Analytic considerations in applying a general economic evaluation reference case to gene therapy

M.F. Drummond, P. Neumann, S. Sullivan, F.U. Fricke, S. Tunis, O. Dabbous, M. Toumi

Research output: Contribution to journalArticlepeer-review


The concept of a reference case, first proposed by the US Panel on Cost-Effectiveness in Health and Medicine, has been used to specify the required methodological features of economic evaluations of health care interventions. In the case of gene therapy, there is a difference of opinion on whether a specific methodologic reference case is required. The aim of this paper is to provide a more detailed analysis of the characteristics of gene therapy and the extent to which these characteristics warrant modifications to the methods suggested in general reference cases for economic evaluation. We argue that a completely new reference case is not required, but propose a tailored checklist that can be used by analysts and decision-makers to determine which aspects of economic evaluation should be considered further, given the unique nature of gene therapy.
Original languageEnglish
Pages (from-to)661-668
Number of pages8
JournalValue in Health
Issue number6
Publication statusPublished - 12 Jun 2019

Bibliographical note

© 2019, ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. This is an author-produced version of the published paper. Uploaded in accordance with the publisher’s self-archiving policy.


  • cost-effectiveness analysis;
  • drug pricing;
  • reimbursement
  • specialty drugs

Cite this