title: Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders.
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.
authors.affiliation: Mannheim Medical Faculty, Department of Public Health, University of Heidelberg, Ludolf-Krehl-Strasse 7-11, D-68167 Mannheim, Germany;
authors.forename: Michael
authors.initials: M
authors.lastname: Schlander
authors.forename: Silvio
authors.initials: S
authors.lastname: Garattini
authors.forename: Søren
authors.initials: S
authors.lastname: Holm
authors.forename: Peter
authors.initials: P
authors.lastname: Kolominsky-Rabas
authors.forename: Erik
authors.initials: E
authors.lastname: Nord
authors.forename: Ulf
authors.initials: U
authors.lastname: Persson
authors.forename: Maarten
authors.initials: M
authors.lastname: Postma
authors.forename: Jeff
authors.initials: J
authors.lastname: Richardson
authors.forename: Steven
authors.initials: S
authors.lastname: Simoens
authors.forename: Oriol
authors.initials: O
authors.lastname: de Solà Morales
authors.forename: Keith
authors.initials: K
authors.lastname: Tolley
authors.forename: Mondher
authors.initials: M
authors.lastname: Toumi
mesh_terms: D006801:Humans; D003362:Cost-Benefit Analysis; D019317:Evidence-Based Medicine; D017048:Health Care Costs; D018803:Models, Economic; D017063:Outcome Assessment, Health Care / Q000379:methods; D019057:Quality-Adjusted Life Years; D035583:Rare Diseases / Q000191:economics / Q000628:therapy; D013673:Technology Assessment, Biomedical; D003245:Consensus Statements as Topic
keywords: consensus statement; cost–effectiveness; economic evaluation; fairness; health technology assessment; medical ethics; medical need; orphan medicines; quality-adjusted life year; social preferences