York Health Economics Consortium, University of York, York, UK.
Centre for Health Economics, University of York, Heslington, York, YO10 5DD, UK.
Pharmacoeconomics. 2018 Dec;36(12):1439-1451. doi: 10.1007/s40273-018-0686-6.
Precision medicine allows healthcare interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information, or treatment response. We analysed what developments are expected in precision medicine over the next decade and considered the implications for health technology assessment (HTA) agencies.
We performed a pragmatic literature search to account for the large size and wide scope of the precision medicine literature. We refined and enriched these results with a series of expert interviews up to 1 h in length, including representatives from HTA agencies, research councils and researchers designed to cover a wide spectrum of precision medicine applications and research.
We identified 31 relevant papers and interviewed 13 experts. We found that three types of precision medicine are expected to emerge in clinical practice: complex algorithms, digital health applications and 'omics'-based tests. These are expected to impact upon each stage of the HTA process, from scoping and modelling through to decision-making and review. The complex and uncertain treatment pathways associated with patient stratification and fast-paced technological innovation are central to these effects.
Innovation in precision medicine promises substantial benefits but will change the way in which some health services are delivered and evaluated. The shelf life of guidance may decrease, structural uncertainty may increase and new equity considerations will emerge. As biomarker discovery accelerates and artificial intelligence-based technologies emerge, refinements to the methods and processes of evidence assessments will help to adapt and maintain the objective of investing in healthcare that is value for money.
精准医学可根据患者的疾病易感性、诊断或预后信息或治疗反应,将医疗干预措施针对特定患者群体进行定制。我们分析了未来十年精准医学的预期发展,并考虑了其对卫生技术评估(HTA)机构的影响。
我们进行了务实的文献检索,以考虑到精准医学文献的规模大和范围广。我们通过一系列专家访谈对这些结果进行了精炼和丰富,访谈时长长达 1 小时,专家包括 HTA 机构、研究理事会和研究人员的代表,旨在涵盖广泛的精准医学应用和研究领域。
我们确定了 31 篇相关论文,并采访了 13 名专家。我们发现,三种类型的精准医学预计将在临床实践中出现:复杂算法、数字健康应用和基于“组学”的测试。这些预计将影响 HTA 过程的各个阶段,从范围界定和建模到决策和审查。与患者分层和快速技术创新相关的复杂和不确定的治疗途径是这些影响的核心。
精准医学的创新有望带来巨大的收益,但将改变某些卫生服务的提供和评估方式。指导的有效期可能会缩短,结构不确定性可能会增加,新的公平性考虑因素将会出现。随着生物标志物发现的加速和基于人工智能的技术的出现,对证据评估方法和流程的改进将有助于适应和维护投资于具有成本效益的医疗保健的目标。