100 ALS Community Members Create New Drug Development Guidance
The ALS Association, the country’s premier organization committed to find a cure for ALS and improving the lives of those affected by this rare disease, has just announced the viral and recently relaunched ALS Ice Bucket Challenge has played an integral role in pushing the creation of a new drug development guidance geared towards accelerating the development of ALS therapeutics, thanks to a nearly 100-strong group of ALS patients, caregivers, scientists, clinicians, and industry experts from across the United States.
The group behind the guidelines will be tackling 7 key areas of ALS drug development from a diverse community-involved perspective: diagnosis, natural history, clinical trials and outcome measures, biomarkers, benefit-risk, frontotemporal dementia, and public policy. The new drug development guidance will soon be ready for submission to the US Food and Drug Administration (FDA) as a comprehensive reference for the industry in approaching ALS research and development. Its goal is to make the clinical development process more efficient, and the treatment evaluation more reliable. Not only will achieving these goals helps companies save on development costs and resources, and encourage more growth in the ALS industry, patients will also gain access to newer and better treatments sooner. The new guide is also pushing a parallel initiative to amend current ALS clinical trial guidelines, which date back 15 years.
“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.”
The diverse group behind this initiative shows the vital role ALS patients and their families play in affecting large-scale changes in how a disease is recognized and addressed. “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 fellow ALS patient. “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 ALS Association and its partners will be focusing on producing consensus in each of the 7 key topics of the guidance in time for public posting come February-March 2016. The material will then be submitted to the FDA for review and eventual integration into the official agency guidelines on ALS.