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# Guidance Answers More Expanded Access Questions
- URL: https://www.fdaweb.com/guidance-answers-more-expanded-access-questions/
- Published: 2025-11-20T12:00:00.000Z
- Updated: 2026-09-14T15:27:19.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160256

An FDA 10/22 guidance, *Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers*, was issued to address some concerns raised about a 2017 guidance, especially about its relationship to requirements in the 21st Century Cures Act and the FDA Reauthorization Act of 2017\. Attorneys **Laura Dona** and **Alan Minsk** (Arnall Golden Gregory) write in an online [post](https://www.jdsupra.com/legalnews/let-it-grow-fda-revises-q-a-guidance-to-4399947/?ref=fdaweb.com) (includes a link to the guidance) that the agency expects the new guidance will help industry understand expanded access requirements and inform patients and healthcare providers about how to obtain investigational drugs when no approved treatments exist.

The attorneys list these highlights from the new guidance:

- reassures the public that multiple safeguards are in place for expanded access use;
- addresses questions raised by industry about the impact of the 21st Century Cures Act and the FDA Reauthorization Act of 2017;
- outlines the two regulatory pathways and key requirements for submitting expanded access requests;
- summarizes expanded access request types, including waiting times and institutional review board requirements;
- clarifies that all expanded access requests are subject to informed consent requirements;
- reviews the criteria that doctors and FDA must use in determining the appropriateness of expanded use for individual patients;
- says that for intermediate-size patient populations and widespread use, sponsors can consolidate expanded access in a single IND or protocol;
- clarifies that treatment can begin once the sponsor has agreed to provide the drug and regulatory requirements are met;
- states that FDA will authorize expanded access only if it will not impede ongoing or planned clinical trials that could support marketing approval;
- notes that sponsors must follow all safety reporting rules for expanded access; and
- says that to comply with the Cures Act, sponsors must publicly post their expanded access policies.

Dona and Minsk make these recommendations:

- industry should leverage existing INDs and consolidate protocols whenever possible to streamline expanded access requests;
- sponsors should use the informed consent template in guidance Appendix B for individual patient expanded access;
- because FDA remains concerned that expanded access for large patient populations can risk slowing drug development, sponsors should provide detailed plans showing how trials and drug development will not be disrupted before seeking authorization;
- in their expanded access requests, sponsors should provide comprehensive data and support to ensure the agency and treating doctors can confidently determine that the investigational drug’s potential benefits outweigh the risks, that the patient faces a serious or life-threatening condition, and that no comparable or satisfactory therapeutic alternatives exist;
- in posting expanded access policies to comply with the Cures Act, sponsors should avoid language that could be interpreted as marketing or endorsing the drug for a particular use;
- sponsors should post expanded access policies by Phase 2 or 3 start or within 15 days of breakthrough, fast-track, or regenerative advanced therapy designation; and
- the agency hopes the guidance will allow expanded access programs to grow.