Objectives: The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service).
Methods: The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members. The criteria weighting was done using two different techniques (nonhierarchical and hierarchical). Reliability was assessed by re-test.
Results: The EVIDEM framework and methodology was found useful and feasible for Orphan drugs evaluation and decision making in Catalonia. All the criteria considered for the development of the CatSalut Technical Reports and decision making were considered in the framework. Nevertheless, the framework could improve the reporting of some of these criteria (i.e., “unmet needs” or “nonmedical costs”). Some Contextual criteria were removed (i.e., “Mandate and scope of healthcare system”, “Environmental impact”) or adapted (“population priorities and access”) for CatSalut purposes. Independently of the weighting technique considered, the most important evaluation criteria identified for orphan drugs were: “disease severity”, “unmet needs” and “comparative effectiveness”, while the “size of the population” had the lowest relevance for decision making. Test–retest analysis showed weight consistency among techniques, supporting reliability overtime.
Conclusions: MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.