Rare Diseases and Orphan Drugs Roadshow


13:30 PM-15:00 PM
  • 第一阶段
  • Phase I
    • Moderator
  • 张爱华, 南京医科大学附属儿童医院副院长
  • ZHANG Aihua, Vice President, Children's Hospital of Nanjing Medical University
13:30 PM-13:35 PM
  • 开幕致辞
  • 李林康，全国罕见病诊疗协作网办公室副主任、国家卫健委罕见病诊疗与保障专家委员会副主任委员兼办公室主任、中国医院协会副会长、中国罕见病联盟执行理事长
  • LI Linkang，Deputy Director of the National Collaboration Network of Rare Diseases Diagnosis and Treatment. Deputy Director and Office Director of the National Health Commission's Disease Diagnosis and Protection Experts. Vice President of the Chinese Hospital Association. Executive Director of the China Alliance for Rare Diseases.
13:35 PM-13:55 PM
  • 主旨报告
  • Keynote Speech
  • 罕见病药物临床研发指导原则的几点思考
  • Thoughts on the Guideline of the Orphan Drug Clinical Research Development
  • 国家食品药品监督管理局药品审评中心
  • Center for Drug Evaluation of the National Medical Products Administration
13:55 PM-14:15 PM
  • 主旨报告
  • Keynote Speech
  • 罕见病药物创新生态构建的思考
  • Thoughts on the Construction of Rare Disease Ecosystem in China
  • 薛群, 中国医药创新促进会药物研发专业委员会副主任委员、北海康成制药有限公司创始人、董事长兼CEO、中国罕见病联盟副理事长
  • James XUE，Vice-Chairman of PhIRDA Drug R&D Specialty Committee, Founder, Chairman of the Board, Director and CEO of CANbridge, Vice Director of the China Alliance for Rare Diseases
14:15 PM-15:00 PM
  • 主题讨论
  • Panel
  • 中国罕见病药物研发创新之路
  • The Road of Orphan Drug R&D in China
    • Moderator
  • 李林康, 全国罕见病诊疗协作网办公室副主任、国家卫健委罕见病诊疗与保障专家委员会副主任委员兼办公室主任、中国医院协会副会长、中国罕见病联盟执行理事长
  • LI Linkang, Deputy Director of the National Collaboration Network of Rare Diseases Diagnosis and Treatment. Deputy Director and Office Director of the National Health Commission's Disease Diagnosis and Protection Experts. Vice President of the Chinese Hospital Association. Executive Director of the China Alliance for Rare Diseases.
    • Panelists
  • 国家食品药品监督管理局药品审评中心
  • Center for Drug Evaluation of the National Medical Products Administration
  • 艾星, 国家食品药品监督管理局药品审评中心化药临床一部主审审评员
  • AI Xing, Chief Reviewer, Medical Review Department I, Center for Drug Evaluation of the National Medical Products Administration
  • 张爱华, 南京医科大学附属儿童医院副院长
  • ZHANG Aihua, Vice President, Children's Hospital of Nanjing Medical University
  • 薛群, 中国医药创新促进会药物研发专业委员会副主任委员、北海康成制药有限公司创始人、董事长兼CEO、中国罕见病联盟副理事长
  • James XUE, Vice-Chairman of PhIRDA Drug R&D Specialty Committee, Founder, Chairman of the Board, Director and CEO of CANbridge, Vice Director of the China Alliance for Rare Diseases
  • 陈智胜（线上）, 药明生物首席执行官
  • Chris CHEN, CEO, WuXi Biologics
15:00 PM-17:00 PM
  • 第二阶段
  • Phase II
    • Moderator
  • 刘洋, 中国医药创新促进会项目合作部主任
  • LIU Yang, Director of Project Co-operation, PHIRDA
15:00 PM-15:20 PM
  • 项目1
  • Project 1
  • 基因编辑疗法在罕见病领域的发展现状和潜力
  • The Development and Potential of Gene Editing Therapies for Rare Diseases
  • 袁鹏飞, 博雅辑因首席技术官
  • YUAN Pengfei，CTO, EdiGene
15:20 PM-15:40 PM
  • 项目2
  • Project 2
  • 单靶点全球首创新药开发: 通过靶向TSP-1阻断TGF-β激活
  • Developing Single-Target First-in-Class Treatment through Blocking TGF-β Activation by TSP-1 Technology
  • 韦沂均, 成都惠泰生物医药有限公司首席执行官
  • WEI Yijun，CEO, Chengdu Huitai Biomedical Co., Ltd.
15:40 PM-16:00 PM
  • 项目3
  • Project 3
  • 以患者为中心的中国孤儿药研发策略
  • Research and Development Strategy of Patient Centered Orphan Drugs in China
  • 谢生荣, 上海柯西医药科技发展有限公司总经理
  • Ron XIE，General Manager, Shanghai Cauchy Medical Technology Development Co., Ltd.
16:00 PM-16:20 PM
  • 项目4
  • Project 4
  • 以患者为中心，探索“药品+科技”的中国罕见病商业模式
  • Drug+Technology: a Patient-Centric Business Model for Rare Disease in China
  • 向宇, 琅钰集团首席执行官
  • Shawn XIANG, CEO, RareStone Group
16:20 PM-16:40 PM
  • 项目5
  • Project 5
  • 罕见，但不罕治：管控式供药，我们在路上
  • Winhealth Pharma, Bringing Real Impact to the Rare
  • 郝景辉, 香港维健医药集团有限公司联席首席执行官
  • Oliver HAO，Researcher，Co-CEO, Hongkong WinHealth Pharma Group Co., Ltd.
16:40 PM-17:00 PM
  • 项目6
  • Project 6
  • 罕见病患者长期用药依从性的解决办法
  • Compliance Plays Key Role in Long-Term Medication of Rare Diseases
  • 蒋鑫, 北京科信必成医药科技发展有限公司创新总监
  • JIANG Xin, Director, Pipeline&Strategy of CoSci Med-Tech Co., Ltd.
Specification:
  • Released: September 17, 2020
  • Last update: September 19, 2020
  • Version: 1.1