PenTAG/PenCLAHRC, University of Exeter, Exeter, UK.
BMJ Open. 2013 May 28;3(5):e002905. doi: 10.1136/bmjopen-2013-002905.
To describe the development process for defining an appropriate model structure for the economic evaluation of test-treatment strategies for patients with monogenic diabetes (caused by mutations in the GCK, HNF1A or HNF4A genes).
Experts were consulted to identify and define realistic test-treatment strategies and care pathways. A systematic assessment of published diabetes models was undertaken to inform the model structure.
National Health Service in England and Wales.
Experts in monogenic diabetes whose collective expertise spans the length of the patient care pathway.
A defined model structure, including the test-treatment strategies, and the selection of a published diabetes model appropriate for the economic evaluation of strategies to identify patients with monogenic diabetes.
Five monogenic diabetes test-treatment strategies were defined: no testing of any kind, referral for genetic testing based on clinical features as noted by clinicians, referral for genetic testing based on the results of a clinical prediction model, referral for genetic testing based on the results of biochemical and immunological tests, referral for genetic testing for all patients with a diagnosis of diabetes under the age of 30 years. The systematic assessment of diabetes models identified the IMS CORE Diabetes Model (IMS CDM) as a good candidate for modelling the long-term outcomes and costs of the test-treatment strategies for monogenic diabetes. The short-term test-treatment events will be modelled using a decision tree which will feed into the IMS CDM.
Defining a model structure for any economic evaluation requires decisions to be made. Expert consultation and the explicit use of critical appraisal can inform these decisions. Although arbitrary choices have still been made, decision modelling allows investigation into such choices and the impact of assumptions that have to be made due to a lack of data.
描述为单基因糖尿病（由 GCK、HNF1A 或 HNF4A 基因突变引起）的治疗策略的经济评估定义合适模型结构的开发过程。
咨询专家以确定和定义现实的治疗策略和护理途径。对已发表的糖尿病模型进行系统评估，为模型结构提供信息。
英格兰和威尔士国民保健服务。
具有单基因糖尿病专业知识的专家，他们的集体专业知识涵盖了患者护理途径的各个方面。
定义了模型结构，包括治疗策略，并选择了适合单基因糖尿病治疗策略经济评估的已发表糖尿病模型。
定义了五种单基因糖尿病治疗策略：不进行任何类型的检测、根据临床医生记录的临床特征进行基因检测、根据临床预测模型的结果进行基因检测、根据生化和免疫测试的结果进行基因检测、对所有 30 岁以下诊断为糖尿病的患者进行基因检测。对糖尿病模型的系统评估确定了 IMS CORE Diabetes Model（IMS CDM）是模拟单基因糖尿病治疗策略的长期结果和成本的良好候选模型。短期治疗策略将使用决策树进行建模，该决策树将输入到 IMS CDM 中。
为任何经济评估定义模型结构都需要做出决策。专家咨询和明确使用关键评估可以为这些决策提供信息。尽管仍然做出了任意的选择，但决策模型允许对这些选择以及由于缺乏数据而必须做出的假设的影响进行调查。
