An Industry Perspective on Compassionate Use in Europe: A Call for Change.

Area of Science:

• Pharmacoeconomics and Health Policy
• Regulatory Science
• Patient Access to Medicines

Background:

• Compassionate use (CU) programs for unauthorized medicines in Europe exhibit significant variability.
• Two main pathways exist: EU-regulated cohort programs and member-state-regulated individual patient requests (IPRs).
• Disparities in national laws create operational challenges and potential delays for patients seeking CU medicines.

Purpose of the Study:

• To compare the availability and operational efficiency of compassionate use programs across 30 European countries.
• To identify key challenges and variations in the implementation of CU pathways.
• To provide data-driven recommendations for improving CU access and harmonization.

Main Methods:

• An exploratory post-hoc analysis of 8,934 compassionate use requests from 30 European countries.
• Data were collected from four major pharmaceutical companies (Merck KGaA, Novartis, Roche, Sanofi) between January 2020 and April 2023.
• Included requests with complete submission, company approval, regulatory, and shipment dates.

Main Results:

• Internal company approval times were similar for cohort programs (median 5 days) and IPRs (median 4 days).
• Time from company approval to medicine shipment varied significantly: median 5 days for cohorts vs. 8 days for IPRs.
• Key challenges included inconsistent terminology, scope, regulatory, and operational requirements across countries.

Conclusions:

• Differing national requirements in Europe create operational hurdles and variability in compassionate use implementation.
• These inconsistencies pose significant challenges for stakeholders involved in providing patient access to medicines.
• There is a clear need for greater harmonization of compassionate use regulations and processes across European member states.