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

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.

This study includes a policy analysis to evaluate CUPs and NPBs in seven high-income European countries (Belgium, France, Germany, Netherlands, Norway, Switzerland, and the UK). We collected data on regulatory characteristics, including reimbursement aspects, from national health authority resources and direct consultations. In an in-depth examination, we assessed CUPs of anticancer and orphan medicines authorized in 2021 and 2022, focusing on availability, duration, and geographic distribution.

Our analysis reveals variability in national regulations, with inconsistent reimbursement options for CUPs and NPBs. For NPBs, reimbursement was often unregulated. The in-depth examination of CUPs revealed disparities in availability and duration before and after EU marketing authorization. We identified 36 CUPs, with 3-9 CUPs per country. Each CUP was available in up to four countries.

We recommend minimizing disparities between CUPs and NPBs across Europe to ensure equitable access for patients with high unmet medical needs. Reducing these differences is essential to protect patients from feeling compelled to travel abroad or bear the financial burden of obtaining medicines that are not authorized in their home country.