Development and validation of an MCDA framework for evaluation and decision-making of orphan drugs in Spain

Abstract

ABSTRACTIntroduction: To develop a reflective multi-criteria decision analysis (MCDA) framework with relevant criteria that allows evaluation, positioning, and aids in decision-making of Orphan Drugs (ODs) at the national level in Spain.Areas covered: The framework was developed following the EVIDEM methodology. The study was conducted in two phases. In the first phase, the literature search was performed to identify additional criteria used for evaluation of ODs. A multidisciplinary Panel of Experts in rare diseases in Spain assessed the criteria for their inclusion in the framework, and proposed modifications to their definition and/or response scale. In the second phase, to validate the criteria, the framework was tested using two case studies. A reflective discussion based on the scores assigned to each criterion was carried out.Expert opinion: MCDA methods can support healthcare decision making by taking into account multiple criteria in an explicit manner and beyond the traditional assessments. A reflective MCDA framework for the evaluation of ODs in Spain has been developed. The OD-specific framework is in line with the one in use to complement OD evaluation in a regional drug evaluation committee in Spain (Catalonia).