New Report to the President Suggests Acceleration of Drug Approvals for People with ALS

Last week, the President’s Council of Advisors on Science and Technology (PCAST) released its “Report to the President on Propelling Innovation in Drug Discovery, Development and Evaluation,” which lays out a number of recommendations for doubling the development of new therapies for diseases with unmet medical needs. The ALS Association is encouraged that this report specifically references ALS, and that the group recognizes that a more aggressive timeline is necessary to find treatments and a cure for this disease.  

PCAST is an advisory group of the nation’s leading scientists and engineers who directly advise the President and the Executive Office of the President. It makes policy recommendations in the many areas where understanding of science, technology, and innovation is key to strengthening our economy and forming policy that works for the American people. 

The report is positive news for people suffering from ALS because it recommends that the U.S. Food and Drug Administration (FDA) should expand the scope of acceptable endpoints used to approve drugs for serious or life-threatening diseases with unmet needs. The FDA establishes acceptable endpoints for clinical trials.  An endpoint is the quantifiable goal or target that would indicate an acceptable improvement in the patient.

“The recommendations in the PCAST report are important steps in the right direction and demonstrate that The ALS Association and the people with ALS across this country are being heard; that the FDA and Congress recognize that much more can and must be done to find a treatment for this disease as soon as possible,” said Steve Gibson, Chief Public Policy Officer for The Association. 

The report provides examples of possible clinical endpoints suitable for accelerated approval by the FDA for two serious diseases lacking good treatments: spinal muscular atrophy, a genetic neuromuscular disease, and ALS. Targets, for example, that might predict a drug’s likely impact could include “the amount of air that a patient can exhale by force (a measure of lung capacity known as forced vital capacity) or functional motor tests as an endpoint for predicting a drug’s likely impact…”

The ALS Association will continue to actively engage the FDA and Congress to develop and implement policies that encourage, facilitate and expedite the development of treatments for ALS and ensure that people with ALS have access to those treatments as soon as possible.  To join the fight in finding treatments and a cure for ALS become an advocate today.