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, Soren Holm, Peter Kolominsky-Rabas, Erik Nord, Ulf Persson, Maarten Postma, Jeffrey Ralph James Richardson, Steven Simoens, Oriol de Sola Morales, Keith Tolley, Mondher Toumi

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45 Citations (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.
Original languageEnglish
Pages (from-to)399 - 422
Number of pages24
JournalJournal of Comparative Effectiveness Research
Issue number4
Publication statusPublished - 2014

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