Department of Psychosocial Research and Epidemiology, Netherlands Cancer Institute-Antoni van Leeuwenhoek Hospital (NKI-AVL), Amsterdam, The Netherlands.
Value Health. 2013 Jul-Aug;16(5):720-8. doi: 10.1016/j.jval.2013.04.013.
In early stages of development of new medical technologies, there are conceptually separate but related societal decisions to be made concerning adoption, further development (i.e., technical improvement), and research (i.e., clinical trials) of new technologies. This article presents a framework to simultaneously support these three decisions from a societal perspective. The framework is applied to the 70-gene signature, a gene-expression profile for breast cancer, deciding which patients should receive adjuvant systemic therapy after surgery. The "original" signature performed on fresh frozen tissue (70G-FFT) could be further developed to a paraffin-based signature (70G-PAR) to reduce test failures.
A Markov decision model comparing the "current" guideline Adjuvant Online (AO), 70G-FFT, and 70G-PAR was used to simulate 20-year costs and outcomes in a hypothetical cohort in The Netherlands. The 70G-PAR strategy was based on projected data from a comparable technology. Incremental net monetary benefits were calculated to support the adoption decision. Expected net benefit of development for the population and expected net benefit of sampling were calculated to support the development and research decision.
The 70G-PAR had the highest net monetary benefit, followed by the 70G-FFT. The population expected net benefit of development amounted to €91 million over 20 years (assuming €250 development costs per patient receiving the test). The expected net benefit of sampling amounted to €61 million for the optimal trial (n = 4000).
We presented a framework to simultaneously support adoption, development, and research decisions in early stages of medical technology development. In this case, the results indicate that there is value in both further development of 70G-FFT into 70G-PAR and further research.
在新医疗技术开发的早期阶段，需要从社会角度做出三个概念上相互独立但又相互关联的决策，分别涉及新技术的采用、进一步开发（即技术改进）和研究（即临床试验）。本文提出了一个框架，可同时支持从社会角度做出这三个决策。该框架应用于 70 基因标志物（一种乳腺癌基因表达谱），用于决定手术后哪些患者应接受辅助系统性治疗。“原始”在新鲜冷冻组织上（70G-FFT）运行的签名可以进一步开发为基于石蜡的签名（70G-PAR），以降低测试失败率。
使用比较当前指南 Adjuvant Online（AO）、70G-FFT 和 70G-PAR 的马尔可夫决策模型，对荷兰的一个假设队列进行 20 年的成本和结果模拟。70G-PAR 策略基于类似技术的预测数据。增量净货币收益用于支持采用决策。为支持开发和研究决策，计算了人群的预期开发净收益和预期抽样净收益。
70G-PAR 的净货币收益最高，其次是 70G-FFT。20 年内，人群的开发预期净收益达到 9100 万欧元（假设每位接受测试的患者的开发成本为 250 欧元）。最佳试验（n = 4000）的预期抽样净收益为 6100 万欧元。
我们提出了一个框架，可同时支持医疗技术开发早期阶段的采用、开发和研究决策。在这种情况下，结果表明进一步开发 70G-FFT 成 70G-PAR 以及进一步研究都具有价值。
