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Background:    There is a perception held by payers that orphan products are expensive. As a result, the current health technology assessment systems might be too restrictive for orphan drugs, therefore potentially denying patients access to life-saving medicines. While price is important, it should be considered in relation to a broader range of disease-related product attributes that are not necessarily considered by many health technology assessment agencies. To overcome these challenges, multi-criteria decision analysis has been proposed as an alternative to evaluate technologies.    Methods:  A targeted literature review was conducted to identify the most frequently cited attributes in multi-criteria decision analysis (MCDA) in rare diseases. From the leading attributes identified, we developed a multi-criteria decision analysis framework with which to aggregate the orphan drug values. We subsequently reviewed and plotted the relationship between single attributes and the average annual treatment costs for 8 drugs used in the treatment of rare endocrine diseases. The annual treatment costs were based on UK list prices for the average daily dose per patient.    Results:  The five most frequently mentioned attributes in the literature were as follows: Disease severity, Unmet need (or availability of therapeutic alternatives), Comparative effectiveness or efficacy, Quality of evidence and Safety &amp; tolerability. Results from the multi-criteria decision analysis framework indicate a wide range of average annual per-patients costs for drugs intended for the same diseases, and likewise for diseases with a similar level of Disease severity.    Conclusions:  Multi-criteria decision analysis may offer a viable alternative to support discussion in reimbursement decisions for orphan drugs. The analyses can be used to inform investigations on the application of MCDAs in rare diseases.

The application of multi-criteria decision analysis to inform in resource allocation