Centre for Health Economics, University of York, York, UK.
Int J Health Policy Manag. 2020 May 1;9(5):215-217. doi: 10.15171/ijhpm.2019.119.
Early economic modelling has long been recommended to aid research and development (R&D) decisions in medical innovation, although they are less frequently published and critically appraised. A review of 30 innovations by Grutters et al provides an opportunity to evaluate how early models are used in practice. The evidence of early models can be used to inform two types of decision: to continue development ("stop or go") or to alter future R&D activities. I argue that early models have limited use in stop or go decisions, as less resource and data undermine the reliability of the models' indicative estimates of cost-effectiveness. Whilst they are far more useful for informing future R&D directions, the best techniques available from statistical decision science, such as value of information analysis, are not regularly used. It is highly recommended that early models adopt these methods to best deal with uncertainty, quantify the potential value of further research, identify areas of study with the greatest potential benefit and generate recommendations on study design and sample size.
早期经济建模长期以来一直被建议用于辅助医学创新的研究和开发（R&D）决策，尽管它们的发表和批判性评估频率较低。Grutters 等人对 30 项创新的回顾提供了一个评估早期模型在实践中如何使用的机会。早期模型的证据可用于告知两种类型的决策：继续开发（“停止或继续”）或改变未来的 R&D 活动。我认为，早期模型在停止或继续决策中的使用有限，因为资源和数据较少会降低模型对成本效益的指示性估计的可靠性。虽然它们对于告知未来的 R&D 方向更有用，但统计决策科学中提供的最佳技术，如信息价值分析，并未得到定期使用。强烈建议早期模型采用这些方法来最好地处理不确定性，量化进一步研究的潜在价值，确定具有最大潜在收益的研究领域，并就研究设计和样本量提出建议。
