Office of Health Economics, London, UK.
Value Health. 2013 Dec;16(8):1163-9. doi: 10.1016/j.jval.2013.10.002. Epub 2013 Nov 14.
To pilot the use of multicriteria decision analysis to establish and apply a framework of weighted attributes to value orphan medicinal products.
Literature searches on the natural history and burden of 40 rare diseases and of how payers assess treatment value and three workshops with, respectively, GlaxoSmithKline managers working on orphan medicinal products, European Union clinical and health economics experts, and representatives of rare diseases patient groups in the European Union.
Eight nonmonetary attributes were identified and weights agreed: four concern the disease being treated and four the treatment itself. About half of the weight went to attributes of the disease treated and half to attributes of the treatment. Patient group representatives gave greater weight than did the experts to patients' and carers' quality of daily life.
The multicriteria decision analysis approach piloted works and could be developed for use by payers and health technology assessment bodies.
试点使用多准则决策分析来建立和应用加权属性框架，以评估孤儿药物的价值。
对 40 种罕见疾病的自然史和负担进行文献检索，以及对支付者如何评估治疗价值进行三次研讨会，分别涉及葛兰素史克公司从事孤儿药物的管理人员、欧盟临床和卫生经济学专家以及欧盟罕见疾病患者群体的代表。
确定了 8 个非货币属性并达成了权重共识：其中 4 个与治疗的疾病有关，4 个与治疗本身有关。约一半的权重用于治疗疾病的属性，另一半用于治疗的属性。与专家相比，患者群体代表更重视患者和照顾者的日常生活质量。
试点使用的多准则决策分析方法有效，并可由支付者和卫生技术评估机构开发使用。
