Today, U.S. Senator Kirsten Gillibrand held a video press conference to announce her bipartisan legislation to expedite provisional FDA approval of drugs intended to treat ALS, Parkinson’s, Alzheimer’s, and other life-threatening diseases. The standard FDA approval process takes many years to get drugs to market, but patients with rapidly progressing illnesses often don’t have many years to wait. Gillibrand’s Promising Pathway Act would accelerate the FDA’s approval process and help get promising treatments to those who need them.
“Patients with life-threatening illnesses can’t wait for years for the FDA to approve new drugs,” said Senator Gillibrand. “They need treatment as soon as possible, and this bill would help make sure they get it. The Promising PathwayAct would open a provisional FDA approval pathway for drugs and therapies that treat conditions where the disease progresses rapidly and few to no treatment options exist while continuing to ensure that these new therapies are safe and effective. I’m proud to be leading this important legislation and look forward to working across the aisle to get it passed.”
The FDA’s drug approval process involves multiple phases: early clinical trials that establish and confirm safety and give evidence of effectiveness, as well as a final confirmatory trial. The final stage is particularly expensive and time-consuming, meaning that many patients die before potentially lifesaving drugs make it to market. The Promising Pathway Act would create a safe, accelerated provisional approval pathway for promising drugs and make these drugs available to those who need them. Specifically, the bill would allow pharmaceutical companies to petition the FDA for provisional approval of a drug that has cleared early-stage clinical trials and shown significant evidence of effectiveness. Provisional approval would make these drugs available to those whose lives depend on them while pharmaceutical companies continue to collect data and pursue full approval. This change would benefit patients fighting ALS, Duchenne Muscular Dystrophy (DMD), Parkinson’s, Alzheimer’s, childhood diffuse intrinsic pontine glioma (DIPG), and a number of other terminal illnesses.
Senator Gillibrand leads the Promising Pathway Act in the Senate with Senator Mike Braun (R-IN). Representatives Mike Gallagher (R-WI) and Mike Quigley (D-IL) are introducing companion legislation in the House.