
This developing story tracks how the pharmaceutical industry adapted its lobbying strategy to capitalize on increased political influence at the FDA under the Trump administration, while rare disease advocates grew frustrated over inconsistent drug approval decisions. The timeline reveals a collision between industry opportunism, political pressure, and controversial regulatory decisions that sparked widespread concern about the agency's independence.
7 events · 4 days · 12 source articles
Pharmaceutical lobbyists revealed to STAT that they believe approval odds increase if decisions can be framed as wins for the Trump administration. The industry began capitalizing on new avenues for influence opened by the administration's injection of political priorities into the historically independent FDA. Three lobbying firms with close Trump connections—Checkmate Government Relations, Miller Strategies, and Ballard Partners—saw a surge in pharmaceutical clients and spending.
In his first State of the Union address of his second term, President Trump claimed he had brought U.S. prescription drug costs from the highest in the world to the lowest through his 'most-favored nation' policy. This claim came despite surveys showing more than half of Americans say health care has become more unaffordable. Trump urged congressional Republicans to codify the policy.
The FDA rejected an experimental therapy for a rare blood cancer despite internal reviewers previously recommending approval and the drug being on a clear path toward clearance. This surprise rejection marked a significant reversal and raised questions about shifting standards at the agency. The decision became a flashpoint for concerns about inconsistent regulatory decisions under new FDA leadership.
Families dealing with rare diseases voiced frustration over what they perceived as whiplash from shifting and contradictory FDA decisions. Parents like Megan Selser, whose infant son Ben has Hunter Syndrome, had been given hope through new treatments in clinical trials, only to face uncertainty as the FDA's approach to rare disease therapies appeared to change. The community began organizing in response to perceived inconsistencies.
The FDA's rejection of a rare disease cell therapy that had received prior internal support and approval in Europe prompted widespread questions in the biotech industry about whether the agency's public rhetoric matched its regulatory actions under new leadership. The rejection became a reality check on claims that the FDA would accelerate access to innovative treatments.
FDA Commissioner Marty Makary defended the agency's recent rare disease drug rejections in a CNBC interview, appearing to reference the FDA's stance on a UniQure gene therapy for Huntington's disease. He also defended top FDA official Vinay Prasad, who oversees the center responsible for many of the controversial rejections. Makary cited safety concerns about invasive procedures in his defense.
SEC filings revealed that President Trump's heavily promoted 'most-favored nation' drug pricing deals with pharmaceutical companies last three years for at least some of the 16 participating companies. Many details of these agreements had not been previously released by the administration or the companies. The limited duration raised questions about the long-term impact of Trump's signature drug pricing policy.