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

*Bijbehorende auteur voor dit werk

OnderzoeksoutputAcademicpeer review

28 Citaten (Scopus)


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.

Originele taal-2English
Pagina's (van-tot)399-422
Aantal pagina's24
TijdschriftJournal of Comparative Effectiveness Research
Nummer van het tijdschrift4
StatusPublished - 1-jul-2014

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