Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Pharmacoeconomics. 2023 Jun;41(6):633-650. doi: 10.1007/s40273-023-01256-9. Epub 2023 Mar 8.
Bladder cancer is common among current and former smokers. High bladder cancer mortality may be decreased through early diagnosis and screening. The aim of this study was to appraise decision models used for the economic evaluation of bladder cancer screening and diagnosis, and to summarise the main outcomes of these models.
MEDLINE via PubMed, Embase, EconLit and Web of Science databases was systematically searched from January 2006 to May 2022 for modelling studies that assessed the cost effectiveness of bladder cancer screening and diagnostic interventions. Articles were appraised according to Patient, Intervention, Comparator and Outcome (PICO) characteristics, modelling methods, model structures and data sources. The quality of the studies was also appraised using the Philips checklist by two independent reviewers.
Searches identified 3082 potentially relevant studies, which resulted in 18 articles that met our inclusion criteria. Four of these articles were on bladder cancer screening, and the remaining 14 were diagnostic or surveillance interventions. Two of the four screening models were individual-level simulations. All screening models (n = 4, with three on a high-risk population and one on a general population) concluded that screening is either cost saving or cost effective with cost-effectiveness ratios lower than $53,000/life-years saved. Disease prevalence was a strong determinant of cost effectiveness. Diagnostic models (n = 14) assessed multiple interventions; white light cystoscopy was the most common intervention and was considered cost effective in all studies (n = 4). Screening models relied largely on published evidence generalised from other countries and did not report the validation of their predictions to external data. Almost all diagnostic models (n = 13 out of 14) had a time horizon of 5 years or less and most of the models (n = 11) did not incorporate health-related utilities. In both screening and diagnostic models, epidemiological inputs were based on expert elicitation, assumptions or international evidence of uncertain generalisability. In modelling disease, seven models did not use a standard classification system to define cancer states, others used risk-based, numerical or a Tumour, Node, Metastasis classification. Despite including certain components of disease onset or progression, no models included a complete and coherent model of the natural history of bladder cancer (i.e. simulating the progression of asymptomatic primary bladder cancer from cancer onset, i.e. in the absence of treatment).
The variation in natural history model structures and the lack of data for model parameterisation suggest that research in bladder cancer early detection and screening is at an early stage of development. Appropriate characterisation and analysis of uncertainty in bladder cancer models should be considered a priority.
膀胱癌在当前吸烟者和曾经吸烟者中较为常见。早期诊断和筛查可能降低膀胱癌的高死亡率。本研究旨在评估膀胱癌筛查和诊断的经济评估中使用的决策模型,并总结这些模型的主要结果。
通过 MEDLINE 下的 PubMed、Embase、EconLit 和 Web of Science 数据库,从 2006 年 1 月至 2022 年 5 月系统地检索了评估膀胱癌筛查和诊断干预措施成本效益的建模研究。根据患者、干预、比较和结果(PICO)特征、建模方法、模型结构和数据源对文章进行评估。两名独立评审员还使用飞利浦检查表评估了研究的质量。
检索到 3082 篇潜在相关研究,其中有 18 篇符合纳入标准。其中 4 篇是关于膀胱癌筛查的,其余 14 篇是关于诊断或监测干预的。4 个筛查模型中有 2 个是个体水平的模拟。所有的筛查模型(n=4,其中 3 个用于高危人群,1 个用于一般人群)都得出筛查要么是节省成本的,要么是具有成本效益的,其成本效益比低于每挽救 1 个生命年 53000 美元。疾病流行率是成本效益的一个重要决定因素。诊断模型(n=14)评估了多种干预措施;白光膀胱镜检查是最常见的干预措施,在所有研究中(n=4)都被认为是具有成本效益的。筛查模型主要依赖于来自其他国家的已发表证据,并且没有报告对其预测进行外部数据验证。几乎所有的诊断模型(n=14 中的 13 个)的时间范围都在 5 年或更短,而且大多数模型(n=11 个)没有纳入健康相关效用。在筛查和诊断模型中,流行病学输入基于专家推断、假设或不确定普遍适用性的国际证据。在建模疾病时,7 个模型没有使用标准的分类系统来定义癌症状态,其他模型使用基于风险的、数值的或肿瘤、淋巴结、转移(TNM)分类。尽管纳入了疾病发生或进展的某些组成部分,但没有模型纳入膀胱癌自然史的完整和连贯模型(即模拟无症状原发性膀胱癌从癌症发生到进展,即无治疗情况下的进展)。
自然史模型结构的差异以及模型参数化的数据缺乏表明,膀胱癌早期检测和筛查的研究仍处于早期阶段。应优先考虑对膀胱癌模型中的不确定性进行适当的特征描述和分析。