FDA panel outlines 2025 expanded‑access guidance, clarifies emergency rules and manufacturer posting obligations

The Food and Drug Administration on a webinar presented its 2025 guidance on expanded access to investigational drugs, detailing the regulatory pathways clinicians and companies must follow to provide treatment outside clinical trials.

Dr. Atasi Padar, health science policy analyst in the Division of Medical Policy Development at FDA, said the guidance has three aims: explain pathways for providing investigational drugs to patients with serious or immediately life‑threatening diseases who lack satisfactory alternatives; clarify regulatory requirements and FDA recommendations for the expanded access program; and update the 2017 guidance to reflect recent statutory changes and questions from stakeholders. "This guidance is essential reading for anyone involved in making investigational drugs available to patients outside of clinical trials," she said.

The guidance defines expanded access as treatment‑focused use of an investigational drug, biological product or device distinct from clinical trials, which have the primary purpose of systematic data collection. FDA identified three categories: individual patient expanded access (including emergency and non‑emergency subcategories), intermediate‑size patient population expanded access, and treatment INDs or protocols for larger or widespread patient populations.

Panelists described the submission options and tools. Companies may submit a new expanded access IND or an expanded access protocol as an amendment to an existing IND; licensed physicians may submit single‑patient INDs and may use Form 3926 (a streamlined form) or the eRequest web app for non‑emergency submissions. Commercial sponsors and protocols generally use Form FDA 1571. Dr. Padar noted that submissions must include the rationale, clinical history, treatment plan, chemistry/manufacturing and toxicology information and any category‑specific materials required under 21 CFR 312.305(b).

The panel clarified timing and IRB requirements. Emergency individual patient use may be authorized orally (phone or electronic communication) and requires a written submission to FDA within 15 working days and IRB notification within five working days. For non‑emergency individual and intermediate INDs, there is typically a 30‑day waiting period after FDA receipt unless the agency authorizes earlier; protocols submitted as amendments to an existing IND may allow treatment to begin once the protocol is submitted and IRB approval is obtained. FDA also described a common efficiency: IRB chair concurrence in lieu of full board review may be allowed (and can be requested via Form 3926 box 10B).

On responsibilities, the guidance reiterates sponsor obligations for IND safety reporting (reporting serious, unexpected suspected adverse reactions), annual reports for treatments that continue a year or more, and final summaries at the conclusion of individual‑patient treatments. Dr. Padar emphasized that sponsors must maintain drug disposition records and withdraw expanded access INDs when the program ends.

The webinar addressed common confusion about authority and access. "The answer is no. FDA cannot require a sponsor to provide expanded access to its drug," Dr. Padar said, adding that sponsors grant access voluntarily even when they post a publicly available expanded access policy. The guidance restates statutory posting obligations from the 21st Century Cures Act and the FDA Reauthorization Act: manufacturers or distributors must post an expanded access policy by specified triggers (for example, at first initiation of a Phase 2 or Phase 3 trial or within 15 days after designation as breakthrough therapy, fast track or regenerative advanced therapy). That policy must include contact information, submission procedures, general evaluation criteria, an acknowledgement time frame and a link or reference to the clinicaltrials.gov record. The panel cautioned that posting a policy does not guarantee that a specific request will be granted.

Panelists answered clinician questions during a broad Q&A. Key clarifications included:
• Emergency use: FDA maintains off‑hours mechanisms and a staffed emergency call center; initial authorization can be immediate with written follow‑up required. "There are phone numbers and email addresses for reaching the right office," an FDA official said, providing an emergency line for off‑hours contact.
• Letters of authorization (LOAs): Manufacturers that agree to supply product under single‑patient INDs should provide patient‑specific LOAs; each single‑patient IND receives its own IND number.
• Charging: Sponsors may charge patients for investigational drugs only with prior FDA approval and an appropriate justification for cost recovery; FDA has a separate guidance on charging updated in 2024.
• International scope: FDA cannot authorize expanded access in foreign countries; patients outside U.S. jurisdiction must work with their local regulatory authorities.
• Interference with trials: FDA considers potential impact on clinical trial enrollment when authorizing broader access, particularly for rare disease development; sponsors may be asked for additional information to demonstrate that expanded access will not interfere with clinical development.

The webinar also highlighted Project Facilitate, an FDA initiative that provides consultation and support for investigational oncology products; Mitchell (Mitch) Chan, the project lead for oncology, said the program offers assistance to physicians and regulatory professionals navigating expanded access requests.

The session closed with links to the 2025 guidance, the 2023 IRB guidance for individual patient expanded access submissions, FDA’s expanded access web page, template informed‑consent wording and example authorization and waiver letters. The recording and supporting materials will be posted on the FDA SBIA web page; certificates of attendance are available on request.

The guidance and the panel’s clarifications aim to reduce administrative burden while preserving safeguards for patients, including additional protections for children. Clinicians and sponsors were urged to consult the full guidance and contact the appropriate FDA review division with specific case questions.