OBJECTIVES: Equal and timely access across Europe remains a long-standing challenge for orphan pediatric medicinal products, despite the existence of specific regulatory framework in the EU. The aim of the present study was to monitor access to orphan medicinal products (MPs) for children and adolescents after the establishment of the national HTA and Reimbursement Committee (January 2018-December 2021) in Greece.

METHODS: Data were collected from the European Medicines Agency (EMA) and the Greek Ministry of Health websites, as well as from the Health Technology Assessment (HTA) Committee’s database. The dataset included MPs with a clear reference in their Summary of Product Characteristic (SPC) to pediatric and/or adolescent use. Data on the orphan status and the therapeutic category (ATC3) of the active substances were also collected. Median times for MPs with a pediatric/adolescent indication and according to their orphan status were calculated based on their marketing authorization date, their dossier submission to the Greek HTA Committee and their inclusion in the Positive Reimbursement List.

RESULTS: Over the 4-year study period, from 54 MPs with orphan pediatric/adolescent indication submitted to EMA, 13 with orphan designation were submitted to the national HTA and Reimbursement Committee. The median time (25^th – 75^th percentile) from marketing authorization to national HTA submission was 365 (246-508) days for pediatric/adolescent MPs in general and 393 (203-521) days for orphan pediatric/adolescent MPs specifically. The majority of pediatric/adolescent MPs referred to immunosuppressants (ATC3: L04A) while orphans pertained to other respiratory system MPs (ATC3: R07A).

CONCLUSIONS: Delays in access to pediatric/adolescent MPs may be enhanced by the presence of orphan status. Forthcoming changes in the European pharmaceutical legislation should consider reforms and incentives for pediatric/adolescent therapies.