Huang Danmeng, Frank Steven J, Verma Vivek, Thaker Nikhil G, Brooks Eric D, Palmer Matthew B, Harrison Ross F, Deshmukh Ashish A, Ning Matthew S
Department of Radiation Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Management, Policy and Community Health, School of Public Health, University of Texas Health Science Center, Houston, TX, USA.
Int J Part Ther. 2021 Jun 25;8(1):339-353. doi: 10.14338/IJPT-20-00058.1. eCollection 2021 Summer.
Proton beam therapy (PBT) is associated with less toxicity relative to conventional photon radiotherapy for head-and-neck cancer (HNC). Upfront delivery costs are greater, but PBT can provide superior long-term value by minimizing treatment-related complications. Cost-effectiveness models (CEMs) estimate the relative value of novel technologies (such as PBT) as compared with the established standard of care. However, the uncertainties of CEMs can limit interpretation and applicability. This review serves to (1) assess the methodology and quality of pertinent CEMs in the existing literature, (2) evaluate their suitability for guiding clinical and economic strategies, and (3) discuss areas for improvement among future analyses.
PubMed was queried for CEMs specific to PBT for HNC. General characteristics, modeling information, and methodological approaches were extracted for each identified study. Reporting quality was assessed via the Consolidated Health Economic Evaluation Reporting Standards 24-item checklist, whereas methodologic quality was evaluated via the Philips checklist. The Cooper evidence hierarchy scale was employed to analyze parameter inputs referenced within each model.
At the time of study, only 4 formal CEMs specific to PBT for HNC had been published (2005, 2013, 2018, 2020). The parameter inputs among these various Markov cohort models generally referenced older literature, excluding many clinically relevant complications and applying numerous hypothetical assumptions for toxicity states, incorporating inputs from theoretical complication-probability models because of limited availability of direct clinical evidence. Case numbers among study cohorts were low, and the structural design of some models inadequately reflected the natural history of HNC. Furthermore, cost inputs were incomplete and referenced historic figures.
Contemporary CEMs are needed to incorporate modern estimates for toxicity risks and costs associated with PBT delivery, to provide a more accurate estimate of value, and to improve their clinical applicability with respect to PBT for HNC.
相对于传统光子放疗，质子束治疗（PBT）对头颈部癌（HNC）的毒性较小。前期交付成本更高，但PBT可通过将治疗相关并发症降至最低来提供卓越的长期价值。成本效益模型（CEM）估计新技术（如PBT）与既定护理标准相比的相对价值。然而，CEM的不确定性可能会限制其解释和适用性。本综述旨在（1）评估现有文献中相关CEM的方法和质量，（2）评估其对指导临床和经济策略的适用性，以及（3）讨论未来分析中有待改进的领域。
在PubMed中查询针对HNC的PBT的CEM。提取每个已识别研究的一般特征、建模信息和方法学方法。通过《综合卫生经济评估报告标准》24项清单评估报告质量，而通过飞利浦清单评估方法学质量。采用库珀证据等级量表分析每个模型中引用的参数输入。
在研究时，仅发表了4篇针对HNC的PBT的正式CEM（2005年、2013年、2018年、2020年）。这些不同的马尔可夫队列模型中的参数输入通常引用较旧的文献，排除了许多临床相关并发症，并对毒性状态应用了大量假设，由于直接临床证据有限，纳入了理论并发症概率模型的输入。研究队列中的病例数较少，一些模型的结构设计未能充分反映HNC的自然病史。此外，成本输入不完整且引用的是历史数据。
需要当代CEM纳入与PBT交付相关的毒性风险和成本的现代估计，以提供更准确的价值估计，并提高其在HNC的PBT方面的临床适用性。
