health-policy3.775 min read
FDA Unveils Plausible Mechanism Framework to Accelerate Tailored Therapies for Ultra-Rare Diseases
2 days ago•Source: fda.gov
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2.095 min•5 days ago
FDA chief vows tighter safety review of antidepressants in pregnancy and RSV protections for infants
In her first staff address, FDA’s top drug regulator Tracy Beth Høeg pledges tougher safety monitoring of antidepressants used in pregnancy and RSV monoclonal antibodies for infants, signaling a broader push to reexamine oversight and encouraging staff to question norms.
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TrumpRx Promises the Cheapest Drugs, Yet Generics Undercut Many Offers
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Don't Copy Denmark: The U.S. Needs Its Own Vaccine Schedule
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FTC Settlement Ushers in Net Pricing Drug Channel
The FTC’s landmark settlement with Express Scripts dismantles the PBM’s current incentives and launches a Net Pricing Drug Channel (NPDC): patient costs will be based on net price, rebates must be passed through to patients, formulary bias toward high-WAC drugs ends, direct-to-consumer pricing via TrumpRx is enabled, manufacturer fees must be delinked from list prices, and retailers will be reimbursed on a cost-plus basis. The Ascent Health Services GPO will move from Switzerland to the US, with a monitor and compliance reports and a deadline of January 1, 2028, signaling sweeping changes for plan sponsors, pharmacies, manufacturers, and PBM competitors.
Senate presses NIH director on vaccines and funding upheaval
During a Senate HELP hearing, NIH Director Jay Bhattacharya faced tough questions on vaccine safety messaging and sweeping funding disruptions that terminated or paused hundreds of grants and trials, including cancer and HIV research. He reaffirmed that vaccines do not cause autism, cited public mistrust as a contributing factor, and said NIH is moving to fill leadership vacancies and restore advisory councils, while avoiding a firm commitment on disruption impact analyses.
Families Rework Immunization Plans Amid Splintered Guidance
With major shifts in federal vaccine guidance—six diseases removed from routine schedules and an advisory panel head suggesting measles and polio vaccines could be optional—parents are scrambling to protect their children, delaying vaccines, switching pediatricians, or vaccinating earlier, even as measles and whooping cough infections rise across the country.
Real-food reform vs. real-world barriers: why RFK Jr.'s diet plan stalls
Vox critiques RFK Jr.’s push to treat food as medicine via an inverted food pyramid, saying his emphasis on personal responsibility overlooks structural barriers that make healthy eating hard, such as food deserts and cost, even as dietary guidelines move toward fewer ultra-processed foods. Experts warn that equating nutrition with medical treatment risks sidelining conventional medicine. While some policy steps (SNAP, school lunch/WIC reforms) are welcome, lasting change requires broader environmental reforms alongside medical strategies.
FDA Drafts MRD-Based Path to Speed Myeloma Drug Approvals
The FDA released a draft guidance proposing minimal residual disease (MRD) and complete response (CR) as endpoints to support accelerated approvals for multiple myeloma. The document outlines trial design, timing, and MRD thresholds (MRD negativity at 10^-5, assessed around CR), with MRD potentially serving as an independent endpoint alongside CR, while noting MRD’s limitations—no use in maintenance or smoldering myeloma—and that the guidance is nonbinding. Issued after ODAC support but amid leadership changes, the guidance aims to speed access to new therapies (notably for CAR‑T and T‑cell engagers), though final approvals remain at the FDA’s discretion and unproven to guarantee OS benefits.
Medical Groups Challenge Kennedy Vaccine Schedule in Court
Six leading medical groups plan to sue to overturn the Kennedy-era reduction of the routine childhood vaccine schedule from 17 to 11 vaccines and to block a February vaccine-advisory meeting, arguing the changes lack scientific basis and threaten public health; the lawsuits echo ongoing disputes over vaccine policy and Covid vaccine access, while the removed vaccines remain available with provider approval and states enforce immunization requirements.
MAHA Momentum: Statehouses Poised to Shape 2026 Health Policy
RFK Jr.'s Make America Healthy Again (MAHA) agenda is gaining steam in state legislatures, with West Virginia enacting a dye ban and about 75 dye-related bills filed in 2025 across 37 states; the Rural Health Transformation Program ties funding to MAHA reforms, fueling policy efforts on vaccines, fluoride, PFAS, and SNAP in numerous states, while California advances ultraprocessed-food rules and industry groups push for faster corporate action—setting the stage for a vigorous state-led health policy push in 2026.
RSV vaccines prove effective as US tightens access restrictions
New data reinforce that RSV vaccines dramatically reduce hospitalizations, with pregnancy-based protection around 70% and newborn monoclonal antibodies about 81%, and four JAMA studies confirm declines. Despite this, the administration’s restrictions to high‑risk infants could raise RSV hospitalizations and complicate vaccine logistics in a fragmented US system. Safety reviews are underway after trial deaths, though officials say there’s no proven vaccine link, and many countries rely on universal prenatal vaccination.
US Officials and Politicians Push to Restrict Gender-Affirming Care for Minors
The U.S. Department of Health and Human Services announced proposed regulations to ban hospitals from performing sex-rejecting procedures on minors under Medicaid and Medicare, citing concerns over irreversible harm and lack of evidence for safety and efficacy, while also taking steps to restrict federal recognition of gender dysphoria as a disability and addressing illegal marketing of related products to children.
US Links Health Aid to Pathogen Data Sharing, Challenging WHO Negotiations
The US is linking its health aid to countries' agreement to share pathogen data, potentially undermining WHO's existing systems and negotiations on equitable benefit-sharing, with a focus on disease outbreak detection and response, especially in Africa.