OBJECTIVES: Orphan diseases such as Huntington’s disease (HD), a progressive neurodegenerative disorder, have significant impact on patients and their families, as well as the healthcare systems and societies. The main aim of this study was to perform multicriteria decision analysis (MCDA) to evaluate various aspects of a medicament providing of tetrabenazine (TBZ) for patients with HD.

METHODS: Analysis of the published clinical trials was conducted to evaluate efficacy and safety of using TBZ for patients with HD. The initial list of value attributes (5 - impact of rare disease and 5 - impact of the drug) was identified from a literature review and expert’s survey. Further experts assigned relative weights to the attributes in 2 groups (the rating scale ranged from 1(less important) to 5 (most importantly)). Then experts rated TBZ against each attribute (the rating scale ranged from 1 (worst score) to 7 (best score)). In the end weighted score for each attribute was identified.

RESULTS: Experts considered that the most important attributes are the impact of disease (scores 27.3 versus 24.7). In the both groups the most important attribute was evidence of treatment clinical efficacy and patient clinical outcome (scores 6.67 and 6.00). A total weighted score for attributes of the disease was 85.78, and for attributes of the impact of the drug – 78.67.

CONCLUSIONS: All experts agreed to give slightly more weight to the attributes of the disease than to the impact of the drug. The present study was the first experience of conducting MCDA for HD. This analysis showed the importance of the different kinds of aspects in deciding on funding drug supply patients with HD.