The Expanded Access Program (EAP), sometimes called Compassionate Use Program (CUP) in Europe, is a regulatory mechanism that allows patients with serious or life-threatening diseases to access investigational medicines before marketing authorization (MAA) when no satisfactory alternatives exist.

It operates under a European framework (EMA guidance, Article 83 of Regulation (EC) No 726/2004), but implementation is national, meaning each EU member state manages access according to its own procedures.

 

Key Takeaways

  • Europe has a common legal basis (Article 83), but implementation is decentralized to each member state.
  • EMA provides recommendations for multinational compassionate use, but national agencies grant access.
  • Programs differ: France (AP1/AP2 via ANSM), Germany (Compassionate use via BfArM), Finland (Special Permit), Sweden/Denmark/Spain/Italy (Compassionate Use), Portugal (Exceptional Use Authorization), UK (EAMS, post-Brexit)…
  • Used primarily as a bridge for patients before full market authorization.

 

Sources

  1. EMA – Compassionate use (legal basis, Article 83)
    https://www.ema.europa.eu/en/human-regulatory/research-development/compassionate-use
  2. European Commission – Regulation (EC) No 726/2004 (Article 83)
    https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32004R0726
  3. EMA – Guidance for companies on compassionate use
    https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-compassionate-use_en.pdf
  4. Scientific review – Expanded access in Europe (BMJ / Orphanet)