OBJECTIVES: Identify if the official criteria of Spanish P&R process are related with P&R approval for ODs.

METHODS: ODs approved by the European Commission between 2012 and 2016 were classified according to their P&R status in Spain (approved, undergoing decision and rejected). Hypotheses on likelihood of reimbursement for ODs were formulated based on the P&R official criteria established by Royal Decree Law 1/2015 of 24 July (disease severity, unmet needs of certain collectives, cost-effectiveness (CE), budget impact (BI), existence of alternative therapies and degree of innovation). Disease severity was related to an oncologic vs. non oncologic indication and to the availability of hard clinical trial outcomes; unmet needs of certain collectives was related to being indicated for ultra-orphan diseases affecting <1/50000 inhabitants; existence of alternative therapies was related to ODs without a therapeutic alternative for the approved indication and degree of innovation was related to ODs with a published Therapeutic Positioning Report (TPR) with a positive opinion, meaning that the drug offers an added therapeutic value. CE and BI were not assessed in this study because of lack of information.

RESULTS: ODs were more likely to be reimbursed in Spain if approved for an oncological indication (56% with P&R approval), approval based on hard clinical outcomes (57% with P&R approval) and a TPR with a positive opinion (88.2% with P&R approval). Indications for a smaller group of patients or for those not having a therapeutic alternative, did not seem to have a positive influence for achieving P&R in Spain.

CONCLUSIONS: P&R approval for ODs in Spain could be positively affected by an indication in oncology, availability of hard clinical outcomes and a positive TPR. BI would be an important variable for P&R approval, but could not assessed as information about sales forecast is not public.