Who Deserves a Cure? The FDA’s New Gatekeeping Game

Jeffrey A. Singer, Akiva Malamet, and Terence Kealey

Last month, Food and Drug Administration Commissioner Marty Makary announced a new initiative: the Commissioner’s National Priority Voucher (CNPV) program. These non-monetary vouchers, valid for two years, let companies shorten the FDA’s final review of a drug application from 10–12 months to just one or two. The companies cannot sell or transfer the vouchers to other companies. Oddly, CNPVs are non-transferable between companies, but the FDA allows companies to apply them to any drug in their own pipeline. This asymmetry is hard to justify and introduces unnecessary arbitrariness into the process.

A Welcome but Highly Flawed Shift

Although we believe this reform has many shortcomings, the increase in fast-track options is an improvement over current regulatory stagnation and systemic dysfunction (what Jonathan Rauch calls “demosclerosis”). The pandemic and other health challenges have underscored the costs of delayed drug approvals, and we are encouraged to see policymakers taking steps to streamline and simplify the approval process—a cause we have long supported. We also appreciate the FDA’s attempt to define fast-track criteria, even if flawed.

However, we also strongly caution that the CNPV and similar approaches heavily incentivize rent-seeking, lobbying, corruption, and excessive bureaucratic discretion in drug value assessments. Additionally, Nobel Laureate in Economics F. A. Hayek and economist Don Lavoie noted that persistent gaps in knowledge affect all forms of government planning and bureaucratically determined market entry. These issues are prominently seen in the FDA, which often exemplifies Lavoie’s criticisms of “non-comprehensive planning.”

The Off-Label Incoherence

Unfortunately, the problems present in the CNPV are nothing new. The field of drug regulation frequently suffers from incoherence and readily apparent arbitrariness. When the FDA permits a pharmaceutical company to market a drug for a specific condition listed on the label, the agency still allows clinicians to recommend the drug to their patients for any condition they believe it could help. Between one-fifth and one-third of all medicines that clinicians prescribe and patients take are for these “off-label” uses. In other words, the FDA forces drugmakers to prove that a drug works for condition A, but it defers to clinicians to determine whether it works for conditions B through Z. Yet the FDA won’t defer to clinicians for condition A. Rationally, in a world of successful off-labelling, the FDA’s restrictions should be limited to Phase 1 clinical trials, which are the immediate safety checks.

Non-professionals also use FDA-approved over-the-counter drugs off-label. People commonly use OTC antihistamines like diphenhydramine (Benadryl) as a sleep aid, for example. Another example is using topical hemorrhoidal creams containing hydrocortisone to diminish the under-eye “bags.”

The FDA’s New Incoherence

An FDA press release stated the agency will award its new CNPV vouchers to companies “aligned with US national priorities,” which it defines as “addressing a health crisis in the US,” “delivering more innovative cures for the American people,” “addressing unmet needs,” and “increasing domestic drug manufacturing as a national security issue.” But this politicizes the process of drug regulation, for who decides what counts as an “unmet need,” a “more innovative cure,” or a “national security issue,” and how do they demonstrate it objectively? Or, as the economist Thomas Sowell puts it: “The most basic question is not what is best, but who shall decide what is best.”

The FDA says it will handpick internal experts to conduct a “team-based review” of applications pre-screened by an agency-selected group of physicians and scientists during a one-day meeting. However, we know that governments will often overrule strictly medical priorities in favor of political ones. So, for example, for political reasons, Presidents George W. Bush and Barack Obama collectively blocked access to “Plan B” emergency contraception for more than twelve years. We also know that selective government programs increase the risk of corruption—witness the centuries-old history of tariffs, which have often resulted in corruption and favoritism. 

Even if we assume limited political influence, many CNPV criteria are subject to bureaucratic discretion and assertions about national needs, with little to no pushback. The political scientist Philip Tetlock has shown that expert judgment and forecasting are often flawed. At the same time, the economist Roger Koppl argues that “expert failure” is a routine hazard when experts have monopoly power and good incentives and knowledge are absent or misaligned. One of us (Singer) has argued that executive decisions can lead to costly scientific gatekeeping.

We expect the CNPV program to both contribute to and exacerbate these issues. In short, the CNPV program, despite its innovative appearance, operates within a bureaucratic and centralized framework. It may ease restrictions for a select few, but it remains just as limiting for everyone else. The CNPV’s criteria promote rent-seeking and shift the focus of pharmaceutical research away from patient needs toward political interests and the priorities of favored companies.

Fast When It Wants to Be

The most important lesson to take from Commissioner Makary’s CNPV program is that the long-drawn-out process of FDA drug approval can be shortened. Indeed, Operation Warp Speed during the COVID-19 pandemic showed that the FDA can review and approve drugs quickly when it chooses to. The agency notably chose to suspend certain regulatory restrictions during a crisis. If these safeguards can be set aside for public health emergencies, why are they considered essential during times of greater public safety? This inconsistent approach suggests that lengthy and complex drug approval processes may not be as crucial for public protection as often believed. It also highlights the significant societal costs of keeping potentially beneficial drugs off the market. So if the agency claims it can cut review times by 90 percent, why limit that speed to only companies it favors?

Economists and other scholars studying pharmaceutical regulation warn of “drug lag” and “drug loss.” Drug lag is the delay that the FDA’s efficacy requirement imposes before consumers can access new treatments—delays that cause people to suffer or die needlessly. Drug loss happens when companies forgo developing drugs they fear won’t justify the high cost of FDA approval – an invisible graveyard of potential medical breakthroughs.

The High Cost of Delay

In 2012, Jack Scannell and his colleagues coined the term Eroom’s law, which is Moore’s law spelled backwards, to note that drug discovery—unlike the exponential advancements of other forms of technology (such as transistors) over time—is becoming slower and more expensive, despite improvements in technology such as high-throughput screening, biotechnology, combinatorial chemistry and computational drug design. Indeed, the inflation-adjusted cost of developing a new drug roughly doubles every nine years. A major contributor to such escalating costs is the FDA’s docket of regulations. 

But if the FDA can move as fast as the CNPV program indicates, it shouldn’t limit faster access—and the thousands or millions of lives it could save—to just those whose conditions align with the agency’s political priorities. Instead, all companies should have access to accelerated approval for all drugs. The right to make your own health decisions is a bedrock of medical ethics, especially when such decisions can mean the difference between life and death. Politically driven criteria, such as prescription requirements, sharply curtail and weaken that right, while costing many people their well-being or even their lives.

Gatekeeping for Whom?

A deeper question is: Should the FDA be approving drugs at all? It is generally in the interests of drug companies to minimize risk, both to themselves and to their patients, due to concerns over reputation and liability. There is also significant empirical evidence suggesting that, on net, the bureaucratization of drug approval has minimal public benefits and delays the introduction of newer, better drugs at public expense.

A decentralized, competitive, multi-layered, and pluralistic approach combining tort law, insurance company reviews (especially integrated systems like Kaiser Permanente that can identify unsafe drugs the FDA failed to block), medical facility vetting, and third-party quality assurance certifiers such as the American Medical Association’s Council on Pharmacy and Chemistry, or Consumer Reports, would serve as more effective, secure, and dynamic regulation. This system could achieve better outcomes with fewer undesirable trade-offs than an executive agency. As our colleague Peter van Doren documents, markets and nonprofits offer various ways to satisfy diverse preferences while protecting the public from excessive risk and medical or financial burdens.

As argued in the new book Your Body, Your Health Care, respect for adult autonomy guides doctor-patient relationships, but disappears when the government takes control. This inconsistency violates the respect for personal freedom that serves as a cornerstone of modern liberal democracy, American culture, and medical ethics:

While informed consent and respect for autonomy govern how health care practitioners interact with their patients, this new ethos is absent when it comes to the government asserting authority over adults’ health decisions. The government dictates what kinds of health professionals adults may consult. It determines what medicines adults may purchase and under which circumstances they may consume them. It bans adults from ingesting substances or engaging in activities that the government decides are unhealthful, regardless of individual risk-benefit priorities. It does all this because the government assumes it knows what is in adults’ best interest.

Because the FDA controls drug access, this creates a zero-sum dynamic over approval standards and pathways to market. They also reflect the values, preferences, and tolerances the agency considers worthy of respect or accommodation. The FDA’s inconsistent approach—delaying some drug approvals while fast-tracking others—fuels those prone to vaccine skepticism and scientific mistrust, particularly following Operation Warp Speed and other pandemic-era initiatives.

The FDA’s implementation of a uniform, top-down risk assessment standard for all US residents ignores that patients have different tolerances for risk, especially when making decisions about their health, which many consider among their most personal and valuable assets. By limiting access based on a centralized risk evaluation, the FDA reduces democratic values of participation, voice, and personal agency.

The emergence of the MAHA movement (Make America Healthy Again) underscores how executive authority and blanket health policies can fuel political conflict and division, while also eroding public trust. Recent studies and polls have found correlations between federal agencies’ breaches of public trust and vaccine hesitancy or medical skepticism. FDA standards are not neutral or uncontroversial. They involve decisions about ingredients, formulations, processing methods, evidence, and other aspects over which many people reasonably disagree. We are very concerned about the potential for public harm, flawed scholarship, and frequent insensitivity to evidence found in the MAHA Report and the broader movement, which Doctors Singer, Kealey, and Bautista Vivanco strongly criticized. But disagreement over specific claims should not obscure a more fundamental issue: people have a legitimate interest in exercising agency over personal health decisions, and a healthy civic culture must leave room for that, even when choices diverge from a scientific or regulatory consensus. If we don’t, we risk further fracturing an already polarized society.

Expand Patient Rights, not Vouchers

Montana offers a more hopeful model. Since 2018, most states and the federal government have passed Right-to-Try laws that allow terminally ill patients access to experimental therapies that have passed Phase 1 (safety) testing but which have not been approved for clinical use by the FDA. But patients’ uptake under these laws has not been extensive because they already had access to such drugs under the FDA’s expanded-access, or “compassionate-use,” program.

Montana, however, has now passed a Right-to-Try law that permits patients with any condition, not just life-threatening diseases, to seek treatments that have passed phase one of FDA clinical trials. This represents an extraordinary expansion of individual patients’ rights and should be widely applauded.

Consider Drug Reciprocity

Let us suggest an additional, beneficial relaxation: “international drug reciprocity.” If Congress implemented international drug reciprocity, American doctors and patients could access drugs and devices approved by regulatory agencies in trusted countries like Canada, France, the United Kingdom, Switzerland, Australia, New Zealand, and Israel, many of which have engaged in mutual reciprocity for years.

Many people can’t imagine a world where the FDA doesn’t have a monopoly on drug approval. Yet there will always be a market for drug safety and efficacy information subject to public or industry demands. Ending the agency’s monopoly will allow innovation and competition in that space, benefiting everyone, even the FDA bureaucrats, whose expertise would remain highly sought after.

And if, in the interim, we are to have government drug approval, let us encourage competition between our state governments and allow different states or different cohorts of states to host their own interstate FDA consortia. Let our regimes of drug approval become the “laboratories of democracy” that Louis Brandeis adumbrated in his dissent in the 1932 case of New York State Ice Co v Liebmann. 

A Glimmer of Hope 

Commissioner Makary’s CNPV program is flawed and likely to entrench favoritism and bureaucracy, but its existence signals a deeper shift. Even the FDA now implicitly admits that drug approval takes too long, costs too much, and adversely affects patients in need. A growing, cross-ideological movement recognizes this and is working to reduce harmful regulatory barriers, increase access, and accelerate scientific progress and societal well-being. Reform shouldn’t reward a politically favored few; it should expand opportunity for all.

The FDA’s new voucher program may appear to be progress, but in reality, it speeds up a few applications while everyone else waits in the slow lane. If we want a health care system that values innovation, abundance, and personal choice, we must ensure that every patient—not just the politically connected—can decide what care they need.