E&C Health Members Discuss Aduhelm, Pandemics, Rare Diseases in FDA User Fee Reauthorization Hearing

By: | February 7, 2022

On Feb. 3, 2022, the Subcommittee on Health of the House Committee on Energy & Commerce (E&C) convened a hearing on the reauthorization of three Food and Drug Administration (FDA) user fee programs (hearing memo). These include the seventh reauthorization of the Prescription Drug User Fee Act (PDUFA VII), the third reauthorization of the Generic Drug User Fee Amendments (GDUFA III), and the third reauthorization of the Biosimilar User Fee Act (BsUFA III). These agreements authorize the FDA to collect user fees, which supplement appropriated funds to ensure that drugs and biologics are reviewed in a timely manner. The programs are reauthorized every five years with modifications based on negotiated agreements between the FDA and industry representatives. The most recent reauthorizations expire on September 30, 2022. The Subcommittee on Health heard testimony from two panels of witnesses, including representatives of the FDA and industry representatives.

Opening statements emphasized the successes of the user fee programs in enabling the FDA to review and approve medications in a timely manner, giving patients more access to medications while propelling the U.S. to the global forefront of medical innovation. Representatives expressed investment in quickly reauthorizing the user fee agreements prior to the expiration of the current user fee agreements. In his opening statement, Committee Chair Frank Pallone (D-NJ) expressed concern that the FDA and industry leaders have not yet reached an agreement on the medical device user fee program, stating that he and Committee Ranking Member Cathy McMorris Rodgers (R-WA) sent a letter to the FDA on the issue. Ranking Member Rodgers also expressed exasperation with the rumor that the Biden Administration may ask for additional COVID-19 funding, asking how long the Administration plans to be the primary buyer of vaccines.

During the hearing, Rep. Fred Upton (R-MI) commended Senators Richard Burr (R-NC) and Patty Murray (D-WA) for their work on the PREVENT Pandemics Act (see WHG summary) and indicated that he looked forward to working with them. Rep. Upton also commented on the areas of drug development that are inadequate to meet the needs of future pandemics, pointing to a lack of new antibiotics. Rep. Upton noted that Cures 2.0 includes the PASTEUR Act, which encourages the development of antimicrobial drugs. Finally, he spoke to the proposal included in the user fee agreements to advance regulatory use of real-world evidence (RWE). He stated that Cures 2.0 also provides for advancing the use of RWE. Several other representatives indicated interest in advancing antibiotic development and using RWE.

Additional key points from the hearing are as follows:

  • Rare Diseases – Several representatives across the aisle voiced concerns about the availability of drugs for rare diseases. Rep. G. K. Butterfield (D-NC) spoke to the importance of including children in clinical research to develop drugs for cancer and urged the FDA to expand the Rare Disease Endpoint Advancement (RDEA) Pilot Program in PDUFA which would support the FDA in measuring efficacy in rare disease drugs.
  • Accelerated Approval – Many representatives asked questions relevant to accelerated approval of drugs. Republican leaders and members voiced frustration with the Centers of Medicare & Medicaid Services’ (CMS’) proposed national coverage determination (NCD) to restrict coverage of monoclonal antibodies including Aduhelm, which was approved by the FDA through an accelerated process, and asked about the safety standards of the accelerated approval process. Ranking Member Rodgers stated that during the COVID-19 pandemic, the FDA has expedited reviews of pertinent vaccines and treatments while maintaining safety standards. Chair Pallone explored the idea of requiring manufacturers to enroll products in post-approval confirmatory studies prior to FDA approval under the accelerated pathway to expedite lengthy studies.
  • Mandatory RecallRep. Kathy Castor (D-FL) discussed the fact that the FDA lacks the authority to mandate recalls on most drugs and must rely on manufacturers to voluntarily recall harmful or ineffective drugs. Witnesses agreed that this inability is a barrier to ensuring drug safety. Chair Eshoo also agreed and pointed out that the America COMPETES Act contains a provision giving FDA mandatory recall authority and will be up for a vote in the House this week.
  • Clinical Diversity – Some representatives spoke to the importance of ensuring diversity in clinical trials. Republican leadership in the committee focused specifically on the inclusion of individuals with intellectual disabilities and developmental disabilities (ID/DD).
  • Patient-Focused Drug Development – Some representatives emphasized the importance of the patient experience in drug development and approval. Witnesses from the FDA indicated that PDUFA builds on programs integrating patient voices and increases the inclusion of patient experience data and meaningful endpoints.

Additionally, on the day of the hearing, the Assistant Secretary of Planning and Evaluation (ASPE) published a report on the costs of generic drug development and approval. The report describes an analytical framework for examining the expected net present value (ENPV) to a generic drug developer depending on the size of the drug market. The report finds that increasing the rate of drugs that are approved by the FDA in its first application cycle to 66 percent would reduce the time to market by approximately 13 months and reduce expected capitalized costs to the developer by $3.5 million. The report also finds that FDA product-specific guidance (PSG) could save years of development. It notes that under GDUFA, the FDA is committed to issuing PSGs for complex products.

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Billy Wynne