OBJECTIVES: Early access programs (EAP) are needed, as long as pharmaceuticals are not approved or commercially available in a certain jurisdiction. They address a patient need and can be a relevant strategy for market access. Legal background is Article 83 of EU Regulation 726/2004. This analysis sets out to explore if and which common denominators exist for EAP within the EU and what are the potential implications for market access.

METHODS: For this analysis current state of EAP regulation and products under EAP for Spain, Italy, France, Germany, and Austria have been reported by respective country experts, as of June 2023. Focus is on program on cohorts.

RESULTS: There are two main EAP for patient cohorts without therapeutic alternatives in Italy. In compassionate use programs (CUP), the product is provided for free by the pharmaceutical company (currently 81 products). Law 648/1996 reimburses the use of certain drugs based on a price negotiated with the NHS (currently 150). All programs require approval from AIFA and require a therapeutic protocol.

In Spain early access is limited to case-by-case decisions; programs for cohorts do not exist.

Like Italy, France has an extensive EAP. Decision is based on a submission by the pharmaceutical company showing medical need, efficacy and safety and presumption of innovation. Product use is reimbursed and price during EAP is determined by the pharmaceutical company; therapeutic protocol and data collection are mandatory with 74 products currently under EAP.

Contrary to France, in Austria and Germany CUPs are not reimbursed. They are also in time, but therapeutic protocol and data collection again are mandatory. 28 products are currently under CUP in Germany and 3 in Austria.

CONCLUSIONS: Even though EAPs are regulated by European law, each country follows different approaches towards EAP. There is no such concept as a common EU EAP strategy.