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House E&C hones in on accelerated approvals

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May 22, 2024
Rep. Cathy McMorris Rodgers

Rodgers at an Energy and Commerce hearing. Photo: Chip Somodevilla/Getty Images

The FDA's drug approvals and oversight of lab-developed tests are again emerging as hot topics as House Republicans crank up oversight of the agency and the FY25 appropriations process begins to play out.

Why it matters: GOP lawmakers want assurances that drugs going through the agency's accelerated approval process can qualify for Medicare coverage.

  • There's also lingering tension over the FDA's recent decision to exercise its authority and treat in-vitro diagnostic tests as medical devices.

Driving the news: At a House Energy and Commerce health subcommittee hearing today, Chair Cathy McMorris Rodgers expressed concern over the failure of CMS to cover drugs that are approved through the accelerated approval process.

  • Rodgers emphasized that the expedited pathway "should be leveraged now more than ever."
  • She pointed to HHS Secretary Xavier Becerra's statement when testifying at another recent hearing that some drugs cleared through the pathway didn't show effectiveness, and questioned whether that is undermining FDA's credibility.
  • She then pressed the top FDA officials to say whether biologics approved under accelerated approval had the full FDA gold standard of approval.
  • "When we approve something through accelerated approval, it meets our approval standard of which is substantial evidence of effectiveness," said Peter Marks, director of the Center for Biologics Evaluation and Research. "So we would expect it to be safe and have an effect in people."
  • "When we use accelerated approval, we make a determination," said Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research. "We stand by our standard."

Flashback: The 2022 omnibus included limited reforms to the accelerated approval pathway allowing the FDA to more easily pull drugs from the market if there is poor efficacy data and to require certain types of trial design and data as a condition for approval.

  • Rodgers also took the agency to task for unilaterally deciding to regulate lab-developed tests instead of wait for authorizing legislation.
  • Rodgers has pressed for the FDA's recently finalized rule on lab-developed tests to be rescinded, saying Congress needs to pass a law to give FDA such authority and that acting alone would quash innovation.
  • "The agency has been failing to adequately accommodate in-person meetings and respond to outreach related to major clinical and scientific development decisions in a timely manner," she said.
  • "This is particularly concerning as the FDA has unilaterally decided it can regulate, by its own estimate, 80,000 tests under the laboratory developed tests final rule."
  • FDA officials responded in the affirmative that they would appreciate a modern framework like the VALID Act, but had to act now in the face of "problematic LDTs."

The other side: Meanwhile, Ranking Member Frank Pallone emphasized the issue of drug shortages, and how Congress could help by with the issue by giving the FDA more authority to prevent them.

  • Particularly he asked if companies should be required to report drug shortages driven by demand — a situation that's received more attention as supplies of weight-loss medicines like Wegovy and Mounjaro have tightened.
  • Cavazzoni responded that would be "incredibly helpful" to because they would be able to intervene before the shortage instead of going into mitigation mode.
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