User Fee Negotiations Heat Up Ahead of September 30 Reauthorization Deadline

In the coming months, Congress will vote on a bill that includes the reauthorization of the Prescription Drug User Fee Authorization (PDUFA) Act, which allows FDA to collect fees from companies that produce certain human drug and biological products, including gene and cell therapies. 

Congress is working to blend different versions of the bill that will establish a new user fee framework to ensure FDA can continue to support product sponsors through the drug development process. 

Since the original PDUFA Act was passed in 1992, companies that produce certain drugs are required to submit application fees to FDA to cover, in part, the cost of application review in exchange for predictable timeline goals that FDA must meet. The fees and related performance goals have helped speed up the FDA review process in a safe and timely manner through alignment of resources, increased transparency with the Agency, and additional public input. Over the past 30 years, the program has expanded to cover over 90% of FDAs drug review budget and includes application and program fees.  

PDUFA must be reauthorized every five years to ensure the fee framework and goals keep pace with technology and the needs of industry, FDA, and Congress. The current PDUFA (PDUFA VI) is set to expire on September 30, along with the analogous fee agreements for medical devices (MDUFA), biosimilars (BsUFA), and generic drugs (GDUFA).   

On June 8, the House of Representatives passed their version of the FDA user fee package (HR7667), including PDUFA, with a solid bipartisan vote (392-28). The fiscal year 2023 base fee authorization for prescription drugmakers is $1.15 billion (which will be upwardly adjusted for inflation, workload, and other enhancements). New authorities and limitations on the accelerated approval pathway are among the most impactful provisions of the bill. All future sponsors must submit post-market study plans to FDA before a product can be approved, and FDA may require such a study to be enrolled. There is also a new streamlined withdrawal process for accelerated approval drugs that do not meet post-market study requirements or demonstrate clinical benefit and requirements for product labeling.  

Other provisions included in the House legislation include requirements for sponsors to submit diverse action plans for clinical trials. ASGCT recently responded to the FDA’s draft guidance on Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials. ASGCT has asked the Agency to consider current proposed legislation when preparing revised, final, or additional guidance on diversity to ensure it is aligned with any new statutory provisions. In addition, the House-passed legislation includes provisions that would require the FDA to convene public workshops and issue reports and guidance on issues such as: decentralized clinical trials, rare disease product development, and new pediatric study requirements for molecularly targeted cancer drugs. Several provisions in the House bills, such as those on clinical trial diversity and drug manufacturing platforms, were included in the HELP Committee’s PREVENT Pandemics Act, which advanced through Committee earlier this year.  

Following the House vote, the Senate Health, Education, Labor, and Pensions (HELP) Committee marked up their version of the reauthorization package. The FDA Safety and Landmark Advancements Act (FDASLA) contains many of the same provisions as its House counterpart, including changes to the confirmatory trial requirements for the accelerated approval pathway. Unlike the House bill, FDASLA includes provisions on drug importation, baby formula, and reforms of the diagnostics, cosmetics, and dietary supplements regulatory regimes.   

These differences will require reconciliation between the two chambers. The staff from the Energy and Commerce and HELP committees are meeting in earnest to blend their bills via “pre-conferencing” before legislation is brought to the Senate floor. The unified bill will then move back to the House before moving to the president for signature. While both chambers have indicated a desire to close out this process before Congress leaves town for its annual August recess, the final vote could be held once Congress returns in September.  

Want to learn more? During the ASGCT Policy Summit, attend a panel on PDUFA reauthorization. Check back to the agenda for weekly updates. Registration opens in July.

Ms. Valdez Martínez is ASGCT's Director of Policy and Advocacy and Ms. McCombs is ASGCT's Advocacy Program Specialist.