Government
While Congress is renewing the priority review voucher program for rare pediatric diseases, the FDA should be required to keep public records of the passes changing hands, too.
Analysts at Jefferies see Makary as a positive for the rare disease space, given his support for accelerated approvals and openness to “customizing regulatory pathways for rare diseases.”
The Senate hearing for FDA Commissioner nominee Marty Makary comes after President Trump’s NIH pick, Jay Bhattacharya, was grilled by the legislative body on Wednesday.
The vaccine space has been battered by strong headwinds in recent weeks, including high-level disruptions to FDA and CDC advisory committee meetings.
A BioSpace analysis of all 80 priority review vouchers that have been handed out across the three FDA programs that offer them found that 2024 was the busiest year yet. Companies have disclosed spending $513 million on vouchers that were earned in 2024 so far.
The last few years have been tough for the insulin market, with recent policies and high-level pressure forcing companies to lower drug prices.
Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.
Last week, Eli Lilly also responded to the President’s tariff warnings by investing $27 billion to construct four manufacturing facilities across the U.S. in five years.
In the five weeks since Donald Trump returned as U.S. president, the FDA, NIH and CDC have been thrown into disarray, with meetings regarding vaccines and rare diseases canceled or indefinitely postponed—all without a clear reason why.
The move comes after President Donald Trump warned Big Pharma leaders that he would impose tariffs on them if they refuse to reshore their manufacturing operations.
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