FDA in Flux — June 2025 Newsletter

Welcome to FDA in Flux – A Mintz newsletter tracking rapid changes in policy and agency actions that impact medical, life sciences, and consumer product investment decisions and development strategies. 

Meeting on Cell and Gene Therapies Raises Many Questions

What is happening: Leadership from the Department of Health and Human Services (HHS), including the FDA Commissioner, held a roundtable discussion in early June on advancing cell and gene therapy product research and use in patients, especially those with rare diseases. Participants included biopharmaceutical industry executives, medical researchers, practicing physicians, and patient advocates.

Why it matters: HHS commentary during the roundtable signaled a shift towards policies focused on patients’ right to try and on accelerating market access to cell and gene therapy products.

• The right-to-try approach centers on the idea that patients with terminal conditions and no available or reasonably accessible options should be able to request access to investigational therapies, which could prolong or even save their lives.
• Risks are inherent in this model because, by definition, investigational therapies lack sufficient evidence of safety and effectiveness, and their use could result in serious harm.
• However, HHS leadership and medical researchers appeared to agree that FDA’s high benchmarks for cell and gene therapy safety and effectiveness are stifling progress in the field and that the agency should start making such products available to patients as soon as the products show signs that they can positively affect patient outcomes.
• This HHS viewpoint seems inconsistent with the recent deep funding cuts to the National Institutes of Health (NIH) and other scientific research projects funded by the federal government, which is likely a larger threat to cell and gene therapy research. It’s possible that HHS, and the Trump administration more generally, believes that private investors will (and should) fill in the gap to fund such early-stage development and clinical research projects in the absence of NIH support. If so, FDA’s desire to lower the barriers to market for cell and gene therapies may be intended to decrease the financial downside risks traditionally associated with such products, thus enticing investors.

Who may be affected: If HHS takes coordinated actions to accelerate market access to cell and gene therapies, it would create a seismic shift in the biopharmaceutical industry and likely result in a major reorientation of research, development, clinical, and investment priorities. Although expedited pathways to patient access and marketing authorization are always welcome in the biopharmaceutical industry, they could come with substantial strings attached, such as requirements for confirmatory studies, patient registries, or other post-market restrictions or surveillance to minimize potential harms. 

Furthermore, although many view right-to-try policies as a potential solution for all patients with deadly diseases, companies that make investigational therapies available to patients with an incomplete understanding of a therapy’s safety and effectiveness may still be subject to significant liability if serious injuries occur. To make these plans workable, the federal government may need to take actions to limit liability in such circumstances. 

MAHA Report Is All Talk but Portends Massive Policy Changes

What is happening: The White House recently published its first highly anticipated Make America Healthy Again (MAHA) Report, complying with a directive in President Trump’s February 2025 executive order establishing the MAHA Commission led by Health Secretary Robert Kennedy, Jr. The report highlights four overarching negative trends affecting the health of American children: ultra-processed foods, environmental exposure to chemicals, access to digital technologies, and “overmedicalization.” The report sharply criticizes the food, medical, and life science industries and existing regulatory frameworks while asserting many potential root causes of perceived issues in health care and wellness. But it also offers scant and often misleading evidence in support of its conclusory statements.        

Why it matters: The MAHA Report will form the basis for a strategic plan for the Trump administration to address a putative health crisis affecting American children, regardless of how well-researched or analyzed it may be. The report describes the focal points and priorities of the administration’s upcoming reform efforts, which will likely roil multiple large and well-capitalized industries. The MAHA Commission’s strategic plan is due to the administration in August 2025. Stakeholders in the affected industries and their financial backers should be aware of several points:

• Although the MAHA Report harshly criticizes food and drug companies and the regulatory bodies and legal frameworks governing such entities, it contains very little substance describing how the perceived risks and harms may be addressed, other than vague recommendations for enormous “clinical studies” and the use of AI.
• The report likely was not subjected to the rigorous review and quality control processes historically used by the US government for similar reports. In fact, the MAHA Commission was forced to submit an amended report on May 29 (just a week after its original release) after many news outlets revealed that the document cited multiple nonexistent publications.
• The circumstances surrounding the report indicate that the MAHA Commission harbors a dim view of the food and life sciences industries, a position that is supported by limited evidence but that nonetheless will shape the Trump administration’s policies toward these key industries. Given the content of the MAHA Report, it is likely that the upcoming strategic plan will propose remedial or punitive measures targeting large food and life sciences companies.

Who may be affected: Companies in the food and biopharmaceutical industries may experience significant impacts to their business operations as a result of the MAHA Report and implementation of the administration’s subsequent strategic plan. Even by itself, the report could encourage consumers to seek alternatives to conventional products due to its painting of the food and pharmaceutical industries in an extremely bad light. Reasonable consumers reading the document would come away with the view that all such entities are corrupt and their products unsafe. Such changes in consumer behavior could adversely affect the business and financial condition of many food and drug companies.

Looking ahead, the MAHA Commission’s strategic plan may call for major changes to government regulations and policies relating to safety, quality, advertising, and public funding in the food and drug industries, which would likely require substantial commitments of money, time, and resources by all stakeholders. In addition, if these efforts lead to changes to FDA’s pre-market requirements for demonstrating drug safety and effectiveness, biopharmaceutical companies could be forced to shift development plans and regulatory activities without the benefit of prior planning and resource allocation. On the other hand, companies offering alternative products and services aligned with Secretary Kennedy’s MAHA agenda could see an increase in business and revenue. 

FDA Rapidly Rolls Out AI Tool for Product Reviews

What is happening: In early June, FDA announced that it has implemented an AI tool, called Elsa, to assist product review teams throughout the agency. The launch comes weeks ahead of schedule as Commissioner Marty Makary previously stated that the software would go live agency-wide by June 30. In his announcement, Dr. Makary expressed confidence that the software would make scientific review and regulatory decision making on medical products more efficient. However, there are early indications that the software needs major improvements and is not useful to scientific reviewers at the agency.

Why it matters: FDA had previously engaged several contractors for a deliberate, planned consolidation of the various Centers’ technology and data systems into a single, agency-wide platform. The new administration jettisoned that approach in favor of rapid implementation of the announced AI tool. Elsa was originally designed and developed for use by the Center for Drug Evaluation and Research, but it has apparently been re-purposed for use by all agency Centers. The software’s current capabilities appear to be limited to summarizing product information submitted by applicants seeking marketing authorization, although Dr. Makary expressed hope that it could be used to predict toxicity and adverse events. The Commissioner and Jeremy Walsh, FDA’s chief AI officer, expect that use of Elsa will lead to a significant decrease in product review times. The software’s actual performance is unclear, however, because FDA has provided no details on how the AI model was trained or tested and the agency-wide launch took place after a minimal pilot period. In fact, early reports from FDA employees indicate that Elsa’s performance is lackluster and that it cannot be used for anything other than basic administrative tasks.

Who may be affected: FDA appears poised to use Elsa to facilitate agency reviews of all product applications, both current and future. Although FDA reviewers’ use of Elsa may produce some efficiencies in the various product authorization processes, it is equally (if not more) likely that the AI tool will lead to misinterpretation of data or information in applications. Inaccuracies or hallucinations by Elsa may lead to confusing requests for further information, which would require companies to devote time and resources to identify and explain inconsistencies to the FDA review teams. Such miscues could lead to longer review times and ultimately delay product launches. Companies will need to carefully consider FDA review team communications and be prepared to identify possible AI-generated inconsistencies with submitted product candidate information. 

Administrative Action to Remove Fluoride Drugs Suggests Greater Scrutiny of All Drugs

What is happening: In mid-May, FDA Commissioner Marty Makary announced that the agency would “begin action” to remove prescription formulations of ingestible fluoride, which for historical reasons have never received FDA approval, from health care markets in the US. The announcement included a goal date of October 31 for the agency to “complete a safety review and public comment period and for taking appropriate action regarding removal of these products from the market.” There is currently no mechanism for submitting public comments on this FDA proposal.

Why it matters: FDA’s announcement is concerning given its apparent conclusion that these drug products will be “removed from the US market” irrespective of the outcomes of the planned safety review or comment period. The agency traditionally has made decisions based on scientific information and following consideration of various policy options, rather than acting in response to political or public pressure. The recent announcement on fluoride drugs suggests a significant shift in the agency’s willingness to shape the commercial marketplace and available options for doctors and consumers, even when serious safety or efficacy problems have not been identified with a particular product.

Who may be affected: If FDA moves forward with plans to remove ingestible prescription fluoride drugs from pharmacies and the US supply chain, directly affected parties are limited to companies that market those products. However, such regulatory actions may herald significant uncertainty and instability for all prescription and over-the-counter drugs, whether they contain fluoride or not, because:

1. a large number of OTC drugs, such as anticavity toothpastes and rinses, contain fluoride and could be next in line for marketplace removal, which could affect not only their manufacturers but also the many retailers that carry those ubiquitous consumer products; and
2. more broadly, agency actions taken against a specific category of grandfathered, unapproved drugs could lead to scrutiny of other older, unapproved drugs regardless of their safety profiles.

Companies should consider whether they may have exposure to any such risks and whether they can leverage existing safety and effectiveness data to resist potential efforts by FDA to remove similarly situated products from the market. Alternatively, companies may want to shift their business strategies to rely on more recently developed, FDA-approved products. 

Major GLP-1 Drug Shortages End; Whether FDA Will Enforce Applicable Law Is Uncertain

What is happening: Compounding pharmacies must figure out a path forward for GLP-1 drug products now that FDA-approved versions of many drugs are officially no longer in shortage. The popular and highly effective diabetes and obesity drugs in the GLP-1 agonist class, primarily semaglutide and tirzepatide, were listed in FDA’s Drug Shortage Database for the past several years. This led to an explosion of pharmacist-compounded versions of the injectable drug products, which are legally authorized during a recognized shortage in order to meet patient and prescriber needs. Today, after transitional periods following FDA’s decisions to remove both semaglutide and tirzepatide from the drug shortage list (and litigation resolved in the agency’s favor), pharmacies and outsourcing facilities cannot legally compound what would be considered “essentially a copy” of the FDA-approved, commercially available products. Many businesses, however, have indicated that they will continue making compounded versions available with added vitamins or other changes sufficient to render them not “copies” of FDA-approved GLP-1 products.

Why it matters: Although FDA has taken enforcement actions against unapproved or counterfeit versions of GLP-1 drugs being sold to US consumers, such prior activities generally have involved commercial-scale, direct-to-consumer sales without any health care professional supervision of the patients’ treatment plans. To date, FDA has not taken action against any compounding pharmacies, outsourcing facilities, medspas, or telehealth platforms that produce or offer “copies” of approved GLP-1 drugs that are no longer considered to be in shortage. If the agency chooses not to act in this space and compounded copies continue to proliferate, the legal framework underpinning US drug law may be irreparably harmed. The potential long-term outcome is that companies may no longer have the same incentives to pursue FDA drug approval for innovative molecules if they will have no assurances that competition will be limited to other FDA-regulated entities.

Who may be affected: Should FDA exercise its legal authority to take enforcement action against compounded GLP-1 “copies” as non-compliant and potentially unsafe compounded drugs, it would directly affect compounding businesses. Such enforcement would also impact related entities that do business with the compounders but are not generally FDA regulated, such as telehealth platforms that connect patients, prescribers, and pharmacies or medspas and local clinicians that may prescribe and source compounded GLP-1 drugs for their patients. On the flipside, however, if FDA remains silent in the face of open violations of compounding laws, it will likely lead to more aggressive marketing of compounded drugs and, eventually, to the development of compounded versions of other popular or expensive drugs, even where there is no shortage and no true clinical need for such compounded products in light of available FDA-approved options. Based on cost differentials, which favor unapproved compounded drugs, prescribers may shift away from FDA-approved drugs, making the standard drug development pathway that culminates in marketing authorization less attractive to investors. This scenario also risks creating a less transparent and less safe pharmaceutical supply chain in the US given that many federal regulatory requirements do not apply to compounded drugs (e.g., supply chain security and serialization provisions).

Subscribe To Viewpoints