Medicines for rare diseases may be effectively valued using multi-criteria decision analysis

A study just published by the Office of Health Economics (OHE) and its collaborators demonstrates how medicines for rare disease may be effectively valued using an innovative approach -- multi-criteria decision analysis (MCDA) -- that can include all relevant stakeholders, including patients' groups.

Assessing value for treatments of rare diseases has long been a challenge. The small number of patients affected by each disease make it markedly more time consuming and expensive to compile evidence of treatment effects for rare diseases than for other diseases. Payers recognise that a different approach may be needed to determine reimbursement eligibility and levels for rare disease treatments.

To explore one promising approach, OHE collaborated with GlaxoSmithKline to test the feasibility of using MCDA techniques for valuing medicines for rare disease. The three one-day workshops involved a range of stakeholders, including clinical and health economics experts and representatives of patients' groups. The objective was to establish and use an explicit framework of decision criteria to value medicines for rare diseases. Participants "weighted" various attributes of the disease and the treatment to reflect the relative importance of each in decisions.

All participants assigned as much weight to attributes of the disease as to attributes of the treatment. Patients' group representatives, however, gave greater weight than the clinical and health economics experts to the patient's quality of daily life and less weight to clinical factors.

Jon Sussex, Deputy Director of OHE and lead researcher on the project, emphasised: "This innovative MCDA approach proved very much 'fit for purpose'. We are confident that it can help health care payers and their advisers capture and consistently make decisions based on what is most important about new treatments for rare diseases."