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.
2025 was a dizzying year of regulatory change for the biopharma industry. FDA guidance to accelerate the development of cell and gene therapies and rare disease treatments, along with the introduction of new expedited approval pathways, drove considerable momentum—which remains palpable heading into 2026.
But a series of sweeping vaccine policy changes enacted by Health Secretary Robert F. Kennedy, Jr., along with concerns around the Commissioner’s National Priority Voucher (CNPV) program and greater FDA disagreement with its advisory committees, are contributing to a persistent air of controversy.
Indeed, criticism of the FDA, Kennedy and the Department of Health and Human Services has begun to grow. In late January, Moderna halted all late-stage development of its mRNA vaccines for infectious diseases, citing a lack of return on investment due to policy headwinds in the U.S. That same week, Albert Bourla, CEO of Moderna’s COVID-19 rival Pfizer, had harsh words for the secretary, calling Kennedy’s vaccine rhetoric and policies “anti-science” and “almost like a religion.”
Regulatory experts are also sounding the alarm about what they say is the politicization of the agency, particularly alluding to a possible connection between the CNPV program and other Trump administration priorities, such as drug pricing.
As 2026 continues, BioSpace will keep a close eye on key policy changes as they are made, updating this space with our coverage.
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FDA to Open RMAT Designations Even for Drug Candidates on Hold
March 19
The FDA will now allow its Regenerative Medicine Advanced Therapy (RMAT) designation to be awarded to investigational products even if they are on hold.
In considering RMAT requests for products on hold, the “circumstances of the hold/partial hold will be considered to determine how they may affect the RMAT designation,” according to the FDA’s new standard operating procedures. That is, the regulator will weigh whether the reasoning for the hold would prevent it from assessing whether the investigational product “has the potential to address an unmet need”—a key criterion that RMAT hopefuls must satisfy.
The new procedures took effect March 12.
These new guidelines are a departure from the FDA’s previous approach to products on hold. The Center for Biologics Evaluation and Research “will not accept requests for RMAT designation for [investigational new drugs] that are inactive or on clinical hold,” according to the agency’s industry guidance document published February 2019. The regulator will also suspend the processing of RMAT requests for candidates that are put on hold while the application is active, the document continued.
Animal Testing Alternatives
March 19
In April 2025, the FDA announced its push to move away from animal testing and replace them with “more effective, human-relevant methods,”—though the agency at the time did not release detailed guidelines for using these alternatives.
Now the FDA has. In an 11-page draft document published March 18, the agency noted that instead of using animal models, drugmakers can make use of other testing platforms, such as complex in vitro or 2D in vitro models or in chemico and in silico studies. Taken together, these alternative methods comprise what the regulator called new approach methodologies (NAMs).
The FDA’s Center for Drugs Evaluation and Research “encourages the use of NAMs in regulatory submissions,” the guidance noted, especially where these methods offer better “predictivity, reliability, and human relevance of nonclinical tests” over animal models. Still, the regulator will need to validate whether a proposed NAM is indeed suitable for what the drug sponsor is trying to demonstrate. Some key considerations for determining validity include the context of its use, its relevance to human biology and if the alternative test is fit for the purpose it is being proposed for.
Drugmakers should “consult with the applicable review division if there is uncertainty about the suitability of the method for regulatory use when considering use of a nonanimal testing method,” according to the draft guidance.
FDA Defaults to One-Trial Drug Applications
February 27
The FDA will now look for just one well-controlled clinical trial as the basis for drug approvals.
In a Feb. 18 article in The New England Journal of Medicine, Commissioner Marty Makary and Center for Biologics Evaluation and Research Director Vinay Prasad argued that while requiring two studies “has a powerful theoretical basis to reduce false-positive conclusions,” it “no longer makes sense” in practice.
Drug development has become “increasingly precise and scientific,” the regulatory officials continued, adding that overall survival isn’t the only metric of efficacy that the FDA looks at. Rather, the agency also assesses a drug’s effects on biomarkers and other secondary outcomes to “tell a complete biologic story.”
Alongside the singular adequately-run trial, the FDA will also require drug sponsors to file what Makary and Prasad called “confirmative evidence,” such as mechanistic data, results from related indications or findings from animal models.
Personalized Therapies Get Bespoke Pathway
February 27
To support the development of medicines for rare diseases, the FDA on Feb. 23 released draft guidelines that could facilitate the development of these personalized therapies.
The document puts particular focus on genome editing and RNA therapies, and applies to drug candidates that target the “specific pathophysiologic abnormality serving as the root cause of a disease.” After identifying this biological root cause, the regulator recommends that companies use the disease’s natural history in untreated patients as a control to measure how effective their drug candidate is.
Sponsors should also provide evidence that their therapy can directly engage their biological target.
While the FDA is prioritizing rare diseases, this framework can also be used for personalized treatments meant for other indications.
Rare Pediatric Vouchers Reauthorized After 13-Month Delay
February 3
President Donald Trump signed a spending package into law that ended a short partial government shutdown—and restarted the FDA’s rare pediatric disease priority review voucher (PRV) program, which was tucked into the legislation.
The program, which lapsed at the end of 2024 when Congress failed to reauthorize it, provides a PRV to a company or other drug sponsor upon approval of a rare disease therapy. Companies can then either use the voucher to secure a speedier review for another product in their pipeline or sell the voucher for much-needed revenue to continue their development efforts. The going price for a PRV was $150 million at the time the program lapsed.
Its reauthorization was welcome news to biotech companies developing rare disease therapies and rare disease advocates. According to a recent pipeline review by the Rare Disease Company Coalition, there were 200 or more therapies that were at risk of losing eligibility for a PRV if the program was not reinstated, at a potential cost of more than $4 billion in lost revenue.
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FDA Launches PreCheck Manufacturing Program
February 1
On Feb. 1, the FDA began accepting applications for its PreCheck Pilot program, an initiative undertaken in response to an executive order issued by President Donald Trump in August 2025 aimed at providing regulatory relief for U.S.-based pharma manufacturers. Applications will be accepted until March 1, with finalists selected a month later.
The goal of the program is to make the pharmaceutical supply chain more predictable by “facilitating the construction of manufacturing sites in the U.S,” the FDA said in a press release.
In developing the program, the agency met with representatives from Eli Lilly, Merck and other pharma companies. The primary feedback revolved around eliminating the possibilities of FDA product rejections due to shortfalls at manufacturing facilities, possibly by frontloading the facility evaluation process. Lucy Chang, associate vice president in Global Regulatory Affairs CMC Biologics at Merck, called on the FDA to say whether it will waive inspections of facilities that participate in PreCheck and clarify how much faster the evaluation process will be.
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FDA Positions New Endpoints for Multiple Myeloma Trials
January 20
Due to recent success in treating multiple myeloma, the FDA issued draft guidance in January recommending that clinical trials for novel therapies for the disease use an additional endpoint, minimal residual disease (MRD), to secure accelerated approval.
MRD refers to a small, difficult-to-spot population of cancer cells that can cause a relapse of disease even after a patient is said to have achieved a complete response. If patients have fewer than one myeloma cell out of 100,000 healthy cells, they can be declared MRD negative, according to the draft guidance.
In an interview with BioSpace last month, Nicholas Richardson, former deputy director of the FDA’s Division of Hematologic Malignancies 2, explained the rationale behind the move.
Therapies in myeloma are reaching an 80–90% overall response rate (ORR), he said. “So if you’re thinking about new therapies, it’s harder to tell if there’s an improvement.”
For full approval, companies will still need to provide ORR and complete response data.
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FDA Lifts Suicide Warnings From GLP-1 Drug Labels
January 13
In another boost to the already soaring GLP-1 space, the FDA requested that market leaders Eli Lilly and Novo Nordisk remove warnings about suicidal behavior and ideation from their products. In mid-2023, reports of such adverse effects in patients taking the popular weight loss and diabetes treatments prompted regulators around the world—including the FDA and European Medicines Agency—to investigate the potential risks. But these probes ultimately found no causal connection between GLP-1 medicines and suicidality. The FDA announced the results of its preliminary review in January 2024.
These early analyses, however, were hindered by a small number of reported incidences of suicidal thoughts and behaviors, resulting in what the FDA on Jan. 13 called “considerable uncertainty in the risk estimate.” In an announcement that same day, the regulator revealed that a more detailed meta-analysis of different GLP-1 trials, encompassing more than 90 studies and nearly 108,000 patients, did not indicate a heightened risk of suicidality in patients taking these medicines.
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FDA Manufacturing Carve-Out Aims To Accelerate CGT Development
January 11
The FDA is implementing what Commissioner Marty Makary called “common-sense reforms” in how it regulates the manufacturing of cell and gene therapies. These reforms, communicated in a press release on Jan. 11, take into account the complexity of these medicines, which are often individualized to a patient or are manufactured in small batches.
Among the FDA’s concessions, investigational gene therapies will not require a manufacturer to comply with certain manufacturing specifications for biologics, as outlined in Chapter 600 of the Code of Federal Regulations. The regulator will also be lenient regarding “minor manufacturing changes,” as a cellular or genetic medicine moves into later stages of development, providing a sponsor is able to show that such alterations in production do not meaningfully change the product.
These modifications “will enable progress while not compromising or undermining the FDA’s ability to assure safety, purity and potency of a product,” according to the agency’s announcement.
