1、?-?曹海俊 博士Haijun Cao M.D，Ph.D副总裁，创新医疗解决事业部医渡科技集团开心生活科技公司Vice President,Innovative Healthcare SolutionHappy Life Technology,Yidu Tech曹海俊 博士Haijun Cao M.D Ph.D加州大学洛杉矶分校流行病学博士;复旦大学医学院 医学士，硕士;15+年研究经验，超过10年外企临床研究、医学事务、药物经济学和真实世界研究经验Ph.D in Epidemiology,University of California,Los AngelesBachelor of Medi

2、cine,Shanghai Medical University15+years research experience,and over 10 years experience in MNCpharma companies in clinical development,medical affairs,real world evidence and HEOR Overview外对照臂研究现状外对照臂的方法学23Methodology approach外对照臂的案例 Case Sharing1?版权 2022 HLT保留所有权利RCT?在特殊疾病领域，安慰剂或者对照臂都可能会存在伦理的挑战；在

3、罕见病等领域存在患者招募难、样本量小等问题；3-6 年6-7 年0.5-2 年临床前$25.7mPhase I$5.8mPhase II$11.7mPhase III$25.5m监管$6.9mPhase IV$13.3mRCT耗时长、花费高，且产出结果的外推性有限；患者招募/入组试验组对照组随机分组版权 2022 HLT保留所有权利?RWD?为单臂临床试验构建一组来自临床研究之外的接受不同干预措施的对比组数据源：既往RCT，EMR,医保数据，可穿戴设备，社交媒体，登记注册等试验比较两组人群对照结果试验组接受所研究的干预手段外部对照臂从外部数据获得的对照组别结果经数据处理后比较两组结果应用场景!

4、随机分组不可行；!在全面临床试验之前进行快速初步试验!对于罕见疾病或危及生命的疾病，安慰剂的使用有伦理问题；!存在严重的临床未满足需求或有限的治疗选择版权 2022 HLT保留所有权利2018.2美国Duchenne muscular dystrophy and related dystrophinopathies:developing drugs for treatment2019.3美国Rare diseasesnatural history studies for drug development2019.22019.92006.72014.52000.6&2001.5欧盟Guideli

5、ne on Clinical Trials in Small Populations美国Expedited programs for serious conditions美国ICH E10:choice of control group and related issues in clinical trialsalso published as an FDA final draft guidance dated May 2001美国Use of Bayesian statistics in medical device clinical trials2010.2美国Interacting wi

6、th the FDA on complex innovative trial designs for drugs and biological products2020.1美国Human gene therapy in rare diseases美国Expedited Programs for regenerative medicine therapies for serious conditions美国Rare diseases:common issues in drug development(Revision 1)中国真实世界证据支持药物研发与审评的指导原则（试行）?2020.12022