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 explore the proportion of Orphan Medicines (OM) centrally authorised by European Medicines Agency (EMA) that have P&R or have had an EAP in Portugal.
METHODS: The data was extracted from the EMA database and then filtered in order to only consider centrally authorised human medicines. Furthermore, the INFARMED’s P&R and EAP databases were consulted. The proportions of OM with P&R and/or EAP were then calculated.
RESULTS: In total, 1477 medicines were centrally authorised, 10% of authorised which were OM. The type of MA granted to OM were standard authorisations in 74% of the cases, 13% were granted under Exceptional circumstances and 13% of the MA were granted under Conditional approval. From all the MA granted to OM, 65% were associated with the need of additional monitoring, 16% were considered priory medicine (PRIME), 10% were considered advanced therapy and 9% were associated with accelerated assessment at EMA level. Of the EMA approved OM, 38% are currently public financed in Portugal and 23% have had an EAP authorised. In Portugal, 77% of the authorized EAP are currently active.
CONCLUSIONS: More than one third of the EMA centrally authorised OM are currently public financed in Portugal and approximately one quarter had already been through an EAP in the country, allowing the early access to medicines to the Portuguese patients.