Track 40: Compassionate Use and Expanded Access Programs

Sub-Topics of Compassionate Use and Expanded Access Programs:

Definitions and Differences Between Expanded Access and Clinical Trials · FDA Expanded Access Types (Individual, Intermediate-size, Treatment IND) · EU Compassionate Use Procedures · Eligibility Criteria for Patients · Role of the Treating Physician and Sponsor Responsibilities · Regulatory Submission Requirements (IND/IDE, IRB, Informed Consent) · Ethics and Risk-Benefit Considerations · Manufacturing and Supply Chain Logistics · Monitoring and Adverse Event Reporting · Data Collection and Limitations · Drug Developer Policies on Access · Impact on Ongoing Clinical Trials · Pediatric and Rare Disease Access · Early Access vs. Named-Patient Programs · International Harmonization and Challenges · Legal and Liability Concerns · Patient Advocacy and Communication

Overview of Compassionate Use and Expanded Access Programs:

Compassionate Use (also known as Expanded Access) refers to regulatory pathways that allow patients with serious or life-threatening conditions to gain access to investigational drugs, biologics, or medical devices outside of clinical trials when no comparable alternatives are available.

These programs are intended for patients who are ineligible or unable to participate in a clinical trial and who have exhausted approved treatment options. The sponsor (typically a physician or healthcare institution) must obtain authorization from regulatory agencies such as the FDA (U.S.), EMA (EU), or local health authorities, and the use must comply with ethical and safety standards.

Expanded access programs provide real-world experience, may generate supportive safety and efficacy data, and are often considered a lifeline for patients with rare diseases, cancer, or advanced-stage conditions.