Department of Epidemiology, University Medical Center Groningen, University of Groningen, The Netherlands.
Stat Med. 2012 Oct 15;31(23):2733-44. doi: 10.1002/sim.5434. Epub 2012 Jul 16.
Many promising biomarkers for stratifying individuals at risk of developing a chronic disease or subsequent complications have been identified. Research into the potential cost-effectiveness of applying these biomarkers in actual clinical settings has however been lacking. Investors and analysts may improve their venture decision making should they have indicative estimates of the potential costs and effects associated with a new biomarker technology already at the early stages of its development. To assist in obtaining such estimates, this paper presents a general method for the early health technology assessment of a novel biomarker technology. The setting considered is that of primary prevention programs where initial screening to select high-risk individuals eligible for a subsequent intervention occurs, for example, prevention of type 2 diabetes. The method is based on quantifying the health outcomes and downstream healthcare consumption of all individuals who get reclassified as a result of moving from a screening variant based on traditional risk factors to a screening variant based on traditional risk factors plus a novel biomarker. As these individuals form well-defined subpopulations, a combination of disease progression modeling and sensitivity analysis can be used to perform an initial assessment of the maximum increase in screening cost for which the use of the new biomarker technology is still likely to be cost effective.
已经确定了许多有前途的生物标志物，可以对患有慢性疾病或后续并发症风险的个体进行分层。然而，在实际临床环境中应用这些生物标志物的潜在成本效益的研究却一直缺乏。如果投资者和分析师在新生物标志物技术的早期阶段就已经对其潜在成本和效果有了指示性的估计，那么他们可能会改进他们的风险投资决策。为了帮助获得这些估计，本文提出了一种用于新型生物标志物技术早期健康技术评估的通用方法。所考虑的环境是初级预防计划，例如，预防 2 型糖尿病，在此计划中，最初的筛选是为了选择有资格接受后续干预的高风险个体。该方法基于量化所有因从基于传统危险因素的筛选变体转变为基于传统危险因素加新型生物标志物的筛选变体而重新分类的个体的健康结果和下游医疗保健消费。由于这些个体形成了明确的亚人群，因此可以结合疾病进展建模和敏感性分析来对新生物标志物技术的使用仍然可能具有成本效益的最大筛查成本增加进行初步评估。
