Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders

Michael Schlander*, Silvio Garattini, Søren Holm, Peter Kolominsky-Rabas, Erik Nord, Ulf Persson, Maarten Postma, Jeff Richardson, Steven Simoens, Oriol De Solà Morales, Keith Tolley, Mondher Toumi

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

28 Citations (Scopus)

Abstract

Drugs for ultra-rare disorders (URDs) rank prominently among the most expensive medicines on a cost-per-patient basis. Many of them do not meet conventional standards for cost-effectiveness. In light of the high fixed cost of R&D, this challenge is inversely related to the prevalence of URDs. The present paper sets out to explain the rationale underlying a recent expert consensus on these issues, recommending a more rigorous assessment of the clinical effectiveness of URDs, applying established standards of evidence-based medicine. This may include conditional approval and reimbursement policies, which should be combined with a firm expectation of proof of a minimum significant clinical benefit within a reasonable time. In contrast, current health economic evaluation paradigms fail to adequately reflect normative and empirical concerns (i.e., morally defensible 'social preferences') regarding healthcare resource allocation. Hence there is a strong need for alternative economic evaluation models for URDs.

Original languageEnglish
Pages (from-to)399-422
Number of pages24
JournalJournal of Comparative Effectiveness Research
Volume3
Issue number4
DOIs
Publication statusPublished - 1-Jul-2014

Keywords

  • consensus statement
  • cost-effectiveness
  • economic evaluation
  • fairness
  • health technology assessment
  • medical ethics
  • medical need
  • orphan medicines
  • quality-adjusted life year
  • social preferences
  • ORPHAN DRUG DEVELOPMENT
  • WILLINGNESS-TO-PAY
  • EFFECTIVENESS LEAGUE TABLES
  • EUROPEAN CLINICAL-RESEARCH
  • RESEARCH-AND-DEVELOPMENT
  • HEALTH-CARE PRIORITIES
  • FAIR INNINGS ARGUMENT
  • DONOR LIVER GRAFTS
  • ECONOMIC-EVALUATION
  • OF-LIFE

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