Early Access Programs in Northwest European Countries

OBJECTIVES: Early access programs (EAPs) refer to patient access to medicines before marketing authorization, potentially extending to price and reimbursement approval. Most European countries have implemented EAPs following the EU legislative/regulatory framework for early access of innovative products at the national level, including compassionate use (CU), named-patient basis access (NP), and expanded access (options arising from participation in clinical trials). However, international research indicates that there are more differences than similarities among national EAPs. This study aimed to compare the EAPs for innovative medicines and their utilization and implementation in Germany, France, the United Kingdom, Sweden, Denmark, and Norway.

METHODS: A mixed-method analysis was employed, incorporating a systematic literature search, semi-structured interviews, and descriptive statistics. Interviews were conducted with national experts from the selected countries, and statistics on the number of EAPs were gathered from national authorities.

RESULTS: All the countries have national EAPs, however, the lack of standardization and common terminology poses challenges for comparison. Differences were observed in the utilization of programs, potentially depending on the funding sources. The main differences include the types of programs implemented, who pays for the pharmaceuticals, the existence of external funding opportunities, whether data collection for health technology assessment (HTA) purposes occurs, the degree of centralized/decentralization, and whether programs could continue post-marketing.

CONCLUSIONS: Significant differences exist between the countries' EAPs, which can create unequal access to new innovative medicine for patients across national borders.