Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.

ABSTRACT:

Background

In the European Union (EU), anticancer and orphan medicines are often granted marketing authorization based on surrogate endpoints and limited clinical trial data. Driven by unmet medical needs and the urgency of providing access beyond clinical trials, there is growing interest in early access, such as compassionate use programs (CUPs) and named patient basis (NPB). Additionally, limited clinical evidence can hinder health technology assessments and, when combined with high costs, delay reimbursement negotiations and patient access. Hence, it is crucial to explore CUPs and NPBs including pricing and reimbursement aspects.

Design

This study includes a policy analysis to evaluate CUPs and NPBs in seven high-income European countries (Belgium, France, Germany, Nethe

SUBMITTER: Rosenberg N 

PROVIDER: S-EPMC12661435 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature