US FDA

The outgoing FDA commissioner, who placed much of the blame for tough calls on small companies that conduct inadequate clinical programs, defended his policy to not overrule center directors, but added that he did not entirely exclude himself from some of the agency's most controversial drug reviews.

During a virtual appearance at a CERSI meeting two days after announcing retirement plans, the CDER director also said she wished agency reviews of unapproved drugs could be published.

Industry should take a long-term view on policy issues, the former Republican legislator told biopharma execs at the J.P. Morgan Healthcare Conference.

Former FDA commissioners Mark McClellan and Scott Gottlieb, former acting commissioner Janet Woodcock and current commissioner Robert Califf offered advice on successfully implementing reforms and preventing a mass exodus of FDA employees as inklings emerge that the Trump team is already engaged on this front.

Cavazzoni, who led the drugs center four-and-a-half years, decided on her own to retire at this time, sources said. The decision may have been a preemptive move knowing her time as head of CDER would have been limited, or at the very least highly challenging, under the new administration.

The draft guidance clarifies that sex and gender are not interchangeable, but could be vulnerable in the Trump Administration as Republicans have argued against transgender medical care and that sex and gender must be congruent.

Pink Sheet editors discuss Center for Drug Evaluation and Research Director Patrizia Cavazzoni’s surprising retirement announcement, the importance of the large bolus of guidance documents that the FDA released 6 January, and the FDA’s decision to continue reviewing and granting rare pediatric disease designations even though the program lapsed.

6 January 2025 will be remembered in Washington, DC, as the date Congress certified Donald Trump’s second presidential election victory and a snowstorm shut down most of the city, but for FDA watchers it will be the day the agency released more than two dozen new draft and final guidances.

The agency’s actions may signal its optimism about near-term reauthorization despite the PRV program and other bills aimed at tackling rare and childhood diseases not making the December 2024 government spending bill.

Retiring CDER Deputy Director Douglas Throckmorton spoke to the Pink Sheet ahead of his early 2025 retirement about regulatory flexibility, AI and other topics.

The FDA will stay open, but its rare disease priority review voucher program will wind down after not being renewed on 20 December. PBM reform and other industry priorities also were nixed at the last minute in an effort to avoid a government shutdown.

Robert Temple, the US FDA’s most experienced drug regulator, is currently serving an emeritus role at the agency. The immediate impact of his departure will be mostly symbolic, but still unsettling.

Namandje Bumpus' departure means there is no obvious, internal candidate to serve as acting commissioner when President-elect Donald Trump takes office in January and the Senate works to confirm a new leader.

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

A safety signal may be platform or even Moderna-specific, the Vaccines and Related Biological Products Advisory Committee said.

Former Sen. Richard Burr suggested to the Pink Sheet that Trump’s HHS leadership picks will have less ability to enact their agendas than people expect.

A new congressional report on the public health response to the COVID-19 pandemic underscores that the FDA and its top managers may be in the hot seat.

Pink Sheet reporter and editors discuss potential threats to the COVID-19 vaccines under the Trump Administration, the DOGE commission’s search for spending cuts and its impact on the FDA, as well as a new 340B rebate pricing model that some pharma companies are implementing to contain the program’s costs.

Pulling a product that was granted an emergency use authorization is easier than pulling a fully licensed vaccine, which could place Pfizer and Moderna’s COVID-19 vaccine pediatric indications at risk, along with Novavax’s vaccine in all age groups.

The pharma industry arguably suffered none of the possible downside risks during the first Trump Administration, but its relationship with Biden could not have been much worse. And while sponsors may hope a Trump return could improve their fortunes, having Robert F. Kennedy Jr. supervising the FDA is likely not the prescription they prefer.