药物全生命周期中的患者参与：以患者为中心的行业实践

近年来，医疗卫生行业都在向“以患者为中心”的价值体系转变，政府监管部门、医院、药企等利益相关方都迫切需要听到更多的患者声音，以更好地满足患者需求，助力健康中国2030计划实现。本书从四个方面阐述如何构建“以患者为中心”的理念和工作模式：①患者参与的内容概述，包括定义、形式与现状。②药物全生命周期各阶段患者参与的工作模式与企业案例。③患者参与的相关政策和合规考量等管理要求。④目前患者参与的挑战与机遇。本书是一本关注药物全生命周期中的患者参与议题的行业探索实践手册，是首次收集整理国内医药企业患者参与案例，与“以患者为中心”的医疗卫生行业价值体系高度一致。值得企业患者参与代表、药物经济学相关专业人员等参考和学习。

吴云，广州市红棉肿瘤和罕见病公益基金会“患者倡导”负责人， 医学硕士。前罗氏中国医学部“患者合作”团队负责人，前罗氏全球 “患者合作”核心团队成员。2000年进入药企工作，先后任职于复星、安斯泰来、罗氏等国内知名及跨国药企市场部和医学部，在患者教育、患者支持、患者参与等方面开展了大量的工作，并积极推动中国患者组织与国际患者组织的连结和交流，帮助提升中国患者组织能力建设和长期发展。在乳腺癌、淋巴瘤、脊髓性肌萎缩、血友病、移植、帕金森、阿尔茨海默病等多个领域，开展药物发展全生命周期中的患者合作，以支持和推动药物研发和准入，提升患者可及和获益。

目　录 第一章　药物全生命周期中的患者参与 ···········································1 一、为什么要开展患者参与 ·························································1 二、什么是药物全生命周期 ·························································4 三、如何开展患者参与 ·······························································9 第二章　药物研发阶段的患者参与 ················································31 一、概述 ················································································31 二、实践案例 ··········································································39 案例2.1　淋巴瘤之家参与企业药物研发的探索实践 ···················39 案例2.2　亨廷顿舞蹈症患者社群参与药物研发的探索 ················44 案例2.3　SMA患者组织参与药物临床研究实践 ························53 案例2.4　脑白质营养不良患者组织推动ALD在研药物引进 ·········58 案例2.5　Dravet综合征患者参与的实践与思考 ·························63 第三章　药物准入阶段的患者参与 ················································70 一、概述 ················································································70 二、实践案例 ··········································································79 案例3.1　淋巴瘤领域疾病负担及社会经济价值研究 ···················79 案例3.2　患者生存报告助力完善罕见病防治和保障 ···················88 案例3.3　罕见病患者组织如何参与政策推动 ····························95 案例3.4　基于调研报告的患者参与解决方案 ···························105 案例3.5　治疗偏好研究赋能患者参与医疗决策 ························110 案例3.6　全国范围大样本调查惠及中晚期结直肠癌患者 ·················117 案例3.7　通过卫生技术评估提升患者公共决策参与度 ···············122 第四章　临床应用阶段的患者参与 ···············································132 一、概述 ···············································································132 二、实践案例 ·········································································137 案例4.1　探索肿瘤患者院外管理闭环模式 ······························137 案例4.2　中国IBD患者关爱项目 ··········································145 案例4.3　基于疾病特点和患者需求的数字化医学服务 ···············149 案例4.4　多发性硬化患者全病程自助管理探索 ························152 案例4.5　赋能患者全程管理 ················································157 案例4.6　“以患者为中心”，让患者的“声音”被听见 ··············162 案例4.7　激发正能，赋能患者 ·············································167 第五章　患者参与的内外部赋能 ··················································170 一、概述 ···············································································170 二、实践案例 ·········································································176 案例5.1　建立与患者沟通的跨部门合作机制 ···························176 案例5.2　患者组织成长地图项目探索 ····································179 案例5.3　患者创新联盟与合作组织 ·······································190 后　记　患者参与的挑战和机遇 ··················································192