Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers

The CDER Small Business and Industry Assistance (SBIA) program is hosting an educational webinar focused on the FDA guidance “Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers.” The session explores the expanded access pathway, which allows patients with serious or immediately life‑threatening conditions to obtain investigational treatments outside of clinical trials when no comparable alternatives are available. The webinar aims to build a clear, practical understanding of the FDA’s regulatory framework and recommendations for expanded access.

Through expert presentations and a panel discussion, the event will cover key aspects of the expanded access program, including:

• The three categories of expanded access: individual patient, intermediate‑size population, and treatment INDs or protocols
• FDA submission, review, and reporting requirements
• Common questions, misconceptions, and available tools and resources such as forms and templates

Designed for a broad audience, including drug sponsors, researchers, healthcare professionals, regulators, and patient advocates, the webinar provides guidance to help stakeholders support safe, compliant, and timely access to investigational treatments for patients in need.