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一项关于治疗新生血管性年龄相关性黄斑变性的现有成本效益研究的质量、有效性和有用性的系统评价。

A systematic review on the quality, validity and usefulness of current cost-effectiveness studies for treatments of neovascular age-related macular degeneration.

机构信息

Maastricht University Medical Center, University Eye Clinic Maastricht, Maastricht, The Netherlands.

出版信息

Acta Ophthalmol. 2018 Dec;96(8):770-778. doi: 10.1111/aos.13824. Epub 2018 Jun 4.

Abstract

PURPOSE

Ophthalmologists increasingly depend on new drugs to advance their treatment options. These options are limited by restraints on reimbursements for new and expensive drugs. These restraints are put in place through health policy decisions based on cost-effectiveness analyses (CEA). Cost-effectiveness analyses need to be valid and of good quality to support correct decisions to create new treatment opportunities. In this study, we report the quality, validity and usefulness of CEAs for therapies for nAMD.

METHODS

A systematic review in PubMed, EMBASE and Cochrane was performed to include CEAs. Quality and validity assessment was based on current general quality criteria and on elements that are specific to the field of ophthalmology.

RESULTS

Forty-eight CEAs were included in the review. Forty-four CEAs did not meet four basic model quality and validity criteria specific to CEAs in the field of ophthalmology (both eyes analysed instead of one; a time horizon extending beyond 4 years; extrapolating VA and treatment intervals beyond trial data realistically; and including the costs of low-vision). Four CEAs aligned with the quality and validity criteria. In two of these CEAs bevacizumab as-needed (PRN) was more cost-effective than bevacizumab monthly; aflibercept (VIEW); or ranibizumab monthly or PRN. In two CEAs, ranibizumab (PRN or treat and extent) was dominant over aflibercept. In two other CEAs, aflibercept was either more cost-effective or dominant over ranibizumab monthly or PRN.

CONCLUSION

Two of the CEAs of sufficient quality and validity show that bevacizumab PRN is the most cost-effective treatment. Comparing ranibizumab and aflibercept, either treatment can be more cost-effective depending on the assumptions used for drug prices and treatment frequencies. The majority of the published CEAs are of insufficient quality and validity. They wrongly inform decision-makers at the cost of opportunities for ophthalmologists to treat patients. As such, they may negatively influence overall patient outcomes and societal costs. For future ophthalmic treatments, CEAs need to be improved and only published when they are of sufficient quality and validity.

摘要

目的

眼科医生越来越依赖新药来拓展他们的治疗选择。这些选择受到新的昂贵药物报销限制的限制。这些限制是通过基于成本效益分析(CEA)的健康政策决策制定的。成本效益分析需要有效且质量良好,以支持做出正确的决策,为新的治疗机会创造条件。在这项研究中,我们报告了治疗 nAMD 的疗法的 CEA 的质量、有效性和实用性。

方法

在 PubMed、EMBASE 和 Cochrane 中进行了系统综述,以纳入 CEA。质量和有效性评估基于当前的一般质量标准和特定于眼科领域的要素。

结果

综述纳入了 48 项 CEA。44 项 CEA 不符合眼科领域 CEA 的四项基本模型质量和有效性标准(分析双眼而不是单眼;时间范围超过 4 年;超出试验数据现实地推断 VA 和治疗间隔;包括低视力成本)。四项 CEA 符合质量和有效性标准。在这四项 CEA 中的两项中,贝伐单抗按需(PRN)比贝伐单抗每月更具成本效益;阿柏西普(VIEW);或雷珠单抗每月或 PRN。在两项 CEA 中,雷珠单抗(PRN 或治疗和范围)优于阿柏西普。在另外两项 CEA 中,阿柏西普要么更具成本效益,要么优于雷珠单抗每月或 PRN。

结论

两项质量和有效性足够的 CEA 表明贝伐单抗 PRN 是最具成本效益的治疗方法。比较雷珠单抗和阿柏西普,根据药物价格和治疗频率的假设,任何一种治疗方法都可能更具成本效益。发表的大多数 CEA 质量和有效性不足。它们以牺牲眼科医生治疗患者的机会为代价错误地为决策者提供信息。因此,它们可能会对整体患者结局和社会成本产生负面影响。对于未来的眼科治疗,CEA 需要改进,只有在质量和有效性足够时才应发表。

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