User fee amendments (UFAs) are intended to expedite and modernize the landscape of drug approvals and drug oversight regulations. The UFAs must be renewed every 5 years, and often serve as a vehicle for other policies to move through Congress as part of this “must-pass” legislation. The first Congressional hearings for user fee reauthorization begin on February 3, 2022, and current legislative authority for UFAs will expire in September 2022.

Background

The Prescription Drug User Fee Act (PDUFA) is utilized by Congress to modernize regulatory expectations and get safe and effective products on the market sooner. PDUFA I (P.L. 102-571) was first passed by Congress in 1992 to provide supplemental revenue in the form of sponsor-supplied user fees for the US Food and Drug Administration (FDA) to use on oversight and review efforts. In exchange, PDUFA required the FDA to standardize and expedite review of product applications to increase predictability for manufacturers. Prior to 1992, the length of time between manufacturer product filing and FDA approval decisions was substantially longer and more unpredictable than it is today, attributed by the FDA to a lack of adequate product review staff.

PDUFA I was created to automatically sunset after 5 years, with the idea that Congress could review its effectiveness before deciding on re-authorization. Three decades later, the reauthorization process remains and has expanded to other user fee programs.

* OMUFA was first signed into law as part of the Coronavirus Aid, Relief, and Economic Security Act in March 2020 and is not up for reauthorization until 2025/

The UFAs have ushered in significant changes to the regulatory oversight of medical products in the US, and their reauthorization has become a vehicle for reforming product development. The process for reauthorization typically begins about 2 years prior to the sunset of the current term.  The FDA, industry, and stakeholders meet periodically to discuss successes and proposed revisions to the program, which culminates in the FDA release of a final commitment letter that heads to Congress. Using PDUFA as an example, we can see how the program has led to major changes that contribute to the current fabric of manufacturer-FDA collaboration we see today:

• Introduced the “fast track” designation (PDUFA II)
• Required the FDA to negotiate with industry on how to improve the program prior to publishing the commitment letter (PDUFA III)
• Authorized pilot programs for “rolling” review of new drug applications (PDUFA III)
• Provided risk evaluation and mitigation strategies authority to the FDA (PDUFA IV)
• Authorized the Sentinel Initiative to expand collection of safety data to provide stronger oversight of products on market (PDUFA IV)
• Advanced the use of patient-reported outcomes in drug review (PDUFA V)
• Initiated the Real-World Evidence (RWE) Program (PDUFA VI)

FY 2023–2027 UFA Reauthorization

The FDA released final commitment letters in 2021 for 3 of the 4 UFAs up for reauthorization, which Congress will need to reauthorize by September 30, 2022. The finalized commitment letters focus on themes such as expediting development of products for rare diseases, advancing the use of RWE, and increasing communication between the FDA and sponsors. Highlights of the commitments include:

PDUFA VII (Commitment Letter)

• RWE Advancement: Expands the use of RWE-based strategies in support of new labeling claims, post-approval study requirements, and analytic strategies for safety monitoring systems
• Pilot Program for Split Real-Time Application Review: Aims to shorten the time from application submission to FDA decision through a rolling review process for therapies that address an unmet medical need
• Drug Development for Rare Diseases: Provides increased FDA perspective on novel approaches to drug development (e.g., adaptive study designs) and will initiate a pilot program to advance the use of endpoints in rare diseases

GDUFA III (Commitment Letter)

• Communication Enhancements: Provides more opportunities for sponsors to meet with the FDA to clarify application deficiencies (e.g., Enhanced Mid-Cycle Review Meeting), which helps minimize the number of assessment cycles required for approval, speeding access to generics
• Original Abbreviated New Drug Application (ANDA) Assessment: Provides FDA consultation on the proposed labeling of a generic drug to help ensure that it is appropriately aligned with its reference listed drug, reducing risks of patent infringement that prevents generic products from entering the market
• Pre-ANDA Program: Continues focus on enhancing the development of complex generic products through FDA guidance and consultation

BsUFA III (Commitment Letter)

• Interchangeable Biologics Labeling and Promotion: Commits to publishing guidance on topics covering biosimilar biological device combination products and review of interchangeable biosimilars to provide greater clarity on data and information expectations for sponsors
• Regulatory Science Program for Interchangeable Products: Enhances expectations for interchangeable products through a demonstration project that will focus on evaluating the data and information needed, including through RWE, to meet safety standards for determining interchangeability
• Regulatory Science Program for Biosimilars: Focuses on the use of advanced analytical and pharmacological assessments that can streamline biosimilar product development

MDUFA V

The FDA did not meet the January 15, 2022, deadline to send the finalized commitment letter for the MDUFA V to Congress, but missing this deadline in the past has not stalled the package. Industry negotiations on user fee goals for MDUFA V have been underway since February 2021 between FDA, industry, and associated stakeholders. Potential enhancement provisions for reauthorization include:

• Total Product Lifecycle Advisory Program: Introduced to foster earlier sponsor engagement and help facilitate continued innovation in the medical device space

The Role of Congress

Once the commitment letters are finalized, Congress will provide its perspective on the proposed policies and programs. It will be important to monitor these discussions, particularly with upcoming mid-term elections in 2022 and the ensuing transition period in Congress, as well as other priorities on which the committees with jurisdiction over the UFAs are focused (e.g., Cures 2.0, Prepare for and Respond to Existing Viruses, Emerging New Threats, and Pandemics Act [PREVENT Pandemics Act]).

These priorities could be attached to the UFAs if there is sufficient political support. As evidenced, the agency and industry have used the UFAs to include related healthcare provisions that are exclusive of user fees. In Congress and the administration, must-pass UFA legislation has historically been incorporated into larger bills (e.g., PDUFA V enacted as Title I of the FDA Safety and Innovation Act), and precedent exists where Congress has included provisions as legislation “riders.”

Inclusion of additional FDA-related bill text for this UFA reauthorization is thus not out of the question, as there is considerable overlap between other policies and the UFA commitment letters on topics ranging from clinical trial diversity to the use of RWE in regulatory decision making. While provisions of the UFAs have been negotiated since before the public health emergency, the urgency and importance assigned to improving certain aspects of the healthcare system following COVID-19 will add another layer of complexity to the interplay between the must-pass UFA packages and these other priorities.

As a result, stakeholders should closely follow developments on advancement of the UFA packages, in addition to parallel discussions of related legislation.

Avalere’s Perspective

Avalere has industry-leading knowledge of US healthcare policy that intersects with regulatory operations at the FDA, combining our dedicated policy and regulatory strategy teams to provide updates on cross-cutting actions and implications that affect the broader healthcare enterprise. We monitor activity with potential to shape the outlook of medical product development, review, and access from both an agency and Congressional perspective. If you have questions or would like to strategize on how provisions may impact your company and ways to plan for upcoming decisions, please reach out to schedule a call with us.