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- PMID: 25275236
- UKPMCID: 25275236
- DOI: 10.2217/cer.14.34
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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; Solà Morales, Oriol de; Tolley, Keith; Toumi, Mondher
Journal of comparative effectiveness research. 2014;3(4):399-422.
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Full-text held externally
- PMID: 25275236
- UKPMCID: 25275236
- DOI: 10.2217/cer.14.34
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.
Keyword(s)
consensus statement; cost–effectiveness; economic evaluation; fairness; health technology assessment; medical ethics; medical need; orphan medicines; quality-adjusted life year; social preferences