The Musella Foundation is a nonprofit charity founded by Al Musella that funds potential breakthrough brain tumor research and helps patients emotionally and financially. The Foundation also has a brain tumor patient clinical database allowing medical experts to pinpoint the best treatment attuned to a given patient’s unique health condition. Invariably, the best treatment is a combination therapy involving a not-yet-approved drug. Therein lies a problem and an opportunity.
In a recent conversation, Al noted that every day he receives at least one phone call from a parent who lost a child after being unable to access a not-yet-approved drug. Imagine if you were that parent on such a call. This humanitarian tragedy can be fixed by passing the Promising Pathway Act, a bipartisan bill sponsored by Senators Mike Braun (R-IN) and Kirsten Gillibrand (D-NY) that is currently in committee hearings in the Senate.
The Promising Pathway Act (PPA) continues the spirit of Operation Warp Speed that sped so many COVID treatments and vaccines to market. PPA will enable patients suffering from rare life-threatening diseases to make informed decisions (advised by their doctors) about using drugs that are conditionally approved up to five to seven years earlier than would be the case for drugs going through the full, conventional FDA approval process. To receive conditional approval, a drug must have successfully passed Phase I safety testing and demonstrated effectiveness via a completed or ongoing Phase II test. For a terminal pediatric rare disease, effectiveness can be shown via animal model testing.
With ongoing satisfactory safety and efficacy demonstrated with real world data (as opposed to randomized control trials), the conditional status could be renewed every two years for a maximum of eight years. If the real-world data is particularly compelling, then the FDA could grant full approval—a significant incentive for drug developers. The proposed PPA timeline is years ahead of the normal time for FDA approval and at sharply lower regulatory costs thereby enabling a significant reduction in drug prices. Importantly, the Promising Pathway Act requires insurance providers to treat conditional approval drugs the same as conventional FDA-approved drugs.
Treatment results along with patients’ health condition, genetic data, and biomarkers would be recorded in a data registry accessible by doctors and medical researchers. The data registry is designed to facilitate a dynamic, self-adjusting system. Improved treatment results versus conventional FDA-approved drugs leads to increased usage and vice versa. The build-up of real-world data enables identification of subsets of patients (based on genetic data and biomarkers) who do exceptionally well or poorly. This treasure trove of data would be made available quickly and better equip doctors and patients to make decisions that may be life-changing or even life-saving. Moreover, the availability of this unique data would expedite significant insights for the biopharmaceutical industry leading to improved R&D allocations.
The humanitarian benefit of thousands of lives saved is absolutely compelling. There are also compelling economic reasons to support this bill. While we all want better drugs, sooner, at lower cost, a myopic view is that price controls are the economic lever to pull. That is a recipe for directing resources away from innovation, including venture capital for startup biotech firms. In contrast, a road that leads to an environment of fast-paced learning from real world data, reduced regulatory costs, and heightened competition would be transformative.
The Promising Pathway Act would provide uniquely useful feedback about such an environment in which patients make decisions based on their unique health conditions and unique risk preferences—information that is not available to the FDA. This Act could save many lives, including children with rare brain diseases.
