APPLICABILITY OF THE EVIDEM MULTI-CRITERIA DECISION ANALYSIS FRAMEWORK FOR ORPHAN DRUGS – RESULTS FROM A STUDY IN 7 EURASIAN COUNTRIES.

Several Multi-Criteria Decision Analysis (MCDA) models for use in health technology assessment (HTA) have been developed over the years, including some for orphan drugs (OD). However, there is no general consensus yet on MCDA structure and which criteria should be included and implementation of MCDA into HTA practice has been slow so far. Our study tested the criteria preferences and possibilities for implementation of the EVIDEM MCDA framework for OD with a diverse group of 140 stakeholders in Kazakhstan, Netherlands, Poland, Romania, Russia, Turkey and Ukraine (KZ,NL,PL,RO,RU,TR,UA). The research elicited stakeholder preferences (weighting) for EVIDEM domains, criteria and sub-criteria to measure their relative importance. Correlations of work place and HTA/rare diseases experience with weighting results were investigated. Results showed that the ‘Need for intervention’ domain was assessed as the most important in: RO/NL/RU/TR, in KZ/PL the ‘Type of benefit of intervention’ and in UA ‘Economic consequences of intervention’. ‘Population Priorities’ was uniformly given a low priority. Further research, sharing of experiences and multi-stakeholder discussion is necessary to define a path forward for a robust and sustainable improvement of MCDA models and its application in orphan drug HTA. Model simplification and clarification of outcomes would be beneficial.