Expanded Access to Investigational Drugs for Treatment Use - Questions and Answers
Why It Matters
By tightening procedures and enhancing transparency, the guidance speeds access to life‑saving investigational therapies while ensuring patient safety, a critical balance for innovators and regulators alike.
Key Takeaways
- •FDA's 2025 guidance updates expanded access pathways for investigational drugs.
- •Three access categories: individual, intermediate-size, and treatment IND protocols.
- •Form 3926 and eRequest streamline physician‑submitted individual patient requests.
- •IRB review required except emergency use; waivers reduce administrative burden.
- •Sponsors must publicly post access policies and meet ongoing safety reporting.
Summary
The FDA released a 2025 guidance document that revises the expanded‑access program for investigational drugs, outlining how patients with serious or immediately life‑threatening conditions can obtain treatment when no approved therapy exists.
The guidance defines three regulatory categories—individual patient, intermediate‑size patient population, and treatment IND/protocol—each with specific eligibility criteria and submission pathways. It clarifies when manufacturers versus licensed physicians act as sponsors, and details the required forms, including the streamlined Form 3926 and the eRequest web app for non‑emergency individual requests.
A key operational change is the ability to obtain an IRB waiver for full‑board review on individual non‑emergency requests, simply by checking a box on Form 3926. The document also mandates that manufacturers post their expanded‑access policies publicly within 15 days of a phase‑2/3 start or breakthrough‑therapy designation, listing contact information, evaluation criteria, and response timelines.
These updates reduce administrative delays, increase transparency, and impose clearer safety‑reporting obligations, enabling faster patient access while preserving oversight. Pharmaceutical companies, clinicians, and patient advocates must adjust their processes to comply, potentially accelerating treatment options for high‑need patients.
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