The U.S. Food and Drug Administration (FDA) and the biopharma industry have experienced a series of regulatory changes over the past year, with several new policies affecting drug development, manufacturing, and approval pathways as 2026 begins.
One major area of debate has been the Commissioner’s National Priority Voucher (CNPV) program. Some regulatory experts have raised concerns about potential politicization within the FDA, suggesting links between this program and broader policy priorities such as drug pricing. The FDA has also faced increased criticism from stakeholders in response to recent vaccine policy changes enacted by Health Secretary Robert F. Kennedy, Jr.
In late January, Moderna announced it would halt all late-stage development of its mRNA vaccines for infectious diseases in the United States, citing insufficient return on investment due to current policy conditions. During that same period, Pfizer CEO Albert Bourla criticized Secretary Kennedy’s approach to vaccine policy, stating: “Kennedy’s vaccine rhetoric and policies [are] ‘anti-science’ and ‘almost like a religion.’”
Meanwhile, President Donald Trump signed a spending package into law on February 3 that ended a partial government shutdown and reauthorized the FDA’s rare pediatric disease priority review voucher (PRV) program. This program had lapsed at the end of 2024 when Congress failed to renew it. The PRV initiative allows companies developing rare disease therapies to receive vouchers for expedited review of future products or sell those vouchers for additional revenue.
Industry groups welcomed this reauthorization. According to an analysis by the Rare Disease Company Coalition, more than 200 rare disease therapies were at risk of losing eligibility for these vouchers if the program was not reinstated—potentially resulting in over $4 billion in lost revenue for biotechnology firms focused on rare diseases (https://www.biospace.com/article/rare-disease-biotechs-stand-to-lose-4b-if-priority-voucher-program-not-reinstated-report-/). The going price for a PRV stood at $150 million when the program expired.
In manufacturing policy developments, the FDA launched its PreCheck Pilot program on February 1 as part of efforts to streamline domestic pharmaceutical production following an executive order from President Trump in August 2025. The initiative aims to make supply chains more predictable by facilitating construction of U.S.-based manufacturing sites and increasing communication between regulators and manufacturers (https://www.biospace.com/article/fda-launches-new-precheck-program-to-lower-regulatory-barriers-to-pharma-manufacturing/). Industry representatives from companies such as Eli Lilly and Merck have called on the agency to clarify whether facility inspections will be waived under PreCheck and how much faster site evaluations might proceed.
For cell and gene therapy developers, FDA Commissioner Marty Makary announced reforms designed to ease certain manufacturing requirements specific to these complex medicines. Under new guidelines communicated January 11, investigational gene therapies may bypass some biologics specifications during early development stages if safety is maintained (https://www.biospace.com/article/fda-carves-out-manufacturing-exemptions-for-cgts-to-accelerate-development/).
In clinical trial guidance updates issued in January, the FDA recommended that studies evaluating novel multiple myeloma treatments use minimal residual disease (MRD) as an additional endpoint for accelerated approval consideration. Nicholas Richardson, former deputy director of one of the agency’s hematologic malignancy divisions, explained: “Therapies in myeloma are reaching an 80–90% overall response rate (ORR)… So if you’re thinking about new therapies, it’s harder to tell if there’s an improvement.” For full approval status however, companies must still provide data on ORR and complete responses.
Separately, after reviewing data from more than 90 studies involving nearly 108,000 patients taking GLP-1 drugs for diabetes or weight loss treatment—including products from Eli Lilly and Novo Nordisk—the FDA concluded there is no evidence linking these medicines with increased suicidality risk. As a result, warnings regarding suicidal behavior are being removed from GLP-1 drug labels (https://www.biospace.com/article/fda-asks-novo-lilly-to-remove-suicide-warnings-from-glp-1-weight-loss-products-/).
BioSpace will continue monitoring further regulatory developments throughout 2026.
