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

Research output: Contribution to journalArticle

24 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 - Jul 1 2014

Fingerprint

Quality-Adjusted Life Years
Cost-Benefit Analysis
Costs and Cost Analysis
Economic Models
Resource Allocation
Evidence-Based Medicine
Delivery of Health Care
Health
Pharmaceutical Preparations

Keywords

  • consensus statement
  • cost-effectiveness
  • economic evaluation
  • fairness
  • health technology assessment
  • medical ethics
  • medical need
  • orphan medicines
  • quality-adjusted life year
  • social preferences

ASJC Scopus subject areas

  • Health Policy
  • Medicine(all)

Cite this

Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. / Schlander, Michael; Garattini, Silvio; Holm, Søren; Kolominsky-Rabas, Peter; Nord, Erik; Persson, Ulf; Postma, Maarten; Richardson, Jeff; Simoens, Steven; De Solà Morales, Oriol; Tolley, Keith; Toumi, Mondher.

In: Journal of Comparative Effectiveness Research, Vol. 3, No. 4, 01.07.2014, p. 399-422.

Research output: Contribution to journalArticle

Schlander, M, Garattini, S, Holm, S, Kolominsky-Rabas, P, Nord, E, Persson, U, Postma, M, Richardson, J, Simoens, S, De Solà Morales, O, Tolley, K & Toumi, M 2014, 'Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders', Journal of Comparative Effectiveness Research, vol. 3, no. 4, pp. 399-422. https://doi.org/10.2217/cer.14.34
Schlander, Michael ; Garattini, Silvio ; Holm, Søren ; Kolominsky-Rabas, Peter ; Nord, Erik ; Persson, Ulf ; Postma, Maarten ; Richardson, Jeff ; Simoens, Steven ; De Solà Morales, Oriol ; Tolley, Keith ; Toumi, Mondher. / Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. In: Journal of Comparative Effectiveness Research. 2014 ; Vol. 3, No. 4. pp. 399-422.
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