Abstract
BackgroundPatient access to orphan medicinal products (OMPs) is limited and varies between countries, reimbursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. This study aimed to determine the most relevant criteria for the reimbursement of OMPs in Spain, from a multi-stakeholder perspective, and using multicriteria decision analysis (MCDA).MethodsAn MCDA was developed in 3 phases and included 28 stakeholders closely related to the field of rare diseases (6 physicians, 5 hospital pharmacists, 7 health economists, 4 patient representatives and 6 members from national and regional health authorities). Initially [phase A], a bibliographic review was conducted to identify the potential reimbursement 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) [phase 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 [phase C], the advisory board review the results using a deliberative process.ResultsThirteen 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%), availability of treatment alternatives (13.51%), disease severity (12.62%), avoided costs (11.21%), age of target population (7.75%), safety (seriousness of adverse events) (4.72%), quality of evidence (3.82%) and size of target population (3.12%). The remaining criteria had a < 3% relative importance: economic burden of disease (2.50%), cost of treatment (1.73%), cost-effectiveness (0.83%) and safety (frequency of adverse events) (0.03%).ConclusionThe reimbursement of OMPs 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.
Highlights
Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reim‐ bursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions
The objective of this study was twofold: first, to review, discuss, and reach a consensus on the most relevant criteria for decision-making about pricing and financing OMPs in Spain; and second, to prioritize them according to their relative importance based on the preferences stated by different stakeholders, following the multicriteria decision analysis (MCDA) methodology
Selection and definition of levels From the 19 publications selected in the literature review, 225 reimbursement criteria related to the treatment, the disease, and the affected population were identified
Summary
Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reim‐ bursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. Rare diseases pose a threat to the health of individuals They are diseases of low prevalence and high complexity de Andrés‐Nogales et al Orphanet J Rare Dis (2021) 16:186 that can lead to death or chronic disability [1] and for which there frequently are no therapeutic options [2]. It is estimated that there are more than 6,000 rare diseases [5], affecting between 6 and 8% of the population at some point in their lives [1] Because of their low prevalence, their specificity, and the high number of people they affect altogether, these pathologies require a comprehensive approach and priority of action to prevent significant morbidity or premature mortality and to improve the quality of life and socioeconomic potential of the people [1]
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