FDA Clarifies Expanded Access Policy and Transparency Obligations

The October 2025 update to the US Food and Drug Administration (FDA)  Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers (Revision 1) provides guidance on expanded access, a pathway that allows patients with serious or life-threatening conditions to potentially access investigational treatments when standard therapies are exhausted. The revision replaces the 2017 version and integrates provisions from the 21st Century Cures Act and the FDA Reauthorization Act (FDARA) of 2017. It creates a unified framework that clarifies expectations for sponsors, investigators, and regulators involved in expanded-access programs.

The updated guidance unifies past requirements and emerging expectations to provide clarity for regulators, consistency for sponsors, and transparency for patients.

Regulatory and procedural updates

The guidance reaffirms the three pathways for treatment use and explains their submission and review processes:

• individual patient, including emergency use
• intermediate-size populations
• treatment Investigational New Drug (IND)/protocols for broader use

Beyond defining access pathways, the guidance clarifies expectations for instituitional review board (IRB) review and emergency-use notifications and includes an informed-consent template designed to support clear, consistent patient communication.

Sponsor responsibilities and timing

FDA reiterates statutory obligations under the 21st Century Cures Act  — §561A of the US federal Food, Drug, and Cosmetic Act (FD&C) Act) — and FDARA 2017.

Manufacturers and distributors of investigational drugs for serious or life-threatening conditions must make their expanded-access policies publicly available, such as on a company website or other public platform. Policies should describe how requests are submitted and reviewed, the expected acknowledgment timeframe, and a link to an existing ClinicalTrials.gov record (as applicable).

Policies must be made available before the first Phase II or III trial begins or within 15 days of receiving a fast-track, breakthrough, or regenerative-therapy designation. While these requirements originate in statute, the guidance offers practical examples to help sponsors meet them and maintain consistency across regulatory and GCP oversight activities.

Transparency and public discoverability

The guidance emphasizes how these policies and records connect to the broader transparency landscape. Public visibility of expanded-access information is achieved through:

• Publicly available sponsor policies, which describe how access requests are handled
• ClinicalTrials.gov, where sponsors indicate when expanded access is available for a registered IND
• The Reagan-Udall Foundation’s Expanded Access Navigator, which compiles company policies and registry listings in a central, searchable directory

Together, these channels make it easier for patients, physicians, and regulators to identify available treatment-use options and ensure consistent, accessible information across public sources.

Safety reporting and oversight

Expanded-access INDs and protocols remain subject to safety reporting (§ 312.32) and annual reports (§ 312.33). The FDA notes that while expanded-access data rarely alter approval decisions, they can contribute to post-market safety understanding.

Takeaway for disclosure and clinical professionals

For disclosure and regulatory teams, the 2025 guidance translates existing law into clearer operational terms that reinforce public posting, cross-referencing, and patient-facing transparency as integral components of compliance. For clinical and patient-engagement leaders, it establishes consistent expectations for ethics, communication, and data stewardship across all treatment-use scenarios.

Read the full guidance: Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers (Revision 1, October 2025)