OBJECTIVES: To develop a Multi-Criteria Decision Analysis (MCDA) framework specific for Orphan Drugs (OD) in Catalonia, aimed to facilitate and homogenize the assessment of OD by the decision-making committee of the Catalan Health Service (CatSalut).

METHODS: A preliminary framework based on EVIDEM (v.4.0) was developed for its use in OD evaluation and was validated by representatives of the decision-making committee of CatSalut. The final version of the framework was agreed and tested by this committee in 3 OD (tolvaptan for autosomal dominant polycystic kidney disease, Alpha 1-antitrypsin for Alpha1-antitrypsin deficiency and eliglustat for Gaucher disease). The committee members rated individually the EVIDEM matrix for each drug assessment according to their preferences and afterwards a reflective discussion was conducted amongst all the members.

RESULTS: In the validation phase, some criteria were removed or not considered from the standard framework based on EVIDEM (v.4.0) (i.e., “size of population”, “non-medical costs”, “rarity” and “rule of rescue”) or adapted (“therapeutic benefit”) for CatSalut purposes.

The assessment of three OD was conducted to rate the evidence matrix. The reflective discussion was seen as very relevant to support inputs for health decision-making processes reflecting drug value and positioning medicines within therapeutic algorithms.

CONCLUSIONS: A final MCDA framework specific for OD was developed and validated to be used for the orphan drug evaluations conducted by the CatSalut. The test of the three OD through the developed framework specific for OD showed that MCDA can be considered a useful methodology which adds transparency, predictability and allows a structured discussion that substantiates the agreements adopted by decision-making committees.