Executive Summary 

• The U.S. Food and Drug Administration (FDA) has recently announced several regulatory changes that have varying impacts on the mandate to approve safe and effective drugs. 
• Some of these policy moves can be beneficial to the agency’s mission and fall squarely within its mandate to introduce, while others risk undermining the agency by expanding its role to unprecedented policy areas.  
• This insight explores the impact of these recent announcements and assesses their adherence to the FDA’s mission. 

Introduction 

The U.S. Food and Drug Administration (FDA) recently announced a slew of new initiatives and published new guidance on a wide range of programs, some under its traditional purview and some not. While some of these initiatives create new FDA programs out of whole cloth, and others merely reshape the aims of existing programs, they represent a new, somewhat untested foray into the FDA’s role in the U.S. health care system. Traditionally, the FDA has functioned as a neutral arbiter of safety and efficacy with minimal consideration of short-term political objectives. This is indicated through the statutes establishing and authorizing FDA functions, as well as the statutes of other federal agencies with jurisdiction over various health care-related functions. As an example, the FDA and its advisory committees weigh whether a vaccine is safe and effective, but not whether the general populace should get it. That is the job of the Centers for Disease Control and Prevention and its advisory committees. This separation not only invites additional assessment and expertise but reduces potential conflicts of interest and incentives. 

Nevertheless, the FDA’s recent announcement of its new initiatives and program guidelines calls into question whether the agency will still adhere to its politically neutral mission – or whether it will stray. While there is plenty of opportunity to promote change at the FDA and further enhance the way it operates as a part of the U.S. health care system, care must be taken to prevent the undermining of the agency’s core mission: to evaluate and approve safe and effective pharmaceuticals, biotechnology products, and other health care products. Turning away from this core mission and including additional political considerations – or worse – de-emphasizing that mission in favor of the more political route at the expense of its scientific mission only weakens the FDA. This insight explores some of these noteworthy policy announcements and discusses potential impacts on the U.S. health care system – and whether any might stray from the FDA’s core mission. 

Recent Policy Updates 

The continuous policy announcement and development process creates an ever-changing regulatory environment for the health care industry. Below are some recent, noteworthy initiatives that should be examined. 

Section 804 Drug Importation 

Section 804 of the Federal Food, Drug, and Cosmetic Act has long offered a potential pathway for states and certain entities to import prescription drugs from Canada, but it remained dormant for nearly two decades due to safety and regulatory concerns. Section 804, originally enacted in 2003 through the Medicare Prescription Drug, Improvement, and Modernization Act, authorizes the secretary of Health and Human Services (HHS) to permit the importation of certain drugs from Canada if two key conditions are met: The drug must pose no additional risk to public health and result in a significant cost reduction to U.S. consumers. 

No HHS secretary provided the required certification until 2020, largely due to concerns about the FDA’s ability to ensure the safety and integrity of foreign drug supply chains. At that time, it was determined that Canadian drug importation could meet the law’s requirements. Shortly thereafter, the FDA finalized regulations under 21 CFR Part 251 to establish the framework for State Importation Programs (SIPs), which would allow states and tribes to submit proposals to import eligible drugs from Canada under strict testing, labeling, and monitoring protocols. 

The final rule focused exclusively on Canadian importation – excluding biologics, controlled substances, and certain infused or injectable drugs – and emphasized safety measures such as drug authenticity testing, relabeling with U.S.-compliant packaging, and post-importation adverse event tracking. Only Florida has received FDA authorization for a SIP, though several other states have expressed interest. The agency has recently taken steps to expand and streamline the program, issuing updated guidance and new tools to support broader state participation. 

Between May and July 2025, the FDA released a series of enhancements and guidance aimed at turning the promise of Canadian drug importation into practice. The May 2025 “enhancements” introduced three major reforms: pre-submission reviews for draft SIP proposals, informal meetings to clarify requirements, and development of a “user-friendly” online application toolkit – all designed to improve proposal quality and shorten review timelines. The agency also relaxed cost-savings calculations, allowing states to use static baseline models if assumptions are well-documented. The July 2025 official guidance attempts to further spur Section 804 work. This guidance offers concrete steps to program sponsors: a full checklist of required elements, a helpful “Tips for SIPs” document, and a small-entity Q&A compliance guide. It also clarifies the evaluation process, noting that the FDA may seek additional information, and that SIP authorization may include extensions of up to two years. The guidance also formalizes states’ ability to request optional informational meetings before and after official proposal submissions, a strategic resource for early regulatory coordination. 

While the regulatory pathway continues to be smoothed, political, logistical, and international trade concerns slowed progress. The issues that arise call into question the role the FDA must take and the resources the agency must commit to ensuring the health care system isn’t undermined, or patients are exposed to additional risks due to imported drugs. The agency must also ramp up internal capacity to review submitted proposals, inspect foreign facilities, perform regular and new, specific oversight, and manage post-import data and distribution.  

Notably, Canada responded with regulations restricting bulk exports of drugs that could create domestic shortages. This further complicates the implementation of this program. For states and tribes to realize any savings for their populations, they need to be able to purchase the drugs they seek.  

Commissioner’s National Priority Voucher Program 

The FDA unveiled a new initiative known as the Commissioner’s National Priority Voucher (CNPV) Program, designed to accelerate the review of drugs that serve U.S. national interests. Spearheaded by the FDA commissioner, the program is running as a one-year pilot beginning later this year. It offers drug sponsors the opportunity to receive a significantly expedited review – potentially within one to two months – compared to the standard 10-month timeline or even the six-month priority review. In exchange, companies must align their products with one or more national priorities, such as pandemic preparedness, domestic manufacturing, national security, or addressing serious unmet medical needs. To qualify, applicants must pre-submit key Chemistry, Manufacturing, and Controls information data at least 60 days before submitting a full application and remain highly responsive to FDA queries throughout the review process.  

These vouchers are non-transferable and must be used within two years, though they can survive mergers or acquisitions. Unlike other FDA-issued vouchers created and authorized by Congress, the CNPV is entirely an agency-led initiative and cannot be sold or traded. The review itself will be conducted by a specialized, cross-disciplinary team in a collaborative, case-conference format. The potential benefits include faster access to innovative therapies and alignment of private development incentives with public health priorities.  

The particulars of this program, however, call for additional guardrails and implementation guidelines. Supporters argue this model could help the FDA rapidly approve drugs that are urgently needed in the United States while incentivizing companies to invest in national public health goals. Questions remain, though, about how the FDA will allocate already limited resources, ensure review quality under compressed timelines, and enforce the intended policy without legislative guardrails or backing (which other priority review programs have). Beyond logistical questions, a central pillar of this program is commissioner-focused determinations and prioritizations. The criteria for determining which companies get a CNPV are amorphous and could enable the FDA to pursue broader interests outside its statutory remit. There is thus potential for misuse, creating uncertainty in FDA priorities and predictability.  

Complete Response Letters 

The FDA launched a transparency initiative by publicly releasing more than 200 Complete Response Letters (CRLs) issued between 2020 and 2024 through its openFDA portal. CRLs detail why a New Drug Application (NDA) or Biologics License Application (BLA) was not approved in its current form, commonly citing issues related to safety, efficacy, manufacturing, or bioequivalence. These documents were previously available only to applicants due to their sensitive nature and containment of proprietary information. If they were ever released, they were often redacted in any public disclosures. 

The FDA, under the leadership of the commissioner, is pushing “radical transparency,” aiming to eliminate information asymmetry and reduce redundant development errors by making regulatory feedback accessible to developers, investors, and patients. While most published CRLs correspond to drugs that were ultimately approved – and many were already contained in publicly posted action packages – the FDA assures ongoing document releases, including possibly for unapproved applications. Letters seem to have been redacted to protect trade secrets and commercial confidentiality.  

A desire for transparency is admirable given the weight of the mandate the FDA operates under. Issues arise, however, when that disclosure is premature and without context. If a pharmaceutical product is not approved, there is nothing about that product that needs to be shared with the broader public as a warning, because a consumer doesn’t have access to a product that isn’t approved. It preempts a company’s ability to respond effectively and efficiently. The FDA purports to disclose this information in the interest of shareholders and patients; that extends far beyond its mission to determine whether a drug is safe or effective. The most important part of correspondence between the FDA and drug sponsors is simply whether the drug was approved. If the FDA feels that the drug is safe, but patients may benefit from further information, then it is well within the scope of its authority to require additional labeling. What is not in its scope of authority is iterative information disclosure that may negatively impact a company’s competitiveness in the industry or influence capital markets. 

Cost Factor in Drug Approvals 

The FDA commissioner proposed a groundbreaking shift in regulatory strategy: incorporating international drug price equality – specifically aligning U.S. prices with those in other wealthy Organisation for Economic Co-operation and Development (OECD) countries—as a formal consideration during NDA and BLA reviews. Speaking in a Bloomberg interview, Makary emphasized that drug sponsors who commit to “equalize” their U.S. launch prices with foreign markets could qualify for fast-tracked priority review vouchers, accelerating the process from the standard 10‑month timeline to just one to two months. 

This linkage between pricing and review speed breaks new ground: For the first time, the FDA is expressly acknowledging price parity as a factor in its approval calculus, historically kept siloed from the safety, efficacy, and marketing strategies that FDA is meant to consider. The commissioner framed it as a tool to reduce disparities, ensuring Americans wouldn’t pay more than patients abroad. The mechanism mirrors the recently launched Commissioner’s National Priority Voucher pilot as described above. 

This may cause a legal conflict. The FDA, by its own admission, does not have the authority to consider or leverage its mandate to address drug prices: 

We understand that drug prices have a direct impact on the ability of people to cope with their illnesses as well as to meet other expenses. The FDA, however, has no statutory authority to investigate or control the prices charged for marketed drugs. These prices are established by manufacturers, distributors, and retailers. 

Any alterations to approval processes that lessen the emphasis on efficacy and safety and introduce political considerations create uncertainty for the industry. The pharmaceutical industry requires intensive planning and resources, and an approval process that relies on not only the person sitting in the commissioner’s seat but extraneous factors that pharmaceutical companies don’t control (such as the fiscal state or negotiating posture of OECD governments, particularly those with single-payer health care) does not invite predictability or uniformity. Furthermore, the United States is often the country of first launch for new pharmaceuticals, with more than 50 percent of new drugs being launched first in the United States. Other major markets have some level of delay (years) before they are launched. If the United States is the country of first launch, then there isn’t an international price to be “equalized to,” which further calls into question how these factors are going to be weighted. 

Leveraging Artificial Intelligence 

The FDA has also created a landmark push to use artificial intelligence (AI) as a cornerstone of drug application reviews and approvals. It has taken an enormous step by launching Elsa, a generative AI platform deployed agency-wide to streamline drug and device reviews. Elsa assists with tasks such as summarizing adverse event reports, comparing labeling, reviewing clinical protocols, flagging high‑priority inspection sites, and even generating code for nonclinical data databases. Built within a high-security GovCloud environment, the tool is not trained on industry-submitted data, ensuring proprietary information remains protected. 

Initial pilot results reportedly reduced tasks taking days to mere minutes, according to users in the Center for Drug Evaluation and Research. The rollout was accelerated ahead of schedule, which was met with some concern. The FDA chief AI officer highlighted Elsa’s potential to “enhance and optimize the performance and potential of every employee.” 

Elsa’s deployment is part of a broader FDA modernization strategy under the commissioner’s transparency and efficiency agenda. It is intended to complement other initiatives, like the rapid real-time review process aimed at slashing drug approval timelines from 10–12 months to as little as one to two months. Concerns have emerged, however. Some FDA personnel and external experts warn the rollout was too hasty, citing incomplete guardrails and occasional inaccuracies – typical of large language model systems. The agency emphasizes that human review remains essential, describing Elsa as a tool to reduce reviewer “busywork,” not replace scientific judgment. Elsa marks a significant leap toward integrating AI into regulatory workflows, promising faster, more efficient reviews. But its success will rely on validating accuracy, ensuring oversight, and maintaining scientific rigor as technology evolves. 

Downstream Effects 

Industry relies on regulatory predictability and agency stability to plan the resource-intensive process of bringing a drug or medical device to market. For example, industry sources contend that – from conception to manufacturing to distribution – pharmaceuticals take 10–15 years and nearly $2 billion to bring to market. Unpredictable, heavy-handed government intervention could reduce private-sector incentives to develop new treatments. A 2019 study by the University of Chicago estimated that drug price controls could reduce global R&D spending by up to $2 trillion, or a 29-percent to 60-percent reduction in R&D from 2021 to 2039, translating into 167 to 342 fewer new drug approvals during that period. Trading a scientifically rigorous and accessible market for the whims of whoever is the commissioner of the FDA may lead to fewer treatments and innovation. 

The FDA has long been a target for modernization and process improvements. In the service of its mission for approving safe and effective medical products, this modernization effort is important and should be fruitful. Wrongful implementation, however, could transform the FDA’s role from a neutral regulator into an active political participant. Introducing variability in the determination of priorities through individual discretion, seeking transparency at the potential expense of incentivizing research and development, and embedding global price parity into its approval process threaten to diminish the FDA’s role as the key arbiter of safety and efficacy in the U.S. health care system. 

Conclusion 

There is plenty of opportunity to promote change at the FDA and further enhance the way it operates as a part of the U.S. health care system. Compelling the FDA to expand the scope of its remit beyond its statutory authority, and assuming functions that it was not intended to serve, has the potential to undermine its mandate.