Health policy experts call for stronger accelerated approval reforms

Regulatory NewsRegulatory News | 06 July 2022 |  By 

The US Congress should take concerns raised by the Food and Drug Administration (FDA) seriously and give the agency more authority around accelerated approvals, including the power to expedite the withdrawal of drugs from the market, according to a perspective article from three experts on drug regulation and health policy.
The perspective, written by Rachel E. Sachs, JD, MPH, of Washington University in St. Louis, Julie M. Donohue, PhD, of the University of Pittsburg, and Stacie B. Dusetzina, PhD, of Vanderbilt University, was published in the New England Journal of Medicine on 6 July 2022. It comes as US lawmakers consider accelerated approval pathway reforms as part of the “must-pass” reauthorization of the agency’s medical product user fee programs.
The House passed its version of the legislation in June (H.R. 7667) and the Senate’s version cleared the Committee on Health, Education, Labor & Pensions in June (S. 4348). Sachs and her coauthors said those bills include “weaker versions” of the changes proposed by House Energy and Commerce Chairman Frank Pallone (D-NJ) earlier in the year. Rep. Pallone’s bill, H.R. 6963, would allow for expiration of accelerated approvals one year after the target date to complete a confirmatory trial, or five years after the drug’s accelerated approval without a confirmed benefit. It would also expand FDA’s authority, allowing it to require that confirmatory trials are underway before accelerated approval is granted (RELATED: Legislation would set expiration for FDA’s accelerated approvals, Regulatory Focus 9 March 2022).
The accelerated approval provisions in the user fee reauthorization bills leave out the automatic withdrawal provision proposed by Rep. Pallone and would require FDA to hold an advisory committee meeting at the sponsor’s request and provide opportunities for public notice and comment proceedings before withdrawing accelerated approval. “Specifying these procedural requirements in the statue would limit the FDA’s existing discretion to tailor its withdrawal procedures to the circumstances of a particular case,” Sachs and her coauthors wrote.
While the House and Senate bills both include provisions to speed the completion of confirmatory trials, they would allow those trials to be supported by real-world evidence. Sachs and colleagues said that has the potential to “limit their rigor.”
The perspective authors also point out that changes to the accelerated approval pathway have implications in the drug pricing debate. “How much – if anything – should insurers (including public programs) be required to pay for approved products for which confirmatory trials either haven’t been completed or haven’t demonstrated clinical benefits?” they wrote.
Medicaid programs are required to cover “essentially all FDA-approved drugs,” the authors wrote. Medicare can limit coverage due to a lack of clinical efficacy, as the agency recently did for the Alzheimer’s drug aducanumab, but that is rarely done. “Those coverage requirements impose substantial costs on state and federal budgets,” Sachs and colleagues wrote. “Removing products that haven’t been shown to have clinical benefits from the market might therefore provide relief for resource-constrained state Medicaid programs.”



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