A preliminary model-based assessment of the cost-utility of a screening programme for early age-related macular degeneration.
A preliminary model-based assessment of the cost-utility of a screening programme for early age-related macular degeneration.
复制标题
对早期年龄相关性黄斑变性筛查计划的成本效益进行基于模型的初步评估。
作者：
J. Karnon;C. Czoski;K. Smith;C. Brand;U. Chakravarthy;S. Davis;N. Bansback;C. Beverley;A. Bird;S. Harding;I. Chisholm;Y. Yang
目标
通过开发一个决策分析模型，结合并评估所有国家筛查委员会标准，估计年龄相关性黄斑变性（AMD）筛查的成本效益。另一个目标是确定模型中的主要不确定性领域，从而为该疾病领域未来的研究重点提供信息。
数据源
2004年3月检索了主要数据库，并于2005年1月更新。
复习方法
系统的文献综述涵盖了AMD的流行病学和自然史、筛查和治疗效果以及与AMD相关的健康相关生活质量。采用混合队列-个体采样模型，通过疾病不同阶段的临床表现和治疗，描述年龄相关性黄斑病变（ARM）发病率与死亡之间的途径范围。由于从文献中获得的数据明显不足，因此还使用了一系列原始数据源来填充模型。为了获得被认为属于专家职权范围的参数的估计值，从有关专家那里获得了描述某些参数的数据。数据识别了描述模型参数周围不确定性的知情概率分布。为了纳入联合参数不确定性（即参数之间的相关性），AMD自然历史模型进行了概率校准。随机采样的输入参数集被分配权重，表示它们对一组观察到的模型输出的预测的准确性。AMD筛查模型的分析估计了16种替代筛查方案（包括不筛查）的成本、质量调整生命年（QDs）数量和50岁人群样本中的失明病例。参考病例分析纳入了激光光凝和光动力疗法的当前治疗选择。还进行了敏感性分析，描述了六套替代干预策略，基于对AMD未来潜在治疗的视野扫描。
结果
对于任何形式的AMD筛查是否具有成本效益仍然存在很大的不确定性。然而，从60岁开始的年度筛查似乎提供了最高的平均净效益，但这是基于精确度很差的成本效益估计（高度不确定性）。概率敏感性分析表明，从60岁开始每年筛查的95%可信区间范围从该选项主导先前选项到每QALY超过50万英镑的增量成本。绘制成本效益可接受边界表明，虽然从60岁开始每年筛查的净效益最高，质量调整生命年值为30，000英镑，但该选项是最具成本效益的选项的相关概率仅为20%左右。对未来潜在治疗方案的敏感性分析表明，新的治疗方法可能会使筛查更具成本效益。
结论
结论侧重于从定义主要不确定性领域的角度对结果进行解释，这些领域被定义为疾病进展、临床表现率、筛查测试和配镜师有效性、治疗有效性和失明成本。未来的研究可能是最有针对性的评估如何常规数据可以用来描述临床表现率ARM. Other潜在的研究包括筛选和眼镜商的转介模式AMD和失明的成本研究的有效性的试点研究作为一个连续的关联视力恶化。
OBJECTIVES
To estimate the cost-effectiveness of screening for age-related macular degeneration (AMD) by developing a decision analytic model that incorporated and assessed all of the National Screening Committee criteria. A further objective was to identify the major areas of uncertainty in the model, and so inform future research priorities in this disease area.
DATA SOURCES
Major databases were searched in March 2004 and updated in January 2005.
REVIEW METHODS
Systematic literature reviews covered the epidemiology and natural history of AMD, the screening and treatment effectiveness and health-related quality of life relating to AMD. A hybrid cohort-individual sampling model was implemented to describe the range of pathways between the incidence of age-related maculopathy (ARM) and death via clinical presentation and treatment at different stages of the disease. As significant shortfalls in the data available from the literature were apparent, so a range of primary data sources were also used to populate the model. To obtain estimates for the value of parameters deemed to be within an expert's remit, data describing some parameters were elicited from relevant experts. The data identified informed probability distributions describing the uncertainty around the model parameters. To incorporate joint parameter uncertainty (i.e. correlations between parameters), the AMD natural history model was calibrated probabilistically. Randomly sampled sets of input parameters were assigned weights representing the accuracy of their predictions of a set of observed model outputs. The analysis of the AMD screening model estimated the costs, numbers of quality-adjusted life-years (QALYs) and cases of blindness in a general population sample of 50-year-olds over the remainder of their lifetime, for 16 alternative screening options (including no screening). The reference case analysis incorporated current treatment options of laser photocoagulation and photodynamic therapy. Sensitivity analyses describing six alternative sets of intervention strategies, based on horizon scanning of potential future treatments for AMD, were also undertaken.
RESULTS
There remains significant uncertainty about whether any form of screening for AMD is cost-effective. However, annual screening from age 60 years seems to provide the highest mean net benefits, but this is based on a cost-effectiveness estimate that has very poor precision (high levels of uncertainty). The probabilistic sensitivity analysis shows that the 95% credible interval for annual screening from age 60 years ranges from this option dominating the previous option to an incremental cost per QALY of over 0.5 million pounds sterling. Plotting a cost-effectiveness acceptability frontier shows that although annual screening from age 60 years has the highest net benefits at a value of QALY of 30,000 pounds sterling, the associated probability of this option being the most cost-effective option is only around 20%. The sensitivity analyses around potential future treatment options indicate that screening may become more cost-effective with the new treatments.
CONCLUSIONS
The conclusions focus on the interpretation of the results from the perspective of defining the major areas of uncertainty, which were defined as disease progression, rates of clinical presentation, screening test and optician effectiveness, treatment effectiveness, and costs of blindness. Future research may be best targeted at assessing how routine data may be used to describe clinical presentation rates of ARM. Other potential studies include a pilot study of the effectiveness of screening and opticians' referral patterns for AMD and a costing study of blindness as a continuum of association with deterioration in vision.
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DOI：
10.1001/jama.1996.03540140029022
发表时间：
1996-10
期刊：
JAMA
影响因子：
--
作者：
J. Seddon;W. Willett;F. Speizer;S. Hankinson
通讯作者：
J. Seddon;W. Willett;F. Speizer;S. Hankinson
影响因子：
13.7
作者：
Sunness, JS;Gonzalez-Baron, J;Bergman, A
通讯作者：
Bergman, A
影响因子：
13.7
作者：
Sunness, JS;Gonzalez-Baron, J;Applegate, CA
通讯作者：
Applegate, CA
影响因子：
2.2
作者：
J. Sunness
通讯作者：
J. Sunness