Résumé
The thesis analyzes the orphan medicinal products authorized in Europe between January 1st, 2018 and March 31st, 2021 and in particular the maintenance of orphan status at time of marketing authorization application procedure. The analysis includes a characterization of the criteria used to demonstrate the significant benefit. For this period, 52 orphan medicinal products were authorized covering 60 orphan designations. The analysis is based on 2 publicly available documents which are published for each authorized orphan medicinal product, namely the “European Public Assessment Report” (EPAR) and the “Orphan Maintenance Assessment Report” (OMAR). The analysis shows that the prevalence reported in 62% of orphan status maintenance procedures is less than or equal to 1 patient per 10,000 people. Also, the applicant was invited by the Committee for Orphan Medicinal Products (COMP) for an oral explanation in 28% of the procedures. Moreover, in 25% of the procedures, no satisfactory method existed, so the applicant did not have to demonstrate any significant benefit. For the other procedures, the significant benefit was demonstrated in 91% of the cases using the domain “Clinical relevant benefit” (CRA), only 2 procedures used the domain “Major contribution to patient care” (MCPC) and 2 other procedures used the combination of CRA and MCPC domains. The 2 domains are composed of criteria and sub-criteria that can be used alone or in combination to demonstrate the significant benefit. The use and distribution of these criteria and sub-criteria in the orphan maintenance procedures are detailed in the thesis.