‘Promising Pathway Act’ Will Save Lives Over and Above ‘Right to Try’ and ‘Expanded Access’

The best hope for these Americans and future generations facing terminal and rare diseases is the recently reintroduced Promising Pathway Act.

The U.S. Food and Drug Administration (FDA) in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

Commentary

Approximately one in every ten Americans is affected by one of approximately 7,000 rare diseases, of which many are terminal. For many of these diseases, fully Food and Drug Administration (FDA)-approved cures are not yet available, and in some cases, cures may be available, but take too long to reach the patients. For example, approximately 300 children in America are diagnosed with DIPG (diffuse intrinsic pontine glioma), a rare and highly aggressive brain cancer with a life expectancy of under one year.

The best hope for these Americans and future generations facing terminal and rare diseases is the recently reintroduced Promising Pathway Act (PPA) 2.0 sponsored by Senators Mike Braun (R-Ind.) and Kirsten Gillibrand (D-N.Y.), and co-sponsored by Kevin Cramer (R-N.D.), Joe Manchin (D-W. Va.), Eric Schmitt (R-Mo.), Alex Padilla (D-Calif.), J.D. Vance (R-Ohio), Cory Booker (D-N.J.), Josh Hawley (R-Mo.), Peter Welch (D-Vt.), and Lisa Murkowski (R-Ala.).

PPA fills the gaps in the well-intended Right to Try and Expanded Access in three key areas: faster timelines, a third-party register of results data, and insurance coverage.

Although both Right to Try and Expanded Access have noble goals of connecting patients with promising cures, the timelines are far too long. That is, although the FDA claims that the average access time to a treatment could be quite soon, the real timelines are substantially longer. When connecting a terminally ill patient suffering from a rare disease to a promising cure, the clock starts with the time that it takes for a doctor to first diagnose the rare disease. Next, the doctor must talk with the family and agree to a protocol, followed by developing a dosing schedule with the approving drug company, and then getting approval from the hospital’s internal institutional review board (IRB). The final stage of FDA approval typically consumes four days.

Taken together, under the current Right to Try and Expanded Access regulations, the actual earliest possible timeline from diagnosis to treatment of a rare disease is 22 days. Meanwhile, some diseases have a substantially shorter life expectancy. For example, DIPG patients have just 14 days of life expectancy after progression.

Current options such as Right to Try and Expanded Access are also backward, effectively forcing each patient to “audition” for access to experimental therapies on a one-by-one basis in a “wild West” environment where every patient must fight for themselves.

PPA, however, in rare, terminal cases where other clinical therapies are not available, allows for all patients in a particular disease class to access conditionally approved therapies, thus avoiding delays or inevitable inequities when each patient has to appeal separately. In this way, PPA approaches care both in an equitable manner, no matter where the patient seeks treatment, and in an efficient manner.

Second, PPA shares data by using third-party registries that can better inform the FDA, the drug companies, and the medical community as to responses—both to improve safety and develop key data for potential therapeutic reviews later on.

Finally, PPA includes insurance coverage. Right To Try largely failed because of the lack of insurance coverage. Certainly, the provision of insurance coverage may lead insurance companies to request discounts on drugs from pharmaceutical companies; however, the upside is saved lives as PPA provides a faster route for these drugs to reach patients facing terminal and rare diseases.

Views expressed in this article are opinions of the author and do not necessarily reflect the views of The Epoch Times.