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Medical device user-fees agreement, Aduhelm approval and access among issues in crosshairs.
FDA officials have faced a range of tough questions on Capitol Hill in recent weeks as part of the process for gaining timely agreement on legislation to reauthorize user fees for the agency’s medical product regulatory programs. Last week, members of the Senate Health, Education, Labor & Pensions (HELP) Committee quizzed Center directors for drugs, biologics and medical devices on multiple contentious issues—expedited approvals, vaccines for young children, orphan drug exclusivity, access to medical abortions, conflicts of interest, and hiring initiatives, to name a few.
Less was said by most members of the HELP Committee about the specific provisions in the new user fee packages for drugs (PDUFA), generic drugs (GDUFA), and biosimilars (BsUFA), which were summarized in written testimony by Center for Drug Evaluation and Research (CDER) Director Patrizia Cavazzoni and Center for Biologics Evaluation and Research (CBER) Director Peter Marks.1 A delay in finalizing the agreement on medical device user fees (MDUFA), however, drew sharp criticism from the committee’s ranking Republican, Sen. Richard Burr (R-NC). And Burr questioned whether added fee payments really will enable these key FDA operations to actually reduce sizeable staff vacancies, as new hires have failed to offset even larger attrition rates at the agency.
Several senators challenged FDA’s approval of Aduhelm to treat Alzheimer’s disease and the equally controversial decision by the Centers for Medicare and Medicaid Services (CMS) to limit access to the drug to those Medicare beneficiaries enrolled in postapproval clinical trials. Cavazzoni was adamant about the validity of FDA’s approval of the drug, while also seeking Congressional support for legislation enabling FDA to require manufacturers to start confirmatory trials at time of drug approval, to meet agreed-on timelines and milestones for such studies, and for authority to expedite the market withdrawal of drugs that fail to show benefit in postapproval trials.
In response to queries about a recent court decision involving the Orphan Drug Act, Cavazzoni warned that the ruling “will send a chill” into the development of rare disease treatments, especially for children. That discussion involves the finding in the case of Catalyst vs. FDA by the US Court of Appeals in October 2021 that FDA cannot approve a pediatric version of a drug that has obtained orphan drug exclusivity for the adult version. That ruling, said the CDER director, enables a company to develop a drug for a very narrow patient population and then use the exclusivity for the initial approval to block further research on the therapy for other conditions and patients. The controversy may prompt legislative action or end up for review by the Supreme Court.
The increasingly contentious debate over FDA regulation of access to the abortion pill mifepristone also gained a hearing, as Sen. Roger Marshall (R-Kan) warned that patients will be harmed by recent changes in FDA rules that permit access to the drug through telemedicine, as opposed to requiring a visit to a doctor’s office. Marshall cited the potential for severe complications if women take the pills too late in pregnancy, which he said is more likely without an in-office ultrasound examination. Cavazzoni responded that FDA’s “exhaustive review” of the drug indicates that current procedures ensure its safe use, and that an ultrasound examination is not required. Requests for pills to terminate pregnancy have risen recently as states enact increasingly severe limits on abortions, despite parallel efforts to discourage mifepristone use. FDA’s more flexible position on the issue is expected to be a target for legislation backed by anti-abortion groups.
And in response to a query from Committee chair Sen. Patty Murray (D-Wash) about delays in access to vaccines for children under 6, Marks of CBER said that FDA action on such a vaccine was a “top priority,” that the agency was waiting for manufacturers to file EUA applications, and that CBER would discuss such preventives at a public advisory committee meeting; a highly transparent discussion, said Marks, was important for building public trust in the safety and effectiveness of any preventive for very young patients. CBER’s subsequent announcement that such committee review would not occur until June, however, prompted loud protests about FDA delaying access to vaccines important for keeping very young patients healthy.
The tone and scope of the questioning of FDA officials here and at earlier hearings by the House Committee on Energy and Commerce provide a glimpse of what measures Congress may tag on to the user fee reauthorization package. The outcome of months of discussion will emerge more visibly this month as the process moves forward to “mark up” a legislative proposal that includes all the user fee packages and leading add-on measures. All parties would like to see Congress vote on user fee legislation before its annual recess in August, but the schedule is tight, and the scope of pet initiatives expands every day.