The ALS Association strongly supports initiatives to enable people with ALS to access promising treatments as soon as possible, including prior to FDA approval. Our primary goal is to facilitate the development of effective treatments and help support delivery to everyone with ALS as soon as possible.
The A.C.T. ALS Act is well-intentioned. It could assist some people with ALS to access experimental drugs outside of clinical trials through a regulatory mechanism we have long supported: the FDA’s expanded access program. However, we are concerned that this bill, as it is written today, may delay getting treatments to everyone with ALS as soon as possible. The current iteration appears to support underwriting access for a few people with ALS to expensive experimental therapies that have not yet been proven effective and that are therefore not covered by insurance or Medicare. Below, we summarize concerns about possible unintended consequences of the bill as currently drafted, with the hope that this statement can constitute a first step toward dialogue with Reps. Jeff Fortenberry (R-Ne.), Mike Quigley (D-Il.), and other stakeholders. The objective of this statement is to find ways to improve the bill to maximize its potential for passage.
First, this bill devotes up to $450 million over four years to provide access to experimental therapies through expanded access. This is a very significant sum of money that will provide access to experimental therapies for only a fraction of people living with ALS. We are concerned that it will detract from federal support for ALS research. We ask that this bill be revised to redirect a substantial portion of the funding requested toward research for a cure. In short, we would like to see a net increase in federal clinical trial resources.
Second, the current version of the bill makes no special provision for people who serve as volunteers for experimental therapies in clinical trials. Open label extensions allow people who participated in a trial to take the experimental drug even after they are done with the trial. If someone bears the risk of testing a drug, they should be able to take it if they and their doctor think it offers some benefit. We ask that this bill be revised to set aside funding for open label extensions in honor of the sacrifices and risks research volunteers make on behalf of the entire ALS community by participating in clinical trials.
Third, we are concerned about creating additional federal bureaucracies that are not singularly focused on finding effective ALS treatments as soon as possible. As written, the bill would create a Center of Excellence for Neurodegenerative Diseases with the same authorities and duties as the FDA’s Center of Excellence for Oncology: “to achieve patient-centered regulatory decision-making through innovation and collaboration.” This approach is about improving processes, not helping people. People with ALS need results fast. We ask that this bill be revised to ensure that any new bureaucracy that is created has as its central focus finding effective treatments for ALS as soon as possible.
Our commitment is, and will continue to be, to find and make available therapies and a cure for everyone with ALS, now and in the future. We look forward to working to calibrate this bill in a way that best serves the needs of the entire community of people with ALS and that maximizes the bill’s odds of success.