Department of Rheumatology, Selly Oak and Queen Elizabeth Hospitals Birmingham, University Hospital Birmingham NHS Foundation Trust, Oak Tree Lane Offices, Selly Oak, Birmingham, UK.
Rheumatology (Oxford). 2011 Sep;50 Suppl 4:iv48-iv52. doi: 10.1093/rheumatology/ker246.
Modelling cost-effectiveness of new drugs for RA has become increasingly prevalent and sophisticated. This situation has arisen largely because regulatory agencies, such as the National Institute for Health and Clinical Excellence, have demanded models from industry and have commissioned independent models. Many technical aspects of health economic models have converged-yet the results of models differ greatly. These differences can be accounted for in large part by differences in assumptions about the nature of patients likely to be treated; likely treatment sequences; likely responses to treatment; likely continuation on drug and likely disease progression, in particular. Such parameters cannot be fixed and evolve with changing practice and are ideally captured by contemporary data. Importantly, data from the local setting to which a health economic problem is applied are necessary, but in the absence of ideal sources, for the many contributions needed, considerable differences in opinion and biases are commonplace. In the final analysis, all models are just that, models, and as such an approximation of real life. Thus, although considerable heat is generated during debates about model parameters, model outputs may just yield sufficient light for regulatory agencies allocating resources.
RA 新药的成本效益建模已经变得越来越普遍和复杂。这种情况主要是由于监管机构(如国家卫生与临床优化研究所)向行业要求模型并委托独立模型所致。健康经济模型的许多技术方面已经趋同——但模型的结果却大相径庭。这些差异在很大程度上可以归因于对可能接受治疗的患者的性质、可能的治疗顺序、可能的治疗反应、可能继续使用药物和可能的疾病进展等假设的差异。这些参数不能固定,会随着实践的变化而变化,最好通过当代数据来捕捉。重要的是,应用于健康经济问题的当地数据是必要的,但在缺乏理想来源的情况下,对于许多需要的贡献,意见和偏见的差异是很常见的。归根结底,所有模型都只是模型,因此是对现实生活的一种近似。因此,尽管在关于模型参数的争论中会产生相当大的热度,但模型输出可能只是为监管机构分配资源提供了足够的信息。