Exploring Expanded Access: Balancing Patient Needs and Regulatory Considerations

Session Description

Expanded access, also known as compassionate use, refers to the regulatory pathway that allows patients with serious or life-threatening conditions access to investigational drugs outside of clinical trials when no satisfactory alternative treatment is available. These programs serve as a lifeline for patients who have exhausted standard treatment options and are seeking access to potentially beneficial therapies still under investigation. The decision to grant expanded access is typically based on several factors, including the patient's medical condition, the potential benefit of the investigational drug, and the assessment of risks versus benefits. Expanded access can take different forms, such as single-patient access, intermediate-size patient populations, or widespread access under a treatment IND (Investigational New Drug) or other regulatory mechanisms.

While expanded access provides hope for critically ill patients, it also raises ethical, logistical, and regulatory challenges. Balancing patient access with the need for rigorous clinical data collection, ensuring patient safety, and managing supply chain issues are key considerations. In this session, we will delve into the complexities surrounding expanded access, examining the ethical, regulatory, and practical aspects of providing investigational therapies to patients outside of traditional clinical trial frameworks. Our expert panel will explore the challenges and opportunities associated with expanded access, highlighting best practices and potential solutions to optimize patient access while maintaining scientific rigor and regulatory compliance.

Learning Objectives

1. Understand the ethical, regulatory, and logistical challenges in Expanded Access Programs.
2. Highlight the best practices and potential solutions for optimizing patient access while ensuring safety and scientific rigor.
3. Discuss challenges faced by patients in accessing investigational therapies through expanded access and how patient advocates and stakeholders can collaborate to improve this access.

Session Schedule

Introduction
Mariam Ahmed, PhD and Daria Stypinski, PhD

Patient-Centric Approaches in Compassionate Use: Patient Advocacy Perspectives
Mira (Mitze) Klingenberg, BS, RN

Optimizing Patient Access through Compassionate Use: Navigating Regulatory Considerations and Compliance
Mwango Kashoki, MD, MPH

Industry Perspectives on the Compassionate Use Program: Clinical Pharmacology Considerations and Beyond
Indranil Bhattacharya, PhD

Q&A and Closing Remarks