马兴华 岳立 孙尹晏

摘 要：关于采取医疗干预措施的决定是根据其成本和效果的证据作出的。由于各种原因，与医疗器械有关的证据可能受到限制。在设备生命周期早期，当设备基础最不成熟时，采用设备的决定可能会影响获得进一步证据以减少不确定性的前景。同样，拒绝一个设备将导致在实践中没有吸收，因此没有机会了解性能。决策选项，如“仅在研究中（OIR）”或“通过研究获得批准（AWR）”，可以通过允许患者尽早获得有希望的新技术来克服这些问题，同时在建立更多证据或学习之前，限制与错误决策相关的风险。本文阐述了与不确定性有关的问题和特定于设备的研究价值：学习曲线效应、增量设备创新、投资和不可恢复成本以及动态定价。我们展示了在何种情况下，只有在研究或批准的研究计划可能是一个适当的政策选择。我们还考虑如何在制造商和卫生部门之间分享额外研究的价值，以帮助告知合理预期谁将进行所需的研究。

关键词：医疗器械;医疗技术;不确定性;卫生技术评估

中图分类号：TH789 文献标识码：A 文章编号：1001-5922（2021）07-0159-04

Uncertainty Characteristics in Medical Device Evaluation and Determination of Future Research Needs

Ma Xinghua，Yue Li， Sun Yinyan

（3201 Hospital， Hanzhong 723000， China）

Abstract：The decision to take medical interventions is based on evidence of their costs and effects. For a range of reasons， evidence relating to medical devices may be limited. The decision to adopt a device early in its life cycle when the evidence base is least mature may iMPact on the prospects of acquiring further evidence to reduce uncertainties. Equally， rejecting a device will result in no uptake in practice and hence no chance to learn about performance. Decision options such as‘only in research（OIR） or ‘approval with research（AWR） can overcome these issues by allowing patients early access to promising new technologies while limiting the risks associated with making incorrect decisions until more evidence or learning is established. In this paper， we set out the issues relating to uncertainty and the value of research specific to devices： learning curve effects， incremental device innovation， investment and irrecoverable costs， and dynamic pricing. We show the circumstances under which an only in research or approval with research scheme may be an appropriate policy choice. We also consider how the value of additional research might be shared between the manufacturer and health sector to help inform who might reasonably be expected to conduct the research needed.

Key words：medical devices; medical technology; uncertainty; health technology assessment

0 引言

建立醫疗器械的临床有效性和成本有效性依赖于证据，这种证据通常比许多药品的证据更不广泛和数量更低。与制药公司不同的是，在获得上市许可之前，医疗器械通常只需要证明性能和安全性。设备的早期可用性可能看起来很有吸引力，因为它可以导致快速的临床应用;然而，当临床有效性和成本有效性不足时，医疗设备的投入使用就会具有很大的风险。……