Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs
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- 作者：C. Schey ; P. F. M. Krabbe ; M. J. Postma…
- 关键词：Multi ; criteria decision analysis ; MCDA ; Orphan drugs ; Reimbursement ; Mucopolysaccharidosis II ; Paroxysmal nocturnal haemoglobinuria ; Pulmonary arterial hypertension ; Myelodysplastic syndromes ; Lennox ; Gastaut syndrome
- 刊名：Orphanet Journal of Rare Diseases
- 出版年：2017
- 出版时间：December 2017
- 年：2017
- 卷：12
- 期：1
- 全文大小：970KB
- 刊物主题：Medicine/Public Health, general; Pharmacology/Toxicology; Human Genetics;
- 出版者：BioMed Central
- ISSN：1750-1172
- 卷排序：12
文摘
BackgroundSince the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings.