The purpose of this research is to identify the attributes to include in a value framework for orphan medicinal products (OMPs), determine their relative importance using a multi-criteria decision analysis (MCDA) process, and test whether an MCDA approach can support decision making in practice.
The project included literature searches and three workshops. The latter involved pharmaceutical industry R&D managers, clinical and health economics experts, and rare disease patients’ groups. Participants refined the attributes, weighted them, scored two case study OMPs on those attributes and tested the sensitivity of the overall ratings to changes in weights and scores.
Eight non-monetary attributes were agreed, four about the disease being treated and four about the treatment itself. The weighted attributes readily were applied by workshop participants to two example OMPs and produced distinct value ratings.
The project showed both that an OMP value framework with agreed attributes and weights can be created using an MCDA approach and that this could increase clarity and transparency in decisionmaking about the value of OMPs.
A revised version of this paper has been published in Value in Health and can be downloaded from: https://www.clinicalkey.com/#!/content/playContent/1-s2.0-S1098301513043568?returnurl=null&referrer=null
Please cite as: Sussex, J., Rollet, P., Garau, M., Schmitt, C., Kent, A. and Hutchings, A., 2013. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value in Health, 16(8), pp.1163-1169.