医疗人工智能技术评估与监管的国际经验及启示

人工智能技术在生物技术、医学影像识别、药物研发、辅助诊断等领域发挥了重要作用。介绍了国际上关于医疗人工智能技术的评估与监管框架,包括英国的《数字卫生技术的证据标准框架》、美国的医疗器械独立软件预认证试点项目、欧盟的《可信赖人工智能道德准则》等,指出有效性与安全性是医疗人工智能技术评估与监管的前提,需制订符合我国国情的医疗人工智能设备分类标准,注重医疗人工智能产品的持续监管与风险控制。

Current Situation and Prospect of the Application of Real-World Evidence in Health Care

Objective To summarize the application of real-world evidence(RWE)in the medical and healthcare field of various countries,including relevant policies,application scenarios and application methods.Methods Relevant policies and application scenarios were obtained by consulting the official websites and public documents of various countries’healthcare institutions.Systematic literature retrieval was adopted to search PubMed,EMBASE,Cochrane Library,CNKI,CBM and Wanfang databases,and all papers related to real-world study and application were included.Then,these papers were classified and analyzed by country and application method.Results and Conclusion The RWE was mainly applied to supporting the preliminary approval of a new drug,expanding drug indications,accelerating approval or supporting conditional marketing authorizations and drug safety evaluation,etc.The United Kingdom,the United States,Germany,the Netherlands,Italy,Sweden,and France admitted RWE,but they treated the data obtained from RWE with caution.After systematic literature retrieval,a total of 701 articles were obtained,including relevant studies from 36 countries,among which the United States published 264 in total.The most common study was about using real-world data(RWD)to calculate treatment-related costs,which had a total of 259 studies.Secondly,158 articles were used for epidemiological analysis.Then,138 articles were about establishing risk models to analyze disease risk factors.A total of 70 articles were real-world efficacy evaluation of the drug treatment schemes,54 articles were about pharmacoeconomic evaluation with RWD as parameters.A total of 29 articles used RWD to build predictive models,and 15 articles used RWD to evaluate the health-related quality of life in patients.The application of RWE has been used widely in the medical and healthcare field of various countries.The application scenarios are gradually diversified,the application methods of RWD become mature,and the evidence quality of RWE is also improved greatly.

急性髓系白血病治疗药物经济学评价研究的系统评价

目的系统评价急性髓系白血病(AML)治疗的卫生经济学评价研究。方法计算机检索PubMed、EMbase、Cochrane Library、CBM、CNKI和WanFang Data数据库,以及CRD database卫生经济学专业数据库,并手工检索卫生经济学领域的相关杂志和各国HTA机构网站,全面搜集关于AML治疗药物的经济学评价研究。检索时限均从建库至2022年6月。参考CHEERS清单对文献质量进行评价。总结目前国内外已发表AML治疗卫生经济学评价的基本特征,分析评价模型结构及方法学差异,并比较药物的经济性。结果共纳入17个研究,分别从卫生体系、患者、全社会和医疗保险支付方角度出发进行成本-效果分析,评价模型比较统一,但在方法和结果报告上存在差异,质量有待加强。研究对象主要是去甲基化药、靶向药与传统化疗方案进行比较,以及不同化疗组合方案、不同药物剂量之间的比较。结论基于真实世界数据的研究主要聚焦于传统化疗方案,而马尔可夫等经济学评价模型,更多应用于近年研发的靶向药和去甲基化药物。与对照方案相比,基于阿糖胞苷的化疗方案、米哚妥林以及地西他滨更具有成本-效果。