We are deeply grateful to the eight speakers who shared their wisdom and personal experience with the FDA, as well as the more than 150 others from the community who have done so online. We continue to hear that people with ALS want effective treatments now, even if they offer modest benefit, and are willing to face considerable uncertainty and risk to do so.
We know that 17 FDA staff in attendance heard the same message. It was clear from her remarks that Dr. Patrizia Cavazzoni heard our community. She spoke about the need for new treatments, that treatments might have modest benefit, and that they might not work for everyone with an ALS diagnosis. She talked about the need for regulatory flexibility and speed, and expanded access to experimental drugs. We are heartened by her message, and believe it was a genuine response, we need to make sure this is followed up with action. With words must come concrete actions.
In the immediate future, we will be asking the FDA to recommit to its 2019 ALS clinical trial guidance, to explain how it has implemented it, and how it plans to get effective treatments to people with ALS faster. We will be asking for a detailed report on these issues with a deadline.
This is part of a larger strategy to ensure the FDA is as fast and flexible as possible. This strategy entails several additional actions. In our appropriations request to Congress, we are asking for $50 million for the FDA to fund research on treatment and regulatory issues to benefit people with ALS. Second, we are supporting the Promising Pathways act to give the FDA additional tools to work faster. In addition, not only does the newly released ACT for ALS expand access for ALS treatments, it would give the FDA additional research capacity and public private partnerships to speed the development and approval of new ALS treatments. And, we are asking Congress to fund a study by the National Academies of Science and Medicine to help coordinate the entire ALS research agenda.
This is a complex set of interrelated proposals that should help keep the FDA on task. We will discuss them further at our annual Advocacy Conference, which begins on June 8. (If you have not done so, you can register for the virtual conference here.) The recording of today’s meeting and the portal to submit additional testimony from people living with ALS are accessible at www.als.org/fda.
This morning was very inspiring, we want to genuinely thank the brave individuals with ALS who spoke their truths. Our urgent work continues. We will keep you posted and look forward to discussing these issues further at our Advocacy Conference.