Abstract

Objective

To pilot the use of multicriteria decision analysis to establish and apply a framework of weighted attributes to value orphan medicinal products.

Methods

Literature searches on the natural history and burden of 40 rare diseases and of how payers assess treatment value and three workshops with, respectively, GlaxoSmithKline managers working on orphan medicinal products, European Union clinical and health economics experts, and representatives of rare diseases patient groups in the European Union.

Results

Eight nonmonetary attributes were identified and weights agreed: four concern the disease being treated and four the treatment itself. About half of the weight went to attributes of the disease treated and half to attributes of the treatment. Patient group representatives gave greater weight than did the experts to patients’ and carers’ quality of daily life.

Conclusions

The multicriteria decision analysis approach piloted works and could be developed for use by payers and health technology assessment bodies.