Abstract：
This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint.
Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response.
Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement.
Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.
Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response.
Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement.
Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.
摘要：
目的：本研究旨在说明多系统疾病患者达到条件特异性复合反应终点后，如何评估健康增益的价值。
方法：从国家登记册（不列颠群岛狼疮评估小组生物制品登记册）中提取了在常规实践中治疗的示例性多系统疾病（系统性红斑狼疮）患者的数据。在本研究之前开发了两个定制的复合反应终点(主要临床反应和改善)。差异回归比较了响应者和非响应者在6个月内的健康效用值（EQ-5D的3级版本；英国关税）。自举回归估计了增量质量调整生命年(QALY)，达到响应标准后的QALY增益概率，和人口货币利益的反应。
结果：在样本中（n=171），18.2%的人在6个月时实现了重大临床反应，49.1%的人在6个月时实现了改善。主要临床反应的增量健康效用值为0.0923，改善为0.0454。6个月时，主要临床反应的预期QALY增量为0.020，改善为0.012。达到反应标准后，主要临床反应的QALY增益概率为77.6％，改善为72.7％。主要临床反应的人口货币收益为1106458英镑，改善为649134英镑。
结论：在试验和观察性研究中，Bespoke复合反应终点在测量多系统疾病的治疗反应方面变得越来越普遍。卫生技术评估机构面临越来越大的挑战，以确定这些终点是否与改善的健康收益相对应。健康效用值可以产生这种证据，以增强复合反应终点对健康技术评估的有用性，决策,和经济评价。
方法：从国家登记册（不列颠群岛狼疮评估小组生物制品登记册）中提取了在常规实践中治疗的示例性多系统疾病（系统性红斑狼疮）患者的数据。在本研究之前开发了两个定制的复合反应终点(主要临床反应和改善)。差异回归比较了响应者和非响应者在6个月内的健康效用值（EQ-5D的3级版本；英国关税）。自举回归估计了增量质量调整生命年(QALY)，达到响应标准后的QALY增益概率，和人口货币利益的反应。
结果：在样本中（n=171），18.2%的人在6个月时实现了重大临床反应，49.1%的人在6个月时实现了改善。主要临床反应的增量健康效用值为0.0923，改善为0.0454。6个月时，主要临床反应的预期QALY增量为0.020，改善为0.012。达到反应标准后，主要临床反应的QALY增益概率为77.6％，改善为72.7％。主要临床反应的人口货币收益为1106458英镑，改善为649134英镑。
结论：在试验和观察性研究中，Bespoke复合反应终点在测量多系统疾病的治疗反应方面变得越来越普遍。卫生技术评估机构面临越来越大的挑战，以确定这些终点是否与改善的健康收益相对应。健康效用值可以产生这种证据，以增强复合反应终点对健康技术评估的有用性，决策,和经济评价。
