The European Union regulation for orphan medicinal products (OMPs) was introduced to improve the quality of treatments for patients with rare conditions. To mark 20 years of European Union OMP regulation, this study compared access to OMPs and the length of their reimbursement process in a set of European countries and Canadian provinces. Access refers to their full or partial reimbursement by the public health service.

Data were collated on European Medicines Agency orphan designation and marketing authorizations, health technology assessment (HTA) decisions and reimbursement decisions, and the respective dates of these events for all the OMPs centrally authorized in 14 European countries (Belgium, England, France, Germany, Hungary, Italy, the Netherlands, Norway, Poland, Scotland, Slovakia, Spain, Sweden, and Switzerland) and four Canadian provinces (Alberta, British Columbia, Ontario, and Quebec).

Since the implementation of the OMPs Regulation in 2000, 215 OMPs obtained marketing authorization. We found that Germany had the highest level of coverage, with 91 percent of OMPs being reimbursed. The three countries with the lowest reimbursement rates were Poland, Hungary, and Norway (below 30%). We observed that Germany had the quickest time to reimbursement following marketing authorization, followed by Switzerland and Scotland. We observed that Poland, Hungary, and Slovakia consistently had the longest time to reimbursement.

We observed substantial variation in the levels and speed of national reimbursement of OMPs, particularly when comparing countries in Eastern and Western Europe, which suggests that an equity gap between the regions may be present. The data also indicated a trend toward faster times to reimbursement over the past 10 years.