By Richard Burr, October 17, 2024 | From: RealClearHealth

As attention remains focused on the upcoming election, Congress must not lose sight of its responsibility to hold the Food and Drug Administration (FDA) accountable for the commitments made under the most recent Prescription Drug User Fee Agreements (PDUFA). Over time, PDUFA has been modernized to build efficiencies and greater predictability into the review process to continue providing patients faster access to new, life-saving treatments while upholding FDA’s gold standard. As the next round of negotiations for the prescription drug user fees nears, it is more important than ever to ensure the agency upholds its current obligations before setting new ones.

For nearly three decades in Congress, and having served as the ranking member of the Senate HELP Committee during the last round of negotiations, I’ve closely monitored FDA’s commitments as part of the PDUFA program. I’ve witnessed the huge increase in resources the agency consistently demanded to deliver on its promises for patient communities. The question today, as it has always been, is whether the significant increase in resources helps FDA and the biopharmaceutical industry deliver medical innovations and scientific advances more quickly to patients.

The PDUFA program was created in 1992 to speed the review of medicines at an under-resourced FDA. Each 5-year agreement includes a set of performance requirements to which FDA agrees to adhere in exchange for industry resources that supplement congressional funding. The additional resources PDUFA provided FDA over the last three decades have led to better agency performance. But these topline performance numbers obscure the more recent concerning trends in agency performance resulting in negative consequences on drug development.

Despite dedicated funding to certain areas such as innovative clinical trial design, model informed drug development, and use of real-world evidence to support regulatory and application-based decision making, there has been limited progress. Yet, the PDUFA program has continued to expand to include funding improvements to FDA’s information technology systems, regulatory science initiatives, and facility maintenance and upgrades. While resources are agreed to under the PDUFA negotiations, Congress has a role to ensure that the agreements fund activities in the best interest of patients.

As part of prior negotiations, FDA and industry agreed the goal is to minimize the number of review cycles necessary to approve novel drugs. Fewer review cycles mean less time patients wait to access new therapies. Yet, in recent years, we have seen an increase in the number of cycles it takes FDA to review drug applications, with only 52% of standard review drugs receiving approval within one review cycle, down from 67% in 2019. 

In addition, FDA has increasingly been issuing complete response letters (CRL) to drug developers, signaling that a drug application cannot be approved in its current form. A CRL halts FDA’s review process, requiring drug developers to resubmit an application for review after addressing any deficiencies raised. In 2023, 23% of drug developers received CRLs, up from 12% in 2018. Many of these letters stemmed from manufacturing and quality issues, which may have been resolved during the first review cycle through better communication and transparency between FDA and industry. 

FDA also continues to fall short on its commitment to timely meetings with drug developers—only meeting 5 of 14 meeting goals in 2022, and agency reports indicate that it will only meet 9 of 18 meeting goals for 2023. These meetings occur before and during the drug review cycle and are a fundamental component of facilitating timely resolution of issues, helping reduce the number of review cycles, and ensuring applications meet FDA expectations.  

Maintaining American competitiveness is integral to providing patients access to novel medical treatments; the PDUFA commitments FDA agrees to are critical to ensuring a predictable regulatory framework that provides patients with swift access to these safe and effective treatments. However, the recent trends suggesting that FDA is not consistently meeting its PDUFA commitments jeopardizes that leadership. In fact, the number of novel drugs that receive approval first in the U.S. before any other country is declining, dropping from 86% in 2016 to just 64% in 2023.  The upcoming round of PDUFA negotiations would benefit from a “back-to-basics” approach to refocus on the original intent of PDUFA – ensuring a predictable regulatory framework that helps speed patient access to safe and effective medicines.