• Why should patients and doctors care about the FDA User Fee bill, clinical trial diversity, and accelerated approval? 

Every five years, Congress reauthorizes an FDA User Fee bill that allows the agency to receive payments in the form of “user fees” from drug and medical device manufacturers. These fees provide support in the form of resources and staffing for the FDA to continue its important work of reviewing the safety and efficacy of medical products. Without the reauthorization of this program through the FDA User Fee bill, FDA would not be able to ensure the timely review and availability of new treatments for patients, including for current public health emergencies such as COVID-19 and monkeypox.

Unlike previous FDA User Fee bills, two important patient-centered provisions have been proposed to be included as part of the reauthorizing legislation. One such provision would ensure that clinical trials supporting FDA approval test new drugs and devices are tested in patients that reflect those that would ultimately be prescribed the treatment. This would allow doctors to have confidence that FDA-approved drugs and devices have been proven to be effective and safe in their specific patient population. For years, FDA has encouraged clinical trial sponsors to increase diversity in clinical trials, but these efforts have not been successful in moving the needle, leaving both doctors and patients in the dark, wondering if FDA-approved treatments would work for them.

Another critical provision that is included in the FDA User Fee bill is reforming the accelerated approval pathway. Under accelerated approval, drug manufacturers can get their new drugs approved by the FDA based on a surrogate endpoint such as a blood test or change in imaging that is thought to be reasonably predictive of how patients feel, function, or survive. In exchange for faster approval of their drugs based on an easier-to-obtain surrogate endpoint, manufacturers are required to complete a postapproval study to confirm that the drug truly works in patients and shows a clinical benefit. Unfortunately, more often than not, these studies are delayed or not even started, leaving doctors and patients in the dark about whether these expensive, new drugs are truly effective. Even when these required confirmatory trials show no clinical benefit from the drug, FDA often cannot withdraw the drug from the market promptly due to a complicated, prolonged process. This means that drugs with no proven benefit continue to be administered to patients, causing them potential medical and financial harm.

In this year’s reauthorization of the FDA User Fee bill, there is an opportunity to ensure that public health safeguards to increase clinical trial diversity and improve the accelerated approval program are included. Together, they would provide much-needed reassurance to patients and their doctors that FDA-approved drugs are truly effective and safe across diverse populations.

• Won’t further FDA oversight of clinical trial diversity just add an undue burden on pharmaceutical and medical device companies?

No. FDA has already issued guidance earlier this year recommending Race and Ethnicity Diversity Plans be submitted by drug and device manufacturers as they develop their products. The provisions within the Food and Drug Amendments Act of 2022 (H.R. 7667), which passed through the U.S. House of Representatives would similarly require clinical trial sponsors to submit Diversity Action Plans to the FDA during the drug or device development process. Additionally, under current law, drug and device manufacturers have to submit their study protocols including their enrollment goals upon registering their clinical trials and before enrollment. Thus, requiring clinical trial sponsors to also submit information around enrollment benchmarks for diversity and how they plan to achieve that goal would be similar to current statutory requirements. Moreover, submitting these plans can actually help reduce the burden for pharmaceutical and medical device companies as the FDA can provide feedback and expert insight on how companies can achieve these diversity goals. 

Finally, payers including Medicare and Medicaid are increasingly requiring manufacturers to conduct additional studies in diverse populations if they fail to do so before FDA approval as a condition of coverage. Having manufacturers take proactive steps, before FDA approval, to enroll diverse patients in pivotal clinical trials will not only benefit patients and doctors, but also manufacturers in being more likely to receive broader coverage without additional, costly requirements such as having to perform additional studies.

• Will accelerated approval reforms reduce the amount of innovation in the market and decrease patient access to therapies?

No. The reforms proposed as part of the FDA User Fee reauthorization bill will still allow manufacturers to seek accelerated approval and obtain faster access to the market based on surrogate endpoints. Thus, patients will continue to receive timely access to new therapies through the accelerated approval pathway. The reforms instead ensure that manufacturers uphold their end of the bargain in completing required, confirmatory trials in a timely manner. Moreover, in being able to know whether a drug is truly meaningfully effective for patients more quickly, innovation will be improved and more efficient. 

Instead of patients and insurers including Medicare and Medicaid spending billions of dollars on unproven drugs, having prompt access to the results of these required confirmatory studies would mean more efficient withdrawal of unproven drugs from the market by the FDA. This would also send a signal to the biomedical research system around what drugs to pursue or not through the accelerated approval pathway. Furthermore, the reforms also include further FDA oversight over the quality of the confirmatory studies. This is critical as many of the required confirmatory trials continue to use surrogate endpoints — and often, the same surrogate endpoint that was used to obtain accelerated approval, making it unclear even after these postapproval trials are completed, if the drug’s clinical benefit had been proven. Thus, further FDA review of the confirmatory studies would allow for these to become more robust to more definitively demonstrate clinical benefit. This would ultimately result in greater access to more effective treatments for more patients! 

• What would it mean for patients and doctors if these patient-centered provisions aren’t included in the final bill?

Ahead of each FDA User Fee reauthorization cycle, FDA negotiates with industry sponsors what precisely the user fees can be used for. Over time, these negotiations have led the FDA to adopt more expedited review pathways where the agency reviews and approves new drugs and devices over a shorter time period. This has led to an increasing amount of uncertainty for patients and doctors over whether new treatments approved by the FDA under these pathways are truly effective and safe. Moreover, with each User Fee bill cycle, pharmaceutical and medical device companies have also negotiated increased access to FDA officials ahead of product approval, raising questions around the independence of the agency and integrity of the review process. These negotiations lead to a “Commitment Letter” in which FDA details what has ultimately been agreed to with industry and forms the base of the User Fee bills drafted by Congress. Typically, Congress also includes riders or additional provisions, often catering to industry lobbyists including regulatory incentives such as extended monopoly price protections or exclusivity periods for certain types of products or even further lowering regulatory standards for approval. The FDA User Fee reauthorization process is a window occurring only every 5 years where Congress can mandate changes and oversight to correct these concerns around safety, transparency, and access.

For the first time, in the wake of recent controversial FDA approvals that raised alarms for patients, doctors, and payers, Congress has included patient-centered riders to the House passed FDA User Fee bill including provisions that would increase clinical trial diversity and reform the accelerated approval pathway. Without such additions to the final FDA User Fee reauthorization package, the agency will have its hands tied to what had been negotiated with industry sponsors as part of the Commitment Letter. It would also signal that instead of fixing the problems that led to recent troubling approvals the FDA would continue to approve new drugs and devices with a focus only on speed, but with little attention to their quality in terms of ensuring that they work and are safe for patients. Abandoning such critical provisions would be a missed opportunity for Congress to provide FDA the authority to carry out its primary responsibility of protecting the nation’s public health.