OBJECTIVES: Within value assessment of Orphan Drugs (OD), decision makers across European jurisdictions are adding additional criteria to complement data from traditional economic evaluation. Although described in theory, data on practical implementation of value assessment frameworks (VAFs) such as MCDA or EVIDEM remain scarce. Current study aims to analyse the experience with VAFs for the assessment of ODs in European jurisdictions and formulate specific recommendations to stakeholders.

METHODS: A comparative analysis was carried out, focusing on the practical implementation of VAFs for ODs in Sweden, Scotland, England, the Netherlands and France. The study focused on each jurisdiction separately and additionally mapped combinations of frameworks used in practice. Strengths and weaknesses associated with VAFs of orphan drugs were described. A systematic literature search was performed in scientific databases, in addition legal and policy documents were consulted.

RESULTS: Results show that across the selected jurisdictions, different VAFs apply, such as a variable cost-effectiveness threshold in Sweden or the use of weighted QALYs and MCDA in England. Furthermore, all of them use a different combination of frameworks, and decision-making criteria are prioritized differently. For example, while budget impact analysis affects the decision making process in Sweden and the Netherlands, this is not the case in England, where it is required but does not impact the decision.

CONCLUSIONS: So far, only England and Scotland have implemented a transparent and multi-criteria framework for the value assessment of ODs. Proper use of scoring systems and criteria weights need to be further explored and described, in order to help decision makers across European jurisdictions to develop frameworks that will support consistent and transparent value assessment.