Valuing Rare Disease Medicines – We Can Do Better

London, United Kingdom – Many rare diseases, often severe and life-threatening, cannot be treated. The small numbers of patients affected make it time-consuming and costly to compile evidence about treatments. Organisations that pay for health care recognise that a special approach may be needed to guide reimbursement decisions about such treatments.

The Office of Health Economics (OHE) and GlaxoSmithKline held three one-day workshops in London during 2012. Participants included patients’ group representatives as well as clinical and health economics experts. The goal was to create and use a framework of weighted criteria to value medicines for rare diseases. This method was a form of multi-criteria decision analysis (MCDA).

Patients’ group representatives, clinical experts, and health economics experts all gave about half of the weight, that is they based about half of the potential value of rare disease medicines, to attributes reflecting the disease being treated, and half to the effects 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.

From this project, the authors conclude that it is feasible for health care payers to value medicines for rare diseases using an explicit framework of attributes, weighted to show their relative importance. This value framework can be created with relevant stakeholders.
According to Jon Sussex, Deputy Director of OHE and lead researcher on the study, “The MCDA approach we used proved very much ‘fit for purpose’. We are confident that it can help health care payers and their advisers to capture the information needed for consistent reimbursement decisions that reflect what is most important about new treatments for rare diseases.” The full study, “A Pilot Study of Multi-Criteria Decision Analysis for Valuing Orphan Medicines,” is published in Value in Health.


Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision-makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.

For more information: www.ispor.org

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