The ALS Association announced that a critical effort made possible by the ALS Ice Bucket Challenge is underway, involving nearly 100 people with ALS and caregivers, researchers, clinicians, and industry experts from across the country to create drug development guidance designed to accelerate the development of therapies to slow or stop amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.
The guidance, which will be submitted to the FDA, will serve as a roadmap to help industry navigate the development process and provide the agency 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 effective at assessing drug efficacy. This will speed access, reduce costs, and help ensure resources are most effectively utilized, and incentivize industry to enter the ALS market and develop new treatments for ALS. The guidance initiative will also include a parallel effort to update ALS clinical trial guidelines that were developed more than 15 years ago.
"We are grateful to the millions of ALS Ice Bucket Challenge donors and to our partners across the ALS community for the opportunity to tackle this critical work with the FDA," commented Barb Newhouse, President and CEO of The ALS Association. "We know that a clearer, well-defined regulatory process is critical to bringing new therapies to people with ALS and even for encouraging the biopharmaceutical industry to invest in ALS drug development. In particular, it is essential that people with ALS are at the center of this initiative so that all participants in drug development, including the FDA, understand the urgent need to develop effective treatments and deliver them to people with ALS as fast as possible."
Building on recent decisions by Congress and the FDA that emphasize patient-focused drug development, The ALS Association and its partners have convened an array of talent that includes nearly 30 people with ALS and their family members, more than 10 ALS organizations, 45 of the world's leading ALS researchers and clinicians, and representatives from the National Institutes of Health and the pharmaceutical and biotech industries.
These organizations and individuals will focus on seven specific areas of ALS drug development and 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.
People with ALS and family members are playing vital roles in the guidance drafting process at all stages, including through a Patients and Caregivers Advisory Committee that reviews all work products. "The role of our group is to ensure that the perspective of people living with ALS is incorporated into every element of the guidance," said Ted Harada, the Committee's chair and a person living with ALS. "We must be a part of making drug development and clinical trials faster and more effective, and I am strengthened by the many people who have agreed to help drive this initiative forward."
The Association and its partners are working to produce consensus over the next several months in each of the seven topic areas to be incorporated in the draft guidance. That material will be posted for public comment in the February-March 2016 timeframe. Following that, it will be submitted to the FDA for review and incorporation into an official FDA guidance, which also goes through a public comment process. Every effort will be made to streamline and accelerate the timeline of the project so that it can impact the ALS drug development pipeline as soon as possible.
A list of those organizations and individuals participating in the guidance development process and a summary of the initiative are available on The ALS Association website, www.alsa.org.