ALS TDI joins hundreds of ALS community members in commenting on FDA draft guidelines on ALS clinical trials

Over the last few months an enormous conversation has taken place surrounding a six page document published by the Food & Drug Administration (FDA) entitled Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment; Draft Guidance for Industry. The public comment period recently ended, and it is expected that well over 1,000 individual comments have been posted online, mailed or otherwise delivered to the FDA.

Comments came from all parts of the ALS community, including many from people with ALS themselves. A quick analysis of comments shows that at least 200 people who made comments identified themselves as a person with ALS (or similar disease) or commented on behalf of a person with ALS (or similar disease). Another 500 people who commented identified themselves as being related to a person diagnosed with ALS, including many comments from people who lost a family member to the disease.

Several pharmaceutical companies (including Cytokinetics, Amgen, GlaxoSmithKline, Denali Therapeutics) and ALS organizations (including Answer ALS, Team Gleason, ALSA, MDA, Les Turner) submitted comments. The ALS Therapy Development Institute’s CEO/CSO, Steve Perrin, submitted two separate comments: one submitted as a summary comment together with a group of organizations, neurologists and neuroscientists, and one submitted by Steve individually, the text of which is as follows:

Dear Draft Guidance Review Committee Member:

The draft guidance is a good start and we look forward to the consideration of all comments received from members of the ALS community by the FDA. We participated in a collaborative review effort of the draft guidance together with the ALS Association that included dozens of stakeholders including neurologists, neuroscientists and people with ALS. That effort resulted in a recent submission of a specific series of recommendations to enhance the current guidance.

One of the things that we were excited to read included in the draft version of the guidance is guidance from the FDA to drug developers to apply new clinical trials endpoints as they are validated. These may include endpoints based on changes in progression measured through the use of wearable technology, remote voice recordings or other approaches, in trial design. Initial results from efforts such as our own Precision Medicine Program, suggest the utility of these approaches may have vast implications in shortening the time required to conduct clinical research studies in people with ALS.

We encourage the FDA to continue to be aware of progress being made on the advancement of objective and sensitive endpoints such as these and to update this guidance with clarity on their application in clinical trials as soon as possible. We look forward to providing additional input on the development of this guidance in the future.

Sincerely,

Steve Perrin, Ph.D., CEO & CSO, ALS Therapy Development Institute

The comments provided a diverse range of ideas regarding the best way to guide drug developers in conducting clinical research studies in people with ALS. Without question, the most common thread across comments involved a request of the FDA to consider the lethal nature of ALS, and the rapid progression of loss faced by those diagnosed in writing their guidance to industry.

The next step for the FDA is to review all comments and to work on developing a final guidance document for industry. It is important to note that this document is a guide for industry, it does not limit those submitting clinical trial applications to the FDA to follow every aspect within the guidelines. The FDA will continue to update this document with information about running trials, as effectively and as swiftly as new information, ideas and tools become available.

Based on the comments submitted, the conversation about the guidance has not ended and it will continue as the FDA works to develop a final version of the document. While public comment on the draft guidance has officially closed, FDA rules allow for comments to be submitted anytime on a guidance, even those in development. Comments can be submitted to the FDA online here or via mail to Billy Dunn, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 4332, Silver Spring, MD 20993-0002.

ALS TDI held a public webinar on the drug development process at the FDA, including information about making comments on the guidance document in March. A summary of that webinar and link to its recording can be found online here. Our CEO/CSO, Steve Perrin is holding a public webinar on May 16, during which he will discuss several topics of interest including ALS TDI work to advance the potential application of wearable technology and voice recordings as endpoints in clinical trails to help make all go faster. That webinar is free and open to the public, but advance registration is required and can be completed online here.