View article

Objectives: The purpose of this research is to identify the attributes to include in an orphan medicinal product (OMP) value framework, determine their relative importance via a multi-criteria decision analysis (MCDA) process, and test whether an MCDA approach can practically support decision making.

Methods: The project included literature searches on the natural history and burden of 40 rare diseases and how payers assess treatment value as well as three workshops. Workshops also were held: the first with GlaxoSmithKline managers working on OMPs, the second with EU clinical and health economics experts, and the third with representatives of rare diseases patient groups in the European Union. Participants refined the attributes, weighted them, scored two case study OMPs in terms of those attributes, and tested the sensitivity of the overall ratings to changes in weights and scores.

Results: Eight non-monetary attributes were agreed: four concern the disease being treated and four the treatment itself. Workshop participants agreed consensus weights for the attributes. The patient group representatives and the clinical and health economics experts both attributed about half of the weight to attributes reflecting the disease being treated, and half to attributes of the treatment. Patient group representatives gave greater weight than the experts to patients’ quality of daily life and less weight to clinical factors. The weighted attributes were readily applied by workshop participants to two example OMPs and yielded distinct ratings of their respective values.

Conclusions: An OMP value framework with agreed attributes and weights is a viable …