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慎思所求:益处描述对糖尿病患者益处-风险权衡偏好的影响。

Be Careful What You Ask For: Effects of Benefit Descriptions on Diabetes Patients' Benefit-Risk Tradeoff Preferences.

作者信息

von Arx Lill-Brith, Johnson F Reed, Mørkbak Morten Raun, Kjær Trine

机构信息

University of Southern Denmark, Odense, Denmark; Novo Nordisk A/S, Soeborg, Denmark.

Duke Clinical Research Institute, Durham, North Carolina, USA.

出版信息

Value Health. 2017 Apr;20(4):670-678. doi: 10.1016/j.jval.2016.11.023. Epub 2017 Feb 9.

Abstract

BACKGROUND

As more studies report on patient preferences for diabetes treatment, identifying diabetes outcomes other than glycated hemoglobin (HbA) to describe effectiveness is warranted to understand patient-relevant, benefit-risk tradeoffs.

OBJECTIVE

The aim of the study was to evaluate how preferences differ when effectiveness (glycemic control) is presented as long-term sequela (LTS) risk mitigation rather than an asymptomatic technical marker (HbA).

METHODS

People with type 2 diabetes and using insulin (n = 3160) were randomly assigned to four self-administered, discrete-choice experiments that differed by their presentation of effectiveness. Epidemiologic reviews were conducted to ensure a close approximation of LTS risk relative to HbA levels. The relative importance of treatment benefit-risk characteristics and maximum acceptable risk tradeoffs was estimated using an error-component logit model. Log-likelihood ratio tests were used to compare parameter vectors.

RESULTS

In total, 1031 people responded to the survey. Significantly more severe hypoglycemic events were accepted for a health improvement in terms of LTS mitigation versus HbA improvement (0.7 events per year; 95% confidence interval [CI]: 0.4-1.0 vs. 0.2 events per year 95% CI: -0.02 to 0.5) and avoidance of treatment-related heart attack risk (1.4 severe hypoglycemic events per year; 95% CI: 0.8-1.9 vs. 1 event per year; 95% CI: 0.6-1.3). This finding is supported by a log-likelihood test that rejected at the 0.05 level that respondent preference structures are similar across the different experimental arms of the discrete-choice experiment.

CONCLUSION

We found evidence that benefit descriptions influence elicited preferences for the benefit-risk characteristics of injectable diabetes treatment. These findings argue for using carefully defined effectiveness measures to accurately take account of the patient perspective in benefit-risk assessments.

摘要

背景

随着越来越多的研究报告患者对糖尿病治疗的偏好,有必要确定糖化血红蛋白(HbA)以外的糖尿病结局来描述治疗效果,以便理解与患者相关的获益-风险权衡。

目的

本研究旨在评估当将有效性(血糖控制)呈现为长期后遗症(LTS)风险降低而非无症状技术指标(HbA)时,偏好如何不同。

方法

将2型糖尿病且正在使用胰岛素的患者(n = 3160)随机分配到四个自我管理的离散选择实验中,这些实验因有效性呈现方式不同而有所差异。进行了流行病学综述以确保LTS风险相对于HbA水平的紧密近似。使用误差成分logit模型估计治疗获益-风险特征的相对重要性和最大可接受风险权衡。使用对数似然比检验比较参数向量。

结果

共有1031人回复了调查。就LTS缓解方面的健康改善而言,与HbA改善相比,为了健康改善而接受的严重低血糖事件显著更多(每年0.7次事件;95%置信区间[CI]:0.4 - 1.0 vs.每年0.2次事件,95% CI:-0.02至0.5),以及避免治疗相关心脏病发作风险(每年1.4次严重低血糖事件;95% CI:0.8 - 1.9 vs.每年1次事件;95% CI:0.6 - 1.3)。这一发现得到对数似然检验的支持,该检验在0.05水平上拒绝了离散选择实验不同实验组中受访者偏好结构相似的假设。

结论

我们发现有证据表明获益描述会影响对注射用糖尿病治疗获益-风险特征的引发偏好。这些发现支持使用精心定义的有效性指标,以便在获益-风险评估中准确考虑患者视角。

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