Valuing Orphan Medicines Using Multi-Criteria Decision Analysis

OHE has just released a research paper that describes an experimental study intended to test whether and how a multi-criteria decision analysis (MCDA) approach can be applied to orphan medicinal products (OMPs).

OHE has just released a research paper that describes an experimental study intended to test whether and how a multi-criteria decision analysis (MCDA) approach[1] can be applied to orphan medicinal products (OMPs). To be successful, the project had to both establish an explicit framework for defining the value of OMPs and provide an explicit understanding of the trade-offs that must be made in coverage decisions.

In all health care systems, HTA and reimbursement decisions begin with an assessment of value. Many systems take a distinct approach where orphan medicines are concerned. Policy makers and payers continue in seek improvements in approaches to determining the value of medicines. The move towards “value based pricing” in the UK is just one example. As yet, however, no HTA agency explicitly uses MCDA. This method applies more than one criterion to establish value and explicitly identifies the impact and relative importance of each criterion on a decision. Trades offs across attributes is characteristic of this approach; more of one attribute can compensate for less of another.

The purpose of this research was to identify which attributes to include in an OMP value framework, determine their relative importance using an MCDA process, and test whether an MCDA approach can provide a practical aid to decision making.

The project included both literature reviews and workshops. Literature searches were completed for the natural history and burden of 40 rare diseases and for how payers assess treatment value. The three workshops included, respectively (1) GlaxoSmithKline managers working on OMPs, (2) EU clinical and health economics experts, and (3) rare disease patients’ groups based in the EU. 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 focussed on the disease being treated and four addressing the treatment itself. Workshop participants reached consensus on the weights for the attributes. The patients’ group representatives and the clinical and health economics experts both assigned about half of the weight to attributes reflecting the disease being treated and half to attributes of the treatment. Patients’ group representatives, however, gave greater weight than the experts to the patient’s quality of daily life and less weight to clinical factors. The weighted attributes proved usable in practice; they readily were applied by workshop participants to two example OMPs and yielded distinct value ratings.

From this project, we conclude that an OMP value framework with agreed attributes and weights is a viable proposition using an MCDA approach. Moreover, it could improve clarity and transparency in decision making about the value of OMPs.

[1] For a thorough discussion of MCDA and its uses, see Devlin, N.J. and Sussex, J. (2011) Incorporating multiple criteria in HTA: Methods and processes. London: Office of Health Economics. The publication may be downloaded from this website by registered users.

Download: Sussex, J., Rollet, P., Garau, M., Schmitt, C., Kent, A. and Hutchings, A. Multi-criteria decision analysis to value orphan medicines. Research Paper 13/03. London: Office of Health Economics.

For additional information, please contact Jon Sussex.