical trial guidelines that were developed more than 15 years ago.
The development of the draft guidance is a community-wide effort that involves more than 100 individuals representing all segments of the ALS community nearly 30 people with ALS, their families and caregivers, 45 of the world’s leading ALS researchers and clinicians, over 10 different ALS organizations, and representatives from the National Institutes of Health, the Centers for Disease Control and Prevention and the pharmaceutical and biotech industries.
These organizations and individuals are focusing on seven specific areas of ALS drug development to provide an ALS community-wide view on topics that include diagnosis, natural history, clinical trials and outcome measures, biomarkers, benefit-risk, frontotemporal dementia, and public policy. These topic areas will form the content of the guidance.
Currently only one drug is approved to treat ALS; however that drug only moderately extends survival and only in some patients. A clearer, well-defined regulatory process is critical to bringing new therapies to people with ALS as fast as possible. The draft guidance not only will bring more predictability and clarity to drug development, but also ensure that the patient perspective is considered at every stage of the development and approval process. This is essential in order to speed access to new treatments. People living with ALS simply don’t have time to wait.