Biopharma executives applauded a pilot priority review pathway announced by FDA Commissioner Marty Makary Tuesday, anticipating streamlined reviews of innovative drugs, but some industry leaders, FDA officials and patient advocates privately expressed concerns.

In a sharp departure from other FDA programs aimed at advancing innovation, the commissioner’s national priority voucher (CNPV) will be available only to U.S. companies and will be awarded based on criteria that are not directly related to patient benefit. The role of a political appointee in selecting participants in the program is also a break with precedent.

Biopharma executives were quick to express support for the initiative.

“Bravo @DrMakaryFDA @US_FDA! This will be a positive incentive for US innovation!” John Maraganore, chairman of City Therapeutics Inc. and former and longtime CEO of Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY), posted on X.

David Ridley, a health economist at Duke University’s Fuqua School of Business and one of the architects of the priority review voucher (PRV) program, also welcomed the move. “I applaud Dr. Makary and his team for their willingness to explore new approaches, including speeding AI at FDA and the new priority program,” he wrote in on LinkedIn.

Ridley highlighted a key distinction between the pathway Makary announced Tuesday and PRVs: “The new priority program requires the government to pick winners,” unlike PRVs, which operate as market-based incentives. “Winner-picking,” he added, “is standard for NIH grants.”

Having the FDA commissioner select “winners” is concerning, some CEOs told BioCentury.

They worry that the pathway’s broad eligibility criteria — and Makary’s central role in selecting recipients — could invite political favoritism or become a mechanism for rewarding companies with ties to the Trump administration. Some fear it could chill critical discussion of policy disputes or penalize those expressing opposing views.

President Donald Trump’s inclusion of language about FDA in an executive order about most favored nation drug pricing, and Makary’s comments supporting MFN in a recent JAMA commentary, reinforce industry’s concerns that the agency could stray from its traditional focus on medical products regulation.

Current and former senior FDA officials offered mixed reactions. Some welcomed the pilot pathway as a way to remove unnecessary barriers to innovation, but they also cautioned that it could undermine the integrity of FDA oversight and divert resources at a time when the agency is already struggling to meet PDUFA goals.

Patient advocates expressed frustration that the Trump administration is prioritizing a pilot that benefits a select number of therapies instead of pushing for reauthorization of the rare pediatric disease priority review program and committing to maintain NIH funding for basic biomedical research.

Because FDA hasn’t released details about the program, it is possible that feedback from the biopharma industry and FDA staff could lead to changes that would mitigate concerns. These could include clear, objective criteria for awarding vouchers in a process that is controlled by career staff, along with a commitment to ensuring the independence of scientific reviews.

Industry, FDA, and advocacy leaders who spoke with BioCentury requested anonymity to speak candidly.

The commissioner’s voucher program, which Makary announced in a press release, FAQ document and video, will start as a one-year pilot. The agency has not released critical details such as how many products or companies will be eligible for the program.

Unlike priority review vouchers and pathways such as accelerated approval which were created by Congress, FDA did not mention any plans to seek congressional authorization for the program.

Although Makary is calling CNPV a voucher program, it more closely resembles a review pathway. Unlike PRVs, benefits provided under the program will not be transferable.

The vouchers will be issued for a specific therapy, or to a company.

If a voucher is issued to a company, the recipient will have the discretion to use it for any product that is “consistent with the program’s objectives,” FDA stated.

Vouchers will be valid for a two-year period.

A voucher will enable a sponsor to submit an NDA or BLA on a rolling basis and to receive intensive attention from regulators.

Applications will be considered in a “team-based review rather than using the standard review system of a drug application being sent to numerous FDA offices,” the agency stated.

In addition to a multidisciplinary “pre-review” of elements of the application submitted in advance of the final clinical trial data, products reviewed under the program will be considered in a “1-day ‘tumor board style’ meeting,” FDA stated. A tumor board is a group of physicians and scientists who meet to discuss treatment options for individual cancer patients.

The agency said the rolling submission and intensive advice will reduce review times to 1-2 months following final submission of an NDA or BLA. That would be down from 10-12 months.

While a rolling submission can reduce the time required to make a regulatory decision following final data submission, it can be very costly for FDA. If the clinical data doesn’t support approval, all of the work FDA staff put into reviewing the CMC, toxicology and other aspects of the application is wasted.

FDA also said it “reserves the right to extend the review window if the data or application components submitted are insufficient or incomplete, if the results of pivotal trial(s) are ambiguous, or if the review is particularly complex.”

Broad criteria for picking winners

Information released by FDA suggest that Makary will personally decide which products or companies receive a voucher.

“The FDA Commissioner,” FDA said, “will use specific criteria to make the vouchers available to companies that are aligned with the national health priorities of: addressing a health crisis in the U.S.; delivering more innovative cures for the American people; addressing unmet public health needs; [or] increasing domestic drug manufacturing as a national security issue.”

The Trump administration has made enhancing pharmaceutical supply chains, especially through increasing domestic manufacturing, a high priority.

Since January, at least six companies have announced plans to expand U.S. manufacturing, and therefore may be eligible for a voucher: Abbvie Inc. (NYSE:ABBV), Gilead Sciences Inc. (NASDAQ:GILD), Johnson & Johnson (NYSE:JNJ), Eli Lilly and Co. (NYSE:LLY), Novartis AG (SIX:NOVN; NYSE:NVS), and Roche (SIX:ROG; OTCQB:RHHBY).

Some of these companies, however, may be excluded, depending on eligibility criteria.

In his video statement, which was posted on social media, Makary said, “The program will support U.S. drug developers who are addressing our most important U.S. national priorities.”

Responding to BioCentury’s request for clarification about the eligibility of foreign-based companies, HHS Press Secretary Emily Hilliard said the voucher will be available to U.S. drug companies. It isn’t clear how FDA will define a “U.S.” company or if U.S. subsidiaries of foreign companies will be eligible.

The program’s selection criteria could conceivably apply to a wide range of new drug applications and to any company with, or planning, a significant U.S. manufacturing presence.

Three CEOs of publicly traded companies attending the BIO International Convention in Boston told BioCentury they fear the program could be used to reward firms that align themselves with President Donald Trump.

They also expressed concern that the implicit threat of withholding a voucher, or awarding one to a competitor, could deter corporate leaders from publicly criticizing the administration, challenging statements by HHS Secretary Robert F. Kennedy Jr., or supporting Democratic congressional candidates.

Resource intensive

Two senior FDA officials who will be responsible for implementing the policy told BioCentury that they and other FDA staff were not consulted about the new program and were unaware of it prior to the announcements Tuesday. It isn’t clear to staff how the tumor board approach Makary announced will be implemented, or if it will be possible to maintain its traditional standards and ensure consistency with prior decisions if decision-making is highly compressed.

They noted that intensive interaction between regulators and sponsors, for example as part of the breakthrough designation program, has helped avoid pitfalls and allowed companies to streamline development programs.

The process is resource intensive.

Makary’s announcement comes at a time when staffing shortages caused by layoffs, as well as recusals of reviewers who are seeking employment in the private sector, have led FDA to miss legal and regulatory deadlines.

According to FDA sources, up to 25% of the agency’s reviewers, about 600 people, have recused themselves in recent weeks from active reviews as they pursue jobs in the private sector.

FDA cited staffing shortages caused by the April 1 layoffs in a legal document seeking a delay in a hearing regarding the appeal of a complete response letter received by Vanda Pharmaceuticals Inc. (NASDAQ:VNDA).

Friday, Kalvista Pharmaceuticals Inc. (NASDAQ:KALV) announced that FDA would miss a PDUFA goal for sebetralstat, a hereditary angioedema (HAE) treatment. Kalvista said FDA attributed the delay, which the company said would be about four weeks, to “heavy workload and limited resources.”

Paul Kim, principal at Kendall Square Policy Strategies, contrasted the missed Kalvista PDUFA goal with Makary’s plans to accelerate reviews through the new voucher program.

Kim, a board member at Friends of Cancer Research and consultant adviser to the National Organization for Rare Disorders, wrote on LinkedIn that the voucher program, “if implemented successfully, <could> be the most significant, non-legislative Agency regulatory reform since the adoption of accelerated approval.”

He added, however, that FDA’s failure to meet the Kalvista PDUFA goal “makes for a striking and concerning contrast between an incredibly ambitious, highly irregular initiative and growing evidence of delays and friction in FDA’s ongoing new drug reviews, which threaten to impede innovation.”

Another fear expressed by FDA officials and former agency leaders is that drug reviewers could feel pressure to approve therapies that have been singled out by the FDA commissioner for special treatment.

Although Makary and Center for Biologics Evaluation and Review Director Vinay Prasad have criticized senior FDA leaders for overruling staff, disregarding advisory committees and collaborating closely with drug sponsors, the new program could create similar situations.

Prasad, who joined FDA in May, has already personally intervened in regulatory decisions about a COVID-19 vaccine from Novavax Inc. (NASDAQ:NVAX).

FDA stated Tuesday that it will provide information soon about “how companies seeking to apply can indicate their alignment with the FDA Commissioner’s criteria to meet national priorities.”