OBJECTIVES : To determine the most relevant criteria for the reimbursement of orphan medicines in Spain, from a multi-stakeholder perspective.

METHODS : A multicriteria decision analysis was developed in 3 phases. It included 28 stakeholders closely related to rare diseases (6 physicians, 5 hospital pharmacists, 7 health economists, 4 patients’ representatives and 6 members from national and regional Health Authorities). Initially [A], a bibliographic review was conducted to identify potential criteria. Then, a reduced advisory board (8 members) proposed, selected, and defined the final list of criteria that could be relevant for reimbursement. A discrete choice experiment (DCE) [B] was developed to determine the relevance and relative importance weight of such criteria according to the stakeholders’ preferences by choosing between pairs of hypothetical financing scenarios. A multinomial logit model was fitted to analyze the DCE responses. Finally [C], the advisory board review the reviewed the results using a deliberative process.

RESULTS : Thirteen criteria were selected, related to 4 dimensions: patient population, disease, treatment, and economic evaluation. Nine criteria were deemed relevant for decision-making and associated with a higher relative importance: Health-related quality of life (HRQL) (23.53%), treatment efficacy (14.64%), available treatment options (13.51%), disease severity (12.62%), avoided costs (11.21%), age of target population (7.75%), safety (seriousness of adverse events) (4.72%), evidence quality (3.82%) and size of target population (3.12%). The remaining criteria had a <3% relative importance: economic burden of disease (2.50%), treatment costs (1.73%), cost-effectiveness (0.83%) and safety (frequency of adverse events) (0.03%).

CONCLUSIONS : The reimbursement of orphan medicines in Spain should be determined by its effect on patient’s HRQL, the extent of its therapeutic benefit from efficacy and the availability of other therapeutic options. Furthermore, the severity of the rare disease should also influence the decision along with the potential of the treatment to avoid associated costs.