Under the Orphan Regulation, the European Medicines Agency (EMA) intended to incentivize the research and development of new treatments for rare and life-threatening conditions. Marketing authorisation of orphan medicinal products (OMPs) by the EMA is only the first step, as medicines are made available to patients when reimbursement or Health Technology Assessment (HTA) decisions are implemented by national health systems. We analyzed the availability and access to OMPs in the United Kingdom (UK), France, Germany, Italy and Spain. We compared the availability, which is the possibility to prescribe a given OMP, to the access, which refers to the full or partial reimbursement by the public health service.

We collected data on launches, HTA decisions, any centralized commissioning and/or reimbursement decision for all the OMPs authorised since 2000 in the UK countries (England, Scotland and Wales), France, Germany, Italy and Spain.

Since the Orphan Regulation inception, the EMA granted marketing authorization to 143 OMPs. These OMPs are most widely accessible in Germany and France. Reimbursement in Germany is immediate after authorization. France and Italy present a delay of 19 months from authorization to reimbursement, which is shorter than in other countries. In England, less than 50 percent of centrally authorised OMPs are routinely funded by the National Health Service (NHS), including one-third of these recommended by the National Institute for Health and Care Excellence (NICE), and those reimbursed via commissioning policies and the Cancer Drugs Fund.

The assessment of degree of access to OMPs across Europe is limited by differences in the national HTA and reimbursement systems and the heterogeneous information made publicly available on their decisions. Nonetheless, our study suggests that the primary purpose to grant equal availability to OMPs to the patients in the Eropean Union via the implementation of the orphan regulation was partially achieved with important variations of access observed across the countries included in our study.