ALS Association

 


 

Statement on DRAFT FDA Guidance for ALS Drug Development

On February 12, 2018, the FDA announced its DRAFT Amyotrophic Lateral  Sclerosis: Developing  Drugs for Treatment – Guidance for Industry. A copy of FDA’s draft guidance can be found here.

The ALS Association is carefully reviewing the FDA draft and will submit comments. In addition, the Association encourages interested persons to submit comments directly to the FDA. The deadline to submit comments is April 17, 2018 and further instructions on how to submit comments can be found here.

The FDA’s DRAFT guidance is informed by the ALS community’s patient-focused guidance, docketed with the FDA in November 2017. More than 100 individuals from throughout the ALS community worked together to develop the guidance, including people with ALS, caregivers, ALS organizations, the world’s leading clinicians and researchers, industry leaders, and representatives from CDC and the NIH.

It is important to restate that the ALS community’s patient-focused guidance purposely includes content that goes beyond what is typically found in an official FDA guidance. With the encouragement of the agency, the community’s guidance was designed to provide much broader and deeper input to the FDA on topics that inform their understanding of ALS and the community consensus on key therapy development topics.   

A finalized FDA guidance and the community’s inputs will work in tandem to provide greater clarity for researchers in academic institutions and pharmaceutical companies. The goal here is to increase the efficiency, predictability, and speed of the drug development process, including clinical trials, and to accelerate access to new therapies for ALS.

The ALS Association will continue to advocate for the most helpful FDA guidance and expand its array of projects to accelerate treatment to people with ALS.