Presentation (CTI)

OBJECTIVES: A Marketing Authorisation (MA) granted to a medicinal product does not guarantee its local availability. The access to medicines can be made through early access programs (EAP) and pricing and reimbursement (P&R) agreements at a national level. This study aimed to characterize access to Orphan Medicines (OM) by the mechanisms of EAP or P&R in Portugal. We compare availability of OM to Rare Disease Action Plan goal for 2025.
METHODS: Orphan Medicines MA was extracted from the European Medicines Agency (EMA) database [1] on the 17th October of 2025. Additional information on country level registration, commercialization (marketed) and availability was gathered from the Portuguese Medicines database (INFOMED/INFARMED) [2] and INFARMED P&R [3] and EAP [4] public decisions. Data sources were merged by the name of medicine regardless the number of approved therapeutic indications. Counts and proportions were used to describe the characteristics of interest.
RESULTS: In total, 1509 medicines were centrally authorized, 10% of authorized which were OM. The type of MA granted to OM were standard authorizations in 73% of the cases, 12% were granted under Exceptional circumstances and 15% of the MA were granted under Conditional approval. From all the MA granted to OM, 66% were associated with the need of additional monitoring, 17% were considered priory medicine (PRIME), 9.5% were considered advanced therapy and 8.9% were associated with accelerated assessment at EMA level. Of the EMA approved OM, 37% are currently public reimbursed in Portugal and 23% are available through EAP.
CONCLUSIONS: Only one third of the EMA authorized orphan medicines are currently public financed in Portugal and one-fifth are accessible through early access programs in the country. Ambition of ensuring equitable access to 50% of EMA authorized orphan medicines by December 2025 seams elusive, unless current reimbursement patterns dramatically change toward the end of 2025.