Bipartisan legislation would accelerate FDA drug approval process

Nov 7, 2023 by AFP

On average, it takes a decade and hundreds of millions of dollars to get a new drug or medication approved by the U.S. Food and Drug Administration (FDA). That’s bad news for people with life-threatening diseases and illnesses such as ALS (commonly known as Lou Gehrig’s disease) who don’t have the time to wait out the FDA approval process for new treatments.

That’s why Sens. Mike Braun and Kirsten Gillibrand along with House Reps. Mike Gallagher and Mike Quigley sponsored the Promising Pathway Act. This bipartisan effort would allow the FDA to speed up the process, making the treatments for patients available sooner.

The Promising Pathway Act takes the successful Operation Warp Speed (OWS) model that brought multiple COVID-19 vaccines to market in record time and applies it to a wider swath of potentially lifesaving and enhancing new drug therapies.

One of the tenets of AFP’s Personal Option initiative is adopting an OWS-like approach to the development of all therapies. The Promising Pathway Act is an important measure that can significantly speed up access to health care for all Americans patients and reduce the cost of producing critical new cures.

The Promising Pathway Act would streamline the FDA drug approval process

Currently, the FDA drug approval process has three phases of clinical trials to test the safety and efficacy of a new drug.

Under the Promising Pathway Act, once a drug has successfully completed a phase 2 clinical trial and has demonstrated it is both safe and effective, the manufacturer has the option to seek provisional approval from the FDA. The provisional approval lasts for two years, and the manufacturer can request three subsequent two-year renewals for a total of eight years.

In phase 3 of clinical trials, the drug’s marketing may coincide if the FDA grants provisional approval, allowing drugmakers to recoup their investment on a major therapy earlier in the development process. This would not only save lives but also accelerate innovation. Under the new legislation, the FDA would have 90 days to grant or decline a provisional approval application.

Three Key Components of the Promising Pathway Act

Only those drugs for serious or life-threatening conditions would fall under the proposed reform. The drug must align with the following conditions:

The primary purpose of the drug is to treat, prevent, or diagnose a serious or life-threatening disease or condition, especially where there is a reasonable likelihood of premature death without early medical intervention.
The drug provides a positive therapeutic outcome.
The drug matches or surpasses the effectiveness of any currently marketed on-label therapies, all while having fewer negative side effects.

How the Promising Pathway Act would protect patients

To use a provisionally approved drug, patients must agree to participate in the FDA’s ongoing data collection efforts. These efforts are aimed at monitoring a drug’s efficacy and identifying any negative side effects.

Additionally, the legislation requires the FDA to withdraw provisional approval in cases where a substantial number of patients encounter severe adverse effects when compared to patients relying on other, currently approved therapies for the same disease or illness.

Compared to the status quo, where potentially lifesaving and life-changing drug therapies are withheld from patients during trial phases – despite being provisionally deemed safe and effective – this reform is a major step forward.

People with rare and progressive diseases don’t have time for the FDA’s laborious approval process. Congress should pass the Promising Pathway Act, fast.

Learn more about how the Personal Option would bring safe and effective cures to people more quickly.