Policy
A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations.
FEATURED STORIES
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.
Former FDA, CDC and NIH leaders convene at the BIO International Convention to discuss the dismantling of the Department of Health and Human Services under the Trump administration—and where we go from here.
DemeRx is launching the first U.S. clinical trial of an ibogaine-derived drug candidate, marking a pivotal moment for a controversial psychedelic long sidelined by safety concerns.
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A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
Having stuck with local production in the years when offshoring to lower-cost locations was in fashion, Aquestive’s CEO is well placed to explain what the recent reshoring drive means for manufacturers.
The U.S. government has argued that it pays too much of biopharma R&D costs, demanding that other high-income countries contribute fairly to global innovation efforts.
Weeks after Boehringer Ingelheim and Eli Lilly retracted billions of dollar in German commitments, the nation’s government is reportedly changing a contentious element of its planned healthcare reforms.
The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation.
A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.
Robert F. Kennedy Jr.’s health department has consistently touted radical transparency as being key to its mission. Recent instances—the FDA’s decision not to disclose the recipients of three Commissioner’s National Priority Vouchers and FDA and CDC choices not to publish vaccine-related papers—call this intent into question.
Of the 17 companies that were implored by the White House last July to apply Most Favored Nation pricing to their drugs, Regeneron is the last to agree—the same day the FDA greenlit its gene therapy for hearing loss in kids.
The FDA in July 2025 made publicly available over 200 complete response letters—an initiative that the investment community sees as “unanimously positive,” analysts told BioSpace.
Two of the biggest insurance providers have expressed reluctance to participate in the government’s BALANCE program that would have made GLP-1 drugs more affordable to patients.