In a letter to Congress with 70 other health organizations, the Colorectal Cancer Alliance is urging lawmakers to swiftly reauthorize the Prescription Drug User Fee Act (PDUFA) to include new provisions that will enhance access to and improve diversity in clinical trials.
PDUFA, one of our governments more awkward acronyms, pronounced “PA-DUE-FA,” was first passed in 1992. At the time there was great dissatisfaction with the amount of time it took the Food and Drug Administration (FDA) to review and approve new prescription drugs. Patients waited too long for new treatments and industry lost money while the delay ate into the time frame the drug had market exclusivity. FDA needed more staff and resources to accelerate the process without sacrificing quality, and PDUFA enables the FDA to charge industry a fee for the review and approval process. In exchange for receiving the fee, FDA has certain performance standards that must be met.
For PDUFA to continue, it must be reauthorized every five years, and it is set to expire on October 1 of this year. The reauthorization provides the opportunity to include new provisions that will make it easier for patients to participate in clinical trials, as well as increase diversity in the study groups. Doing so can improve both the speed and quality of the trial. At this time the relevant committees in the Senate and House are seeking agreement on the final version of the reauthorization bill. There are three key provisions the Alliance is urging these committees to include:
Decentralized Trials
Clinical trials tend to be conducted by academic medical centers concentrated in just a few large cities, while patients live all around the country. To participate in a clinical trial patients often must travel great distances and many simply are not able to do so. This makes it harder for sponsors to recruit patients and delays or, in some cases, halts the clinical trial. During the pandemic, some flexibilities were allowed as travel was limited or prohibited. These included sending drugs to the patient’s home, allowing tests at local sites, and monitoring through telemedicine. Congress can make this flexibility permanent by directing the FDA to establish guidance for decentralized trials.
Financial Barriers
Participating in a clinical trial usually involves significant out of pocket costs for the patients – time away from work, parking, and other travel costs. Even with decentralized trials, patients may require smart devices, internet connectivity, or other technology. Companies are generally willing to cover these costs, but they do not because they are concerned it may run afoul of kickback laws. Congress should establish safe harbors for companies to cover patient out-of-pocket costs without fear of legal liability.
Diversity Planning
Ensuring trial participants reflect the diversity of the patient population improves the scientific results and can make a drug safer and more effective. Congress should require that trial sponsors have specific plans to enhance diversity in the clinical trial population.
The Colorectal Cancer Alliance joined with the American Cancer Society and 70 other health organizations in a letter to the Senate and House committees working on PDUFA reauthorization urging that these provisions be included. We will keep you apprised of progress on PDUFA, as passage by October 1 is essential to keep FDA’s review and approval process timely.
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