Later this month, the ALS Association will host a virtual session focused on the urgent need for timely access to new therapies. Leaders from the U.S. Food and Drug Administration (FDA) and industry companies will listen to people living with ALS speak directly about their expectations for their experiences with the disease and how important it is to have timely access to therapies that have the potential to provide incremental benefit in improving how people feel, function and live.
This event takes place at a critical time for the ALS community as we continue engage regulators as they review potential new therapies and companies as they decide if and when to provide expanded access to drugs in development. We continue to urge the FDA to exercise its regulatory authority as outlined in its final 2019 ALS Guidance to grant the earliest and widest possible access to drugs that have met their endpoints in well-designed clinical trials. We also continue to urge companies with drugs that have shown benefit in development to provide expanded access at the earliest possible time.
This month’s meeting, while taking place in that context, is about much more than a single company, drug or development program. This meeting is about ensuring that FDA reviewers and leaders understand how our community evaluates and values the potential for incremental benefit – extra months of life, slowed disease progression that could mean extra months of being able to talk, or walk, or even breathe on one’s own. These seemingly modest benefits are real to people living with ALS. They add value and quality to life – and when combined with potential incremental benefit from the next novel agents to demonstrate positive results in a well-designed trial – could lead to significant impact on the trajectory of this disease.
We have invited multiple people living with ALS to speak during the session, and have asked several ALS organizations to invite additional speakers. We would like to provide multiple perspectives about the need for timely access to potential treatments. To ensure that a broader group of people can be heard from by the FDA, the Association invites all member of our community to submit their perspectives in writing and by video at this link. These submissions will be compiled and provided to the FDA and company officials, and may be shared with the public.
Officials from the FDA, as well as the companies, will be in “listening mode” during the meeting. The decision not to livestream the event was made by the FDA, but we have requested that the meeting be recorded and shared with the community so everyone can see and hear the discussion. We will share the recording and details following the meeting.