Factors influencing pharmaceutical companies’ decisions to pursue compassionate use programs in the EU: a qualitative study in The Netherlands

Background: Access to unauthorized medicines in the EU is legally restricted, except in certain cases such as clinical trials, magistral preparations, hospital exemptions, and early access programs, including compassionate use programs (CUPs) and named patient use (NPU). CUPs, regulated under Article 83 of Regulation (EC) No 726/2004, are intended for a group of patients with an unmet medical need. Despite this EU-wide regulation, the implementation of CUPs varies among member states, and the factors driving pharmaceutical companies to pursue them are poorly understood. Methods: This study conducted semi-structured interviews with pharmaceutical companies that had applied for CUPs in the Netherlands, as well as those with potentially eligible medicines that had not pursued CUPs. The interviews explored the decision-making processes and factors influencing CUP applications. Transcripts were analyzed using Atlas.ti software, with coding categories derived from the interview guide and emerging themes. Results: Ten interviews were conducted. Factors influencing CUP applications were classified into four categories: regulatory, medical, operational, and financial. Regulatory factors included recommendations from the Health and Youth Care Inspectorate (IGJ) and European Medicines Agency (EMA), concerns about post-marketing authorization uncertainties, and timelines for CUP approval. Medical factors involved unmet medical needs, patient numbers, and the alignment of CUP indications with authorized indications. Operational factors included prior experience with CUPs, supply availability, and the appeal of NPU due to faster approval times. Financial factors centered on reimbursement expectations and decisions by company headquarters on the free provision of medicines. Conclusion: The decision to pursue CUPs is influenced by multiple factors, with regulatory uncertainties and operational complexities playing significant roles. Improving clarity concerning CUP regulations, particularly data collection and the post-marketing phase, could encourage more pharmaceutical companies to apply for CUPs, which would provide patients with earlier access to potentially promising treatments.