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评估新西兰人为药物覆盖范围决策的价值观:在罕见和常见疾病治疗之间的权衡。

Evaluating New Zealanders' Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions.

机构信息

Pharmacy Intern, Wellington, New Zealand.

School of Pharmacy, University of Otago, PO Box 56, Dunedin, 9054, New Zealand.

出版信息

Pharmacoeconomics. 2021 Jan;39(1):109-119. doi: 10.1007/s40273-020-00974-8. Epub 2020 Nov 5.

DOI:10.1007/s40273-020-00974-8
PMID:33150565
Abstract

BACKGROUND

New Zealand's near static healthcare budget limits access to expensive medications including those for rare conditions. As such, it is necessary to know the public's priority for values in the drug funding decision-making process.

OBJECTIVES

The objectives of this study were to measure the relative societal importance of values of New Zealanders in informing drug funding decisions and to determine how New Zealanders trade off funding in various scenarios between common and rare diseases.

METHODS

An online survey was conducted between 17 April and 17 May, 2019 on a sample of 500 New Zealanders aged ≥ 18 years. Participants ranked 13 values using an analytical hierarchy process. Participants were then presented with different trade-off scenarios to measure their attitudes towards funding drugs for common and rare diseases.

RESULTS

The values ranked in the top five by most were potential effect on quality of life (71.8%), ability of the drug to work (57.6%), severity (57.6%), safety (57%), and potential to extend life (56%). Adherence and rarity held the lowest and second lowest ranking. Most believe that resources should be allocated towards drugs that have been proven to work and have the greatest health benefits. In trade-offs between access to an expensive drug therapy for a rare disease with uncertain benefits or receive a fixed cash payment, the overwhelming consensus was to receive the cash payment.

CONCLUSIONS

New Zealanders ultimately value drug-related factors (e.g. quality of life and efficacy) and disease-related factors (e.g. severity of disease and equity) the most but did not value disease rarity.

摘要

背景

新西兰的医疗保健预算几乎一成不变,这限制了昂贵药物(包括治疗罕见疾病的药物)的可及性。因此,有必要了解公众在药物资金决策过程中对价值观的重视程度。

目的

本研究旨在衡量新西兰人在药物资金决策中价值观的相对社会重要性,并确定新西兰人在常见和罕见疾病的各种情况下如何权衡资金分配。

方法

2019 年 4 月 17 日至 5 月 17 日期间,通过在线调查对 500 名年龄≥18 岁的新西兰人进行了抽样调查。参与者使用层次分析法对 13 种价值观进行了排序。然后向参与者呈现不同的权衡方案,以衡量他们对为常见和罕见疾病提供药物资金的态度。

结果

排名前五的价值观是对生活质量的潜在影响(71.8%)、药物的有效性(57.6%)、严重程度(57.6%)、安全性(57%)和延长寿命的潜力(56%)。依从性和罕见性排名最低和第二低。大多数人认为,资源应该分配给已经证明有效的药物,这些药物能带来最大的健康效益。在为罕见疾病提供昂贵药物治疗的机会与获得固定现金支付之间进行权衡时,绝大多数人选择获得现金支付。

结论

新西兰人最终最看重与药物相关的因素(如生活质量和疗效)和与疾病相关的因素(如疾病的严重程度和公平性),但不看重疾病的罕见性。

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