Using multi-criteria decision analysis to appraise orphan drugs: a systematic review

ABSTRACT

Introduction: Multi-criteria decision analysis (MCDA) could potentially solve current methodological difficulties in the appraisal of orphan drugs.

Areas covered: We provide an overview of the existing evidence regarding the use of MCDA in the appraisal of orphan drugs worldwide. Three databases (Pubmed, Embase, Web of Science) were searched for English, French and German literature published between January 2000 and April 2017. Full-text articles were supplemented with conference abstracts. A total of seven articles and six abstracts were identified.

Expert commentary: The literature suggests that MCDA is increasingly being used in the context of appraising orphan drugs. It has shown itself to be a flexible approach with the potential to assist in decision-making regarding reimbursement for orphan drugs. However, further research regarding its application must be conducted.

KEYWORDS:

• Multi-criteria decision analysis
• MCDA
• orphan drugs
• rare diseases
• reimbursement
• appraisal
• systematic review

Acknowledgments

The authors thank the French Foundation for Rare Diseases for their support and supervision.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.