Daly Marie-Josée, Elvidge Jamie, Chantler Tracey, Dawoud Dalia
Division of Anesthesiology, Department of Anesthesiology, Pharmacology, Intensive Care and Emergency Medicine, Geneva University Hospitals, Geneva, Switzerland.
National Institute for Health and Care Excellence (NICE), London, United Kingdom.
Front Pharmacol. 2022 May 11;13:887298. doi: 10.3389/fphar.2022.887298. eCollection 2022.
In the UK, 4.7 million people are currently living with diabetes. This is projected to increase to 5 million by 2025. The direct and indirect costs of T1DM and T2DM are rising, and direct costs already account for approximately 10% of the National Health Service (NHS) budget. The aim of this review is to assess the economic models used in the context of NICE's Technology Appraisals (TA) Programme of T1DM and T2DM treatments, as well as to examine their compliance with the American Diabetes Association's (ADA) guidelines on computer modelling. A review of the economic models used in NICE's TA programme of T1DM and T2DM treatments was undertaken. Relevant TAs were identified through searching the NICE website for published appraisals completed up to April 2021. The review also examined the associated Evidence Review Group (ERG) reports and Final Appraisal Documents (FAD), which are publicly accessible. ERG reports were scrutinised to identify major issues pertaining to the economic modelling. The FAD documents were then examined to assess how these issues reflected on NICE recommendations. Overall, 10 TAs pertaining to treatments of T1DM and T2DM were identified. Two TAs were excluded as they did not use economic models. Seven of the 8 included TAs related to a novel class of oral antidiabetic drugs (OADs), gliflozins, and one to continuous subcutaneous insulin infusion (CSII) devices. There is a lack of recent, robust data informing risk equations to enable the derivation of transition probabilities. Despite uncertainty surrounding its clinical relevance, bodyweight/BMI is a key driver in many T2DM-models. HbA1c's reliability as a predictor of hard outcomes is uncertain, chiefly for macrovascular complications. The external validity of T1DM is even less clear. There is an inevitable trade-off between the sophistication of models' design, their transparency and practicality. Economic models are essential tools to support decision-making in relation to market access and ascertain diabetes technologies' cost effectiveness. However, key structural and methodological issues exist. Models' shortcomings should be acknowledged and contextualised within the framework of technology appraisals. Diabetes medications and other technologies should also be subject to regular and consistent re-appraisal to inform disinvestment decisions. Artificial intelligence could potentially enhance models' transparency and practicality.
在英国，目前有470万人患有糖尿病。预计到2025年这一数字将增至500万。1型糖尿病（T1DM）和2型糖尿病（T2DM）的直接和间接成本都在上升，直接成本已占国民医疗服务体系（NHS）预算的约10%。本综述的目的是评估英国国家卫生与临床优化研究所（NICE）针对T1DM和T2DM治疗的技术评估（TA）计划中所使用的经济模型，并审查这些模型是否符合美国糖尿病协会（ADA）关于计算机建模的指南。对NICE的T1DM和T2DM治疗TA计划中使用的经济模型进行了综述。通过在NICE网站上搜索截至2021年4月已完成的公开评估报告，确定了相关的TA。该综述还审查了相关的证据审查小组（ERG）报告和最终评估文件（FAD），这些文件都是公开可获取的。对ERG报告进行了仔细审查，以确定与经济建模相关的主要问题。然后对FAD文件进行审查，以评估这些问题如何反映在NICE的建议中。总体而言，确定了10项与T1DM和T2DM治疗相关的TA。两项TA被排除，因为它们未使用经济模型。纳入的8项TA中有7项与一类新型口服抗糖尿病药物（OADs）格列净有关，另一项与持续皮下胰岛素输注（CSII）设备有关。目前缺乏最新的、可靠的数据来为风险方程提供信息，以推导转移概率。尽管体重/体重指数（BMI）的临床相关性存在不确定性，但它是许多T2DM模型中的关键驱动因素。糖化血红蛋白（HbA1c）作为严重后果预测指标的可靠性尚不确定，主要是对于大血管并发症而言。T1DM模型的外部有效性甚至更不明确。在模型设计的复杂性、透明度和实用性之间不可避免地要进行权衡。经济模型是支持市场准入决策和确定糖尿病技术成本效益的重要工具。然而，关键的结构和方法问题仍然存在。应在技术评估框架内认识到模型的缺点并将其置于具体情境中。糖尿病药物和其他技术也应定期且持续地接受重新评估，以为撤资决策提供依据。人工智能可能会提高模型的透明度和实用性。
