Project ALS is encouraged by the introduction of two bills that aim to provide people with ALS faster, more robust access to experimental therapies. These bills are significant for specific, substantive reasons detailed below. Perhaps as importantly, they represent a sea change in ALS advocacy: they are both the product, from start to finish, of collaborative efforts led by ALS patients themselves to change a system that has not been working. We at Project ALS recognize the unwavering work of the patient-led advocacy organization I AM ALS, Team Stevens Nation, Corey Polen, and many others in this community, in bringing this legislation to the Senate, the House of Representatives, and the public.
Our mission at Project ALS is to fund research toward the first meaningful therapies for ALS—we have no lobbyists or policy arm. Our comments on these bills, then, speak to their potential on-the-ground impact for ALS research and drug development.
H.R. 7071, Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), introduced by Congressman Jeff Fortenberry and Congressman Mike Quigley.
This bill would, first, address a critical gap in ALS drug development by making $75M available for expanded access programs (EAPs) in 2021 and 2022. While the FDA has previously made it clear that they support the use of EAPs in ALS, most drug developers have not utilized this mechanism for financial reasons—it is expensive to make drugs available to people with ALS via EAPs, and data from these programs does not typically contribute to drug approvals. As a non-profit that aims to initiate an EAP for our first in-house drug candidate, Prosetin, as early in clinical development as possible, we strongly support this aim. First, all ALS patients should have access to potentially life-saving therapies. Second, in making EAPs the status quo for ALS drug development, the field will generate important data: longer-term treatment data, and data around patients who are normally excluded from clinical trials. We recommend that this legislation include a mandatory central registry reporting data from its funded EAPs.
This bill would also establish an FDA Center of Excellence for Neurodegenerative Diseases, modeled after its existing Oncology Center of Excellence. The Oncology Center of Excellence was established in 2017 to streamline and expedite the review of potential new cancer therapies by uniting all FDA divisions under one roof, and has been successful in implementing innovative approaches and faster drug approvals. Project ALS introduced the idea for an FDA Center of Excellence for Neurodegenerative Diseases at the More than Our Stories patient advocacy conference this February, and we are gratified by its inclusion in this bill. We support this aim and believe that it will incentivize and expedite clinical development for ALS and other fatal, currently untreatable brain diseases including Alzheimer’s, Parkinson’s, and Huntington’s.
Sandy Morris, ALS patient advocate and president of the I AM ALS Community Advisory Council, underscores the vital need to pass ACT for ALS. “There are 30,000 people in the US with ALS right now, and less than 1000 opportunities for these patients to access investigational therapies. This is where the rubber hits the road. This is where we build the pathways for ALS patients that have not existed, but we know are possible from other diseases. This is why we built the ALS caucus. This is where we see who the ALS leaders really are.”
Read this bill, and take action to help pass it, here.
S.3872, Promising Pathway Act, introduced by Senator Mike Braun, Senator Lisa Murkowski, and Senator Martha McSally.
This bill would establish a new FDA approval pathway for ALS and other serious conditions for which there are no meaningful treatments available, allowing promising ALS therapies to be provisionally approved—made available and covered by insurance for all people with ALS—much faster than ever before.
We strongly support the Promising Pathway Act, which provides a new FDA pathway to more rapid drug approval specifically for life-threatening diseases like ALS. At Project ALS, we have been concerned about limitations in the FDA’s existing Accelerated Approval pathway—specifically, its requirement for a fully validated surrogate biomarker—that have made it almost impossible to utilize in ALS drug development. The Promising Pathway Act will allow endpoints and biomarkers that are scientifically substantiated, but have not yet been studied for the years or decades required for the FDA’s standard of validation, to serve as the basis for provisional approval.
Critically, the bill also establishes a requirement of a patient registry for all provisionally approved drugs. This will ensure, first, that in order to stay approved, each ALS drug must show robust, long-term safety and efficacy. Second, this registry will provide invaluable real-world data about ALS to the broader scientific community.
Corey Polen, a person with ALS and constituent of Senator Braun who has worked closely with the Senator’s office toward the Promising Pathway Act, highlights this legislation’s potential impact for ALS and beyond: “This bill is much larger than ALS and pushes us closer to the mantra of Cures For All. This pathway provides treatments that have shown to be effective faster to patients with diseases that truly need it most without jeopardizing safety. We’ve seen how fast the FDA is willing to work for COVID. Our time is now to ensure ALS receives similar expediency.”
Read this bill, and take action to help pass it, here.
Project ALS is a research organization, but we are built on a simple outrage: it is unacceptable that, in 2020 as in 1998, ALS patients are too often told to go home and get their affairs in order—that there are no treatments available for them and there will not be in their lifetimes. We believe that in this moment, when science has advanced so far, but its promise is not yet reaching people with ALS, both of these bills are imperative. We support their passage and urge all other ALS non-profit organizations to do the same.