RARE Daily

Bipartisan Legislation Being Introduced to Accelerate FDA Approval of Drugs to Treat Life-Threatening Illnesses

April 27, 2023

Rare Daily Staff

A bipartisan group of legislators are introducing the Promising Pathway Act, legislation to expedite provisional FDA approval of drugs intended to treat ALS, Parkinson’s, Alzheimer’s, and other life-threatening diseases.

They said 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. The legislation would accelerate the FDA’s approval process and help get promising treatments to those who need them.

The legislative effort is being led by Senator Kristen Gillibrand (D-New York) and Senator Mike Braun (R-Indiana) in the Senate and Representatives Mike Gallagher (R-Wisconsin) and Mike Quigley (D-Illinois) in the House. Braun had introduced similar legislation in 2021.

“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 Pathway Act 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.

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.

Photo: Senator Kristen Gillibrand, D-New York

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