FDA’s Departure from conventional policymaking procedures: policy by journal articles

Josh Oyster
Ropes & Gray LLP, United States
Joshua.Oyster@ropesgray.com

Beth Weinman
Ropes & Gray LLP, United States
Beth.Weinman@ropesgray.com

Jessica Bushman
Ropes & Gray LLP, United States
Jessica.Bushman@ropesgray.com

Introduction

Since the start of the new presidential administration in January 2025, the US Food and Drug Administration (FDA) has announced sweeping reforms to its drug review and approval processes. Recent policy announcements include:

• a new regulatory approach to individualised therapies referred to as the ‘plausible mechanism’ framework published in the New England Journal of Medicine (NEJM) and followed by a draft guidance months later;^[1]
• a one-pivotal-trial default expectation for drug approvals announced in NEJM;^[2]
• a revised Covid-19 vaccination regulatory scheme requiring randomised clinical trials for healthy individuals rather than relying on immune response data first announced in NEJM;^[3] and
• heightened approval standards for chimeric antigen receptor T-cell products in oncology published in The Journal of the American Medical Association (JAMA).^[4]

Notably, FDA has used publications in restricted access^[5] journals as a primary vehicle for communicating these policy changes. Policy pronouncements by FDA in this limited-access format raise serious legal concerns, particularly given the absence of public comment. Such a practice may contravene fundamental administrative law principles and federal requirements established by the Food and Drug Administration Modernization Act of 1997 (FDAMA).^[6]

FDA’s unconventional approach: policymaking outside the prescribed process

The Administrative Procedure Act (APA) establishes the procedural framework for federal executive departments and independent agencies in the United States. The Act requires that, when making policy, executive agencies adhere to comprehensive procedural formalities. Congress imposed these procedural requirements on the executive branch to ensure that the key principles of democratic governance, including public participation, limited government, and accountability, are embedded in the legal fabric of executive branch activity. Agencies like FDA, however, do not always make and announce policy interpretations through formal APA procedures. For example, FDA has historically depended heavily on issuing guidance documents, which are non-binding documents that convey an agency’s current thinking on a topic.

Recognising that neither the APA’s regulatory requirements nor the democratic principles undergirding them extend to guidance documents, Congress amended the Federal Food, Drug, and Cosmetic Act in 1997, through enactment of the Food and Drug Administration Modernization Act of 1997, to, among other things, instruct the Secretary of Health and Human Services (HHS Secretary) to adhere to uniform standards and involve public participation when developing guidance documents that present FDA’s views.^[7] In the provision codified at 21 USC, section 371(h)(5), Congress required that the HHS Secretary promulgate a regulation specifying the ‘policies and procedures of the Food and Drug Administration for the development, issuance, and use of guidance documents’ consistent with the principles outlined in the section.^[8] In 21 USC, section 371(h)(1)(C)(i), Congress also required that:

‘For guidance documents that set forth initial interpretations of a statute or regulation, changes in interpretation or policy that are of more than a minor nature, complex scientific issues, or highly controversial issues, the Secretary shall ensure public participation prior to implementation of guidance documents, unless the Secretary determines that such prior public participation is not feasible or appropriate. In such cases, the Secretary shall provide for public comment upon implementation and take such comment into account.’^[9]

In response, FDA promulgated section 10.115 of Title 21 of the Code of Federal Regulations, which establishes good guidance practices (GGPs) governing how the Agency should develop, issue, and use guidance documents.^[10]

FDA’s recent practice of announcing new policies in restricted-access journal articles that lack a mechanism for formal public comment is in tension with congressional intent and FDA’s GGPs. This tension is plain upon examination of section 10.115. The provision defines ‘Level 1 guidance documents’ as documents that

‘(i) [s]et forth initial interpretations of statutory or regulatory requirements;

(ii) [s]et forth changes in interpretation or policy that are of more than a minor nature;

(iii) [i]nclude complex scientific issues; or

(iv) [c]over highly controversial issues.’^[11]

For such guidance documents, FDA must publish a notice in the Federal Register announcing the availability of the draft and final guidance, post the draft on the internet and have it accessible in hard copy, and solicit public comment followed by amending the document as appropriate in response.^[12] The regulation also advises that FDA may utilise public fora or advisory committees to solicit broader stakeholder input.^[13]

FDA’s recent policy announcements via journal articles reflect the type of agency announcement that should be issued as a Level 1 Guidance. For instance, FDA’s formalisation of a one-pivotal-trial default for drug approvals – announced in an NEJM article in early 2026 – is a quintessential example of a change in policy that is ‘of more than a minor nature’. After all, moving from a two-trial to a one-trial default will generally mean that clinical development plans for all drugs can theoretically obtain the necessary clinical data for an FDA submission through one clinical trial rather than two, as FDA has historically required to meet the ‘substantial evidence’ standard for drug approval established by the 1962 Kefauver-Harris Amendments.^[14] Although the GGP regulation excludes journal articles from the guidance document definition, it also explicitly prohibits FDA from using ‘documents or other means of communication that are excluded from the definition of guidance document to informally communicate new or different regulatory expectations to a broad public audience for the first time’, emphasising that ‘[t]hese GGP’s must be followed whenever regulatory expectations that are not readily apparent from the statute or regulations are first communicated to a broad public audience’.^[15] Announcing policy changes in a journal article, a medium not governed by good guidance practices, is precisely the type of informal communication the regulation expressly prohibits.

Moreover, by publishing policy changes in journal articles, FDA effectively bypasses the exacting procedural requirements that FDAMA and the GGPs impose on formal guidance document development. Some of these steps, particularly soliciting public comment and revising the guidance in response, may be time consuming and resource intensive.

Yet these procedural hurdles do not justify FDA’s departure from its statutory mandate. After all, Congress amended the Food, Drug, and Cosmetic Act to require the Agency to develop and abide by good guidance practices for good reason. Guidances serve as a way for FDA to communicate its thinking widely, ensuring that regulated industry understands and can provide feedback regarding the Agency’s expectations. Public participation fosters an open dialogue between FDA and its stakeholders, providing regulated entities an opportunity to meaningfully shape the Guidances that will apply to them. Guidance creation is meant to be an iterative process in which the finalised document reflects diverse input, rather than solely the Agency’s perspective, and thus may lead to better-informed and more effective policy. Last, and perhaps most importantly, like the APA, GGPs reflect broader democratic norms. Without these practices, policies may become a black box, representative of a select few rather than the many, undermining representative governance. FDA’s announcement of policy in journal articles likely contravenes its statutory obligations and is, at minimum, inconsistent with the broader democratic norms that good guidance practices are designed to uphold.

Looking ahead: what restricted-access policymaking means for regulated industry and the public

Regulated industry must either adapt to FDA’s current policymaking practices or call on the Agency to abide by procedural formalities. Adapting will require creative means of communicating regulated parties’ positions to FDA outside of traditional public comment afforded by guidance document procedures, such as formal meetings with the Agency, engaging third-party advocates on industry’s behalf, or the filing of citizen petitions. Alternatively, industry, or the public itself, may call on FDA to undertake the formal guidance development process and abide by good guidance practices. While FDAMA provides no private right of action against FDA to force the Agency to adhere to the Act’s enumerated legal obligations, 21 USC, section 371(h)(1)(D) provides for an internal appeals process ‘to address complaints that the Food and Drug Administration is not developing and using guidance documents’ appropriately.^[16] As another, more formal option, stakeholders could pursue a procedural claim under the APA; however, a claim based on an informal policy pronouncement like a journal article could face significant procedural hurdles.

Which approach is best may depend on the outcome of a recent leadership change at FDA. At the end of April 2026, Center for Biologics Evaluation and Research Director Vinay Prasad, a contributing author to all of FDA’s journal-article-based policy announcements, left the Agency. It remains to be seen whether FDA will revert to compliance with GGPs following his departure or whether disregarding these requirements will become a permanent fixture of FDA practice. Even so, while the plausible mechanism framework was ultimately published in a draft guidance, the other referenced reforms were not. Thus, regardless of Director Prasad’s departure, there is no foreseeable opportunity for public comment on the already proposed policies. Industry and consumers of regulated products must not stand idly by if FDA continues to disregard good guidance practices, for policy made without the participation of those it governs and those it protects is unlikely to serve either well.

Notes

^[1] Vinay Prasad & Martin A Makary, ‘FDA’s New Plausible Mechanism Pathway’, 393-23 NEJM (12 November 2025): www.nejm.org/doi/full/10.1056/NEJMsb2512695; FDA, Guidance Document, ‘Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause’ (February 2026): www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-plausible-mechanism-framework-develop-individualized-therapies-target-specific.

^[2] Vinay Prasad & Martin A Makary, ‘One Pivotal Trial, the New Default Option for FDA Approval – Ending the Two-Trial Dogma’, 394-8 NEJM (18 February 2026): www.nejm.org/doi/full/10.1056/NEJMsb2517623.

^[3] Vinay Prasad & Martin A Makary, ‘An Evidence-Based Approach to Covid-19 Vaccination’, 392-24 NEJM (20 May 2025): www.nejm.org/doi/full/10.1056/NEJMsb2506929.

^[4] Upendra Mahat et al, ‘Advancing CAR T-Cell Therapy – Evidence-Based Trial Design for Chimeric Antigen Receptor T-Cell Therapy in Oncology’, 335-1 JAMA (8 December 2025): https://jamanetwork.com/journals/jama/article-abstract/2842439.

^[5] JAMA and NEJM have varying public access policies. While the referenced articles may have been openly accessible for a brief period immediately upon publication, three of the four are currently behind a paywall. The Covid-19 vaccination regulation article is freely accessible.

^[6] 5 USC ss 551–559; Pub L No 105–115, 111 Stat 2296 (1997).

^[7] Pub L No 105-115, 111 Stat 2296 (1997).

^[8] Ibid.

^[9] Ibid.

^[10] 21 CFR s 10.115.

^[11] Ibid, s 10.115(c)(1).

^[12] Ibid, s 10.115(g).

^[13] Ibid.

^[14] Pub L No 87–781, 76 Stat 780 (1962).

^[15] 21 CFR s 10.115(e).

^[16] 21 USC s 371(h)(1)(D).