FDA’s Ambitious Priorities Under Commissioner Makary

ByJeff Riegel

For months, everyone in the pharma industry has been trying to divine which direction commissioner Marty Makary will take the FDA in 2025. A change in FDA leadership always brings a new set of priorities—and, inevitably, a flurry of debate about whether the Agency’s updated vision will deliver real progress.

Well, the wait is over. FDA Commissioner Marty Makary and CBER Director Vinay Prasad have recently outlined an ambitious agenda for reform in their JAMA Viewpoint, titled Priorities for a New FDA, and in Agency press releases. But as the dust settles from their announcements, it’s worth taking a closer look both at their bold promises and at some of the practical—and political—questions raised in recent expert commentary.

FDA Commissioner Marty Makary and CBER Director Vinay Prasad have recently outlined an ambitious agenda for reform in their JAMA Viewpoint, titled Priorities for a New FDA

Ambitious Goals

Makary and Prasad’s JAMA article pulls no punches in diagnosing the US health system’s chronic failures, especially for children: “Forty percent of US children now have a chronic medical condition and 1 in 6 has a neurodevelopmental disorder. Life expectancy has plateaued or fallen and is not commensurate with health care spending.” (Makary & Prasad, JAMA, 2025).

They propose nothing less than a transformation of the FDA’s role—from a “purely reactionary health care system to one that is proactive, intellectually curious about underlying causes, and financially aligned to promote health—not just treat sickness.” Their priorities include:

  • Piloting ultra-rapid reviews for high-priority drugs, with decisions “in weeks”
  • Using AI to modernize and speed up reviews
  • A renewed focus on food safety and the role of additives in chronic disease
  • Leveraging “Big Data” for both root-cause research and postmarket surveillance
  • Supporting national priorities such as price equity and domestic manufacturing

These are heady goals, and Makary and Prasad are clear about the need for action: “We must become a user-friendly FDA that partners with industry rather than takes a receive-only posture,” and Markary has vowed to eliminate “unnecessary steps” and “avoidable delays” in the US regulatory landscape.

The Voucher Program: Bold Move or Repackaged Old Ideas?

A centerpiece of this new agenda is the Commissioner’s National Priority Voucher (CNPV) program, announced in a June 2025 press release. The CNPV promises to reduce FDA review times for selected drugs from 10 to 12 months to as little as 1 to 2 months. In an interview with The National Desk, Makary said that the CNPVs are intended to act as “significant incentives for a company to play ball with our national priorities” (AgencyIQ Commentary, 2025).

But are these goals achievable and is this program truly innovative? Although it has some novel elements, analysts point out that the CNPV borrows from longstanding mechanisms in place at FDA for expedited review and prioritization of new drug applications. The CNPV relies on the Agency’s existing pilot programs; namely, Split Real Time Application Review (STAR), Real-Time Oncology Review (RTOR), and Coronavirus Treatment Action Program (CTAP).

Makary proposed that under the new program, companies can submit the bulk of their application (especially the CMC section) before trial completion, with a rapid, “tumor board” style multidisciplinary review of the clinical evidence as soon as final data are available (FDA Press Release, 2025). But he’s provided few details on how they would achieve that unprecedented turnaround time with existing FDA resources and without compromising the rigor of the review process.

According to the FDA, the CNPV is designed to benefit four types of applications:

  • Those intended to address a health crisis in the United States
  • Selected “innovative cures”
  • Those that address an unmet public health need
  • Those that increase domestic drug manufacturing of a critical medicine

The Agency plans to award a limited number of vouchers during a 1-year pilot, which will be nontransferable and good for only 2 years, and it reserves the right to extend the review window if the data are insufficient, trial results are ambiguous, or the application is complex. However, the lack of detail regarding specific criteria used to select who will be granted these vouchers is raising concerns about transparency and the potential for subjective decision making. In response, Prasad has indicated that decisions will be made by committee.

Perhaps most controversially, CNPVs can be awarded to companies, not just products—a departure from the FDA’s usual approach of tying expedited review to specific therapies that address an unmet medical need. AgencyIQ warns this could lead to “vouchers being awarded based on how well a company or its executives [align] with Commissioner Makary personally or the Trump administration,” rather than strictly on scientific merit.

Perhaps most controversially, CNPVs can be awarded to companies, not just products—a departure from the FDA’s usual approach of tying expedited review to specific therapies that address an unmet medical need.

AI and “Big Data”: Do They Offer Solutions?

Commissioner Makary remains bullish on the promise of acceleration, and use of AI could be a key to delivering on that promise. Both Makary and Prasad have been vocal about the FDA’s embrace of AI, including its new ELSA tool, which Makary described at BIO 2025 as “insanely popular in the pilot group.” In their JAMA article, they claim AI “can make a first-pass review of documentation…and aid in generating standardized tables.” They also highlight plans to use AI for computational toxicology, reducing the need for animal testing.

Although these initiatives are promising, skeptics may wonder whether the current enthusiasm for AI is running ahead of the evidence, especially given the complexity of regulatory decision making, and there have been reports that the rollout of ELSA has been difficult.

On the use of Big Data, Makary and Prasad highlight two new opportunities: (1) to use health data to better understand the root causes of chronic disease; and (2) for postapproval monitoring of safety, particularly in rare diseases, wherein randomized trials are challenging. This is a reasonable direction, but many experts caution that real-world data can supplement but not replace the rigor of randomized controlled trials—especially for safety signals.

Healthier Food

On the subject of improving the US food supply, Makary and Prasad point out that, “there has been little research on arguably the most important potential root causes of chronic disease in the United States: our increasingly chemically manipulated diet.” To that end, they have begun a full inventory of “concerning ingredients” in the US food supply and have already begun the removal of petroleum-based food dyes. Work is ongoing to define ultraprocessed foods and consult with experts on the risks of ingesting talc.

Conclusion: A Bold Vision—But Real Challenges Lie Ahead

Makary and Prasad’s agenda is sweeping, and their willingness to challenge the status quo is commendable in some respects and troubling in others. Their focus on chronic disease, food safety, and faster cures reflects real and urgent needs in US health care. A recurring theme in Makary and Prasad’s remarks is a commitment to “gold-standard science and common sense,” with transparency as a core value.

But many of their headline initiatives—most notably the voucher program—are built on existing mechanisms, and critical details remain vague. Without clear, objective criteria and sufficient resources, the risk is that these “innovations” will deliver more in the way of headlines than of lasting impact. For all the talk of acceleration, the true test will be whether the Agency can deliver on its promises without compromising the scientific rigor and trust that are the FDA’s foundation.

Jeffrey S. Riegel, PhD
SVP, Scientific Communications

Jeff combines his scientific expertise in molecular biology and immunology with 30 years of global healthcare agency experience in guiding medical and regulatory communication strategies for biopharma companies. Jeff leads the scientific team at HCG ProEd Regulatory, which helps clients prepare for FDA Advisory Committee meetings and other health authority interactions. Connect with Jeff on LinkedIn.

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