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The Food and Drug Administration is proposing to amend 21 CFR 312 Subparts A, B, C, D and 21 CFR 56, for expedited Investigational New Drug (IND) reform. The proposed rule would make changes to general provisions related to the IND requirements, including process and IND content and format and sponsor responsibilities. 

Statement of Need:

FDA is proposing this action to modernize and streamline the Investigational New Drug (IND) application process for Phase 1 clinical trials by reducing unnecessary regulatory burden through targeted, risk-based flexibilities. The action is needed to accelerate patient access to promising investigational therapies while maintaining appropriate human subject protections. By facilitating earlier clinical development of innovative drugs, this rule is expected to reduce delays that can adversely affect patient health outcomes and to address regulatory risks that are disproportionate to the low-risk nature of many early-phase studies. This type of reform is also a do out of the MAHA Commission as part of the White House’s Make Our Children Healthy Again: Strategy Report (September 2025) and aligned with the Administration’s deregulatory efforts.

Summary of the Legal Basis:

FDA’s authority lies under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act, including 21 U.S.C. sections 321, 331, 351 to 355, 360bbb, and 371, and 42 U.S.C. section 262, to revise IND and IRB requirements for Phase 1 clinical trials. The proposed rule is intended to support risk-based regulatory modernization consistent with recent Executive Orders on deregulatory reform and innovation in healthcare.

Alternatives:

FDA considered maintaining the current IND framework without modification, as well as implementing narrower administrative guidance instead of formal rulemaking. The Agency also considered more limited exemptions applicable only to specific therapeutic categories but determined broader risk-based flexibilities would better reduce unnecessary burden while preserving appropriate human subject protections, especially given Administration interest in these reforms.

Anticipated Costs and Benefits:

FDA is proposing to amend 21 CFR 312 Subparts A, B, C, D and 21 CFR 56, for expedited Investigational New Drug (IND) reform. The proposed rule would make changes to general provisions related to the IND requirements, including process, content, format and sponsor responsibilities. FDA anticipates benefits from this rule emanating from loosening requirements. This streamlining for the use of certain investigational drugs for Phase I clinical trials through targeted and risk-based exemptions to speed the access of investigational drugs to patients would decrease regulatory burden, which may help accelerate access to transformative treatments for patients, which may lead to improved health outcomes. We anticipate costs of this rule would include reading and understanding what new flexibilities would be afforded to sponsors and any potential safety risks of loosening current requirements. 

Risks:

Potential risks include inconsistent sponsor interpretation of new flexibilities, and the possibility that streamlined requirements could increase safety concerns or data quality issues in low-risk studies while firms adjust. FDA expects these risks to be mitigated through existing Institutional Review Board review, sponsor responsibilities, and continued FDA monitoring authority, as well as sponsor education and early engagement.