OBJECTIVES: Orphan medicinal products (OMPs) registered by the European Medicines Agency (EMA) are characterized by considerable heterogeneity. We decided to analyze OMPs by Anatomical Therapeutic Chemical (ATC) classification and therapeutic area and see how these classifications correlated with reimbursement decisions in selected European countries.

METHODS: The study was conducted between 2021 and 2022. Data on ATC classification and therapeutic areas for OMPs were collected from the EMA's web-based registry, while information on reimbursement was collected by a questionnaire survey; experts from 11 countries were included in this analysis: Belgium; Finland; France; Germany; Greece; Iceland; Italy; Netherlands; Scotland; Spain and Poland.

RESULTS: As of September 2022, there were 136 OMPs in the EMA web-based registry. OMPs with classification ATC L (antineoplastic and immunomodulating agents) were the largest group representing 36% (n=49) and the smallest group were those with classification ATC P (antiparasitic products, insecticides, and repellents) representing 0.7% (n=1) of all medicines. OMPs with ATC L received an average 62% reimbursement in the countries surveyed (p<0.001). A OMP with ATC L increased the chance of reimbursement by 3-fold compared to the other ATC classifications in the countries surveyed (p=0.026). The most common therapeutic areas of OMPs were: leukemia, lymphoma, multiple myeloma (each disease had 8 dedicated OMPs) and tuberculosis (4 dedicated OMPs), but one medicine may have had more than one indication. The six OMPs were reimbursed in all studied countries: daratumumab (multiple myeloma; ATC L); pomalidomide (multiple myeloma; ATC L); carfilzomib (multiple myeloma; ATC L); gemtuzumab ozogamicin (leukemia; ATC L); cabozantinib (leukemia; ATC L) and macitentan (hypertension and related; ATC C – intended for cardiovascular system).

CONCLUSIONS: Medicine classifications indicate that OMPs for oncological diseases represent the largest group among medicine types and therapeutic indications, increasing the chances of reimbursement, which translates into high reimbursement rates for these pharmacotherapies in Europe.