OBJECTIVES: Assess clinical and regulatory variables that would influence P&R decisions for ODs in Spain.

METHODS: ODs approved by the European Commission (EC) until 2021 were classified according to their P&R status in Spain: approved, undergoing decision and rejected. P&R evaluation timelines before and after the inclusion of the Therapeutic Positioning Report (TPR) were analysed. Variables that could be relevant for the P&R process in Spain were assessed: therapeutic area, rarity of disease, existence of alternative therapies, availability of survival-related outcomes, safety profile, type of population, conditional approval status granted by the European Medicines Agency and a positive TPR opinion. Economic variables were not assessed due to lack of publicly available information.

RESULTS: 111 ODs have been approved by the EC and have obtained marketing authorisation in Spain until 2021. Out of the 111 ODs, 57 (51.4%) were reimbursed, 24 (21.6%) were undergoing decision and 30 (27%) were rejected. Before the inclusion of the TPR in 2013, the mean time from P&R request to P&R decision was 19,1 months; after the TPR inclusion, the mean time was 18,2 months. P&R evaluation timelines have been slightly reduced since the inclusion of TPRs by an average of less than 1 month. ODs would be more likely to be reimbursed in Spain with a positive TPR opinion (80% were reimbursed), if for an ultra-rare disease indication (63% were reimbursed), based on survival-related outcomes (57% were reimbursed), without conditional approval (55% were reimbursed) and with an oncology indication (51% were reimbursed).

CONCLUSIONS:

51.4% ODs were reimbursed until 2021 and 21.6% ODs were undergoing decision with the potential to be reimbursed in the future. P&R approval for ODs in Spain would be positively affected by a positive TPR opinion, an indication for an ultra-rare disease, availability of survival-related outcomes, non-conditional approval and an oncology indication.