PHP52 - ACCESSIBILITY AND ACCESS TO ORPHAN DRUGS IN PORTUGAL: HOW FAR ARE WE?

The regulatory framework of orphan drugs falls under the mandatory scope of the Centralized Procedure (CP). A positive deferral by the European Medicines Agency (EMA) grants a valid marketing authorization (MA) across European Union. However, national accessibility and access are dependent on pharmaceutical policy and economic environment which are determinant for pharmaceutical companies market strategy. The aim of this study is to assess the accessibility and access to orphan drugs in Portugal. Orphan drug records were extracted from EMA database. Variables collected: MA status, ATC code, conditional (CA) and exceptional circumstances (EC) approvals. Only those with a valid MA were included. Therapeutic areas were categorized according to the first level of ATC code. National accessibility was defined as having at least one registration number per CP. Access was evaluated according to the marketing status. Both were retrieved from INFARMED. Up to June 2018, 120 orphan drugs applications have been assessed under CP: 11 have been refused and 4 have already been withdrawn, leaving a total of 105 orphan drugs with valid MA. Antineoplasic and immunomodulating agents (AIA) (43.8%), alimentary and tract metabolism drugs (ATM) (18.1%) and Anti-infective for systemic use (ASU) (7.6%) were the most frequent therapeutic areas. A total of 15 MA (14.3%) were granted under EC and 14 (13.3%) under CA. The proportion of orphan drugs registered in Portugal is 76.2% and only 33.3% is currently being marketed. Little or no information is available relative to the incorporation of this orphan drugs at hospital level. The respective proportions for AIA, ATM and ASU were 87.0% and 39.1%, 68.4% and 26.3%, 68.4% and 26.3%. Only 13.3% of CA and 7.1% EC are currently marketed. A gap has been observed between European and national accessibility. Access to orphan drugs in Portugal is substantially impaired relative to EMA approvals.