U.S. Senators introduced a bill that would create a provisional pathway to get treatments to patients faster, especially as patients facing life-threatening illnesses continue to hope for effective drugs to treat their conditions.
Sens. Mike Braun (R-IN), Martha McSally (R-AZ), and Lisa Murkowski (R-AK) introduced the Promising Pathway Act on June 4, 2020. The bill would require the U.S. Food and Drug Administration (FDA) to establish a rolling, real-time priority review pathway to evaluate provisional approval drug applications for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions.
For patients suffering from serious, life-threatening, or terminal illnesses such as ALS, the bill would provide more efficient access to new treatment and help eliminate the years of waiting for promising new drug therapies. To receive provisional approval status from the FDA under the bill, a drug would have to demonstrate substantial evidence of safety and relevant early evidence of positive therapeutic outcomes.
“We know that ALS is a tragic disease, but with advances in research, treatment, and advances in clinical trials, my hope is that we are closer to a cure for ALS,” Murkowski stated in a news release. “Our legislative effort recognizes that patients suffering from life-threatening, fast-moving diseases may benefit from access to promising treatments earlier.”
In addition to expediting beneficial outcomes for patients who cannot wait for the lengthy review process, the bill would encourage innovation in drug development and empower patients, Peter J. Pitts, a former FDA associate commissioner and president and co-founder of the Center for Medicine in the Public Interest, told Health Care News.
“It incentivizes innovators to pursue exciting new science through progressive and pragmatic 21st century science,” Pitts said. “Provisional approvals forge a partnership between patients, developers and the FDA—sharing acceptable risks and promising benefits for serious and life-threatening diseases.”
Differs from Other Pathways
There are other expedited pathways for drug approval by the FDA, such as the accelerated approval pathway, but the provisional approval under the Promising Pathway Act is less limited than other methods. For example, the accelerated approval pathway only applies to drugs being reviewed in clinical trials, which can limit access, Pitts says.
“Many of the FDA’s expedited pathways overlap—but they are also additive in important ways,” Pitts says. “The Promising Pathways Act makes it possible to bring new products to market with a guarantee of robust post-market safety and pharmacovigilance. It allows patients to have the final say in how they want to address their own health conditions and work with developers and regulators to identify both unexpected problems as well as exciting outcomes.”
Kelsey Hackem, J.D., (firstname.lastname@example.org) writes from Washington state.