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

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

2014 J Comp Eff Res

Schlander, M. | Garattini, S. | Holm, S. | Kolominsky-Rabas, P. | Nord, E. | Persson, U. | Postma, M. | Richardson, J. | Simoens, S. | de Sola Morales, O. | Tolley, K. | Toumi, M. | Volume: 3, Issue: 4, Pages: 399-422, Consensus, *Cost-Benefit Analysis, Evidence-Based Medicine, Health Care Costs, Humans, Models, Economic, Outcome Assessment (Health Care)/*methods, *Quality-Adjusted Life Years, Rare Diseases/economics/*therapy, Technology Assessment, Biomedical, consensus statement, cost-effectiveness, economic evaluation, fairness, health technology assessment, medical ethics, medical need, orphan medicines, quality-adjusted life year, social preferences,

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.

https://www.doi.org/10.2217/cer.14.34