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An overview of Compassionate Use Programs in the European Union member states

Gayathri Balasubramanian1, Suman Morampudi1, Pankdeep Chhabra1

  • 1FSRC, Part of phamax, Bangalore, India.

Intractable & Rare Diseases Research
|December 2, 2016
PubMed

Insights

Compassionate Use Programs (CUP) offer patients earlier access to novel therapies when clinical trials are not an option. Most EU member states have established CUP regulations, facilitating access to new medicines.

Area of Science:

  • Pharmaceutical Medicine
  • Health Policy

Background:

  • Novel drug development has accelerated, but marketing authorization can be lengthy, delaying patient access.
  • Clinical trials are the traditional route for early drug access, but eligibility criteria exclude many patients, especially those with rare or severe diseases.
  • Early Access Programs (EAPs), such as Compassionate Use Programs (CUPs), provide alternative pathways for patients needing timely therapeutic interventions.

Approach:

  • This literature review systematically examined the status and legislation of CUPs across all 28 European Union (EU) member states.
  • Data collection involved a comprehensive review of scientific literature, grey literature, and country-specific healthcare search terms.
  • The review focused on identifying the presence of CUPs and the nature of their national regulations and defined processes.

Key Points:

  • Compassionate Use Programs (CUPs) are operational in 20 out of 28 EU member states (71%).
  • Nationalized regulations and well-defined processes for CUPs exist in 18 EU member states (approximately 64%).
  • The study provides a comprehensive overview of CUPs, their current status, and legislative frameworks within the EU.

Conclusions:

  • The widespread establishment of CUP legislation indicates a European commitment to improving early access to innovative medicines.
  • Further research and regular reviews are essential to track evolving regulatory trends in EAPs.
  • Enhanced understanding of CUPs can help optimize patient access to potentially life-saving treatments.
Keywords:
Compassionate useEuropean UnionEuropean Union member statesearly accessorphan drugsrare diseasesspecial access

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