An Expanded Access Program (EAP) — also known as Compassionate Use — is the United States regulatory mechanism that allows patients with serious or life-threatening diseases to access an investigational medicine outside of clinical trials, when:
- No satisfactory treatment alternatives exist, and
- The patient is not eligible to participate in a clinical trial.
EAPs address urgent unmet medical needs while maintaining oversight on safety and data collection. They are not substitutes for clinical trials, but operate in parallel to provide treatment options for patients in critical conditions.
Key Features of Expanded Access Programs (United States)
- Purpose: Provide access to potentially life-saving therapies before FDA approval.
- Eligibility: Serious or rare diseases, no alternatives, and no trial eligibility.
- Regulator: FDA, with specific provisions under Expanded Access rules and the “Right to Try” pathway.
- Forms of Access:
- Individual/named patient access
- Cohort/group access (company-led programs)
- Data obligations: Safety data must be collected and reported; limited efficacy data may also be gathered.
- Pricing: Cost-recovery pricing allowed under FDA rules.
Professional Relevance
- Enables early patient access to innovative therapies.
- Generates real-world safety/efficacy insights that may complement clinical trial data.
- Serves as a bridge to market authorization and reimbursement decisions.
Important distinction
- United States: The official term is Expanded Access Program (EAP).
- European Union: The equivalent concept is called Compassionate Use (Article 83 Regulation (EC) 726/2004) and is implemented nationally.
- France: The framework is AAP (Accès Précoce) and AAC (Accès Compassionnel).
- See: What is the Expanded Access Program (EAP) in Europe?
Sources
- EMA – Compassionate Use (Expanded Access in EU)
https://www.ema.europa.eu/en/human-regulatory/research-development/compassionate-use - European Commission – Regulation (EC) No 726/2004, Article 83
https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32004R0726 - FDA – Expanded Access
https://www.fda.gov/news-events/public-health-focus/expanded-access - BMJ – Expanded Access in Clinical Practice
https://www.bmj.com/content/371/bmj.m4050 - Orphanet Journal of Rare Diseases – Expanded Access Review
https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0705-y
