患者参与在药品监管中的作用——美欧日药品监管机构经验启示

摘要/Abstract

摘要：

患者参与药物开发和药品监管是公共卫生领域一个动态且不断发展的议题，是患者或其代表主动介入决策过程及流程的一种实践，国际上目前就“患者参与”尚无统一定义。“以患者为中心的药物研发”强调制药公司、研究机构以及政府监管者与患者之间的多方合作，倡导深入聆听患者意见，以促进药物研发的各个环节。患者参与有助于加强监管意见的深度和质量，提升监管决策的科学性、透明度和公众信任，促进监管机构与患者群体之间的相互尊重。患者视角在药物研发过程中可补充重要信息，并对药品审评审批产生影响。美国、欧盟、日本等创新活跃的国家或地区在促进患者参与药品监管，以及建立患者与监管机构之间的沟通渠道方面积累了丰富的经验。本研究在梳理美国、欧盟、日本药品监管机构“患者参与”实施情况的基础上，总结当前我国药品监管机构在“以患者为中心的药物研发”方面开展的工作，充分借鉴国际已有经验，并为“患者参与”在我国未来的发展和有效实施提出建议，以期未来患者的声音能更科学、更常规地融入我国药品监管活动。

关键词: 患者参与, 以患者为中心, 药物研发, 药品监管, 法规

Abstract:

Patient engagement in drug development and drug regulation is a dynamic and evolving topic in the field of public health. It is a practice where patients or their representatives actively intervene in the decision - making process and procedures. Currently， there is no unified international definition for "patient engagement". "Patient - focused drug development" emphasizes the multi - party cooperation among pharmaceutical companies， research institutions， government regulators， and patients. It advocates for in - depth listening to patients' opinions to promote every aspect of drug development. Patient engagement helps to enhance the depth and quality of regulatory opinions， improve the scientific nature， transparency， and public trust of regulatory decisions， and promote mutual respect between regulatory agencies and patient groups.The patient's perspective supplements important information during the drug development process and has an impact on drug review and approval. Innovation - active countries or regions such as the United States， the European Union， and Japan have accumulated rich experience in promoting patient engagement in drug regulation and establishing communication channels between patients and regulatory agencies. Based on a review of the implementation of "patient engagement" by drug regulatory agencies in the United States， the European Union， and Japan， this study summarizes the current work carried out in China's drug regulatory agencies on "patient - focused drug development". By fully drawing on existing international experience， it puts forward suggestions for the future development and effective implementation of "patient participation" in China， to help that patients' voices can be more scientifically and routinely integrated into China's drug regulatory activities in the future.

Key words: , Patient engagement； , Patient-focused； , Drug development； , Drug regulation； , Regulations

中图分类号:

R951

唐凌, 艾星, 宋媛媛, 杨志敏.

患者参与在药品监管中的作用——美欧日药品监管机构经验启示 [J]. 中国医药导刊, 2025, 27(1): 7-13.

TANG Ling, AI Xing, SONG Yuanyuan, YANG Zhimin.

The Role of Patient Engagement in Drug Regulation： Insights from the Experiences of Drug Regulatory Agencies in the United States， Europe， and Japan [J]. CHINESE JOURNAL OF MEDICINAL GUIDE, 2025, 27(1): 7-13.

参考文献

［1］ Food & Drug Administration.Patient-Focused Drug Development Glossary［EB/OL］.（2018-06-08）［2024-12-09］.https：//www.fda.gov/drugs/development-approval-process-drugs/patient-focused-drug-development-glossary.

［2］ Geißler J， Isham E， Hickey G， et al. Patient involvement in clinical trials［J］.Commun Med，2022，（2）：94-99.

［3］ Bertelsen N， Dewulf L， Ferrè S， et al.Patient engagement and patient experience data in regulatory review and health technology assessment：a global landscape review［J］.Ther Innov Regul Sci，2024，58（1）：63-78.

［4］ Perfetto EM， Burke L， Oehrlein EM， et al. Patient-focused drug development：a new direction for collaboration［J］.Med Care，2015，53（1）：9-17.

［5］ Busch IM， Rimondini M. Empowering patients and supporting health care providers-new avenues for high quality care and safety［J］.Int J Environ Res Public Health，2021，18（18）：9438-9441.

［6］ Mavris M， Furia Helms A， Bere N. Engaging patients in medicines regulation： a tale of two agencies［J］.Nat Rev Drug Discov，2019，18（12）：885-886.

［7］ Pharmaceuticals and Medical Devices Agency. Pharmaceuticals and Medical Devices Agency Guidance on Patient Participation ［EB/OL］.（2022-09-07）［2024-12-09］.https：//www.pmda.go.jp/files/000243407.pdf.

［8］ Food & Drug Administration.Evolution of patient engagement at the FDA［EB/OL］.（2024-07-29）［2024-12-09］.https：//www.fda.gov/patients/evolution-patient-engagement-fda.

［9］ Food & Drug Administration.About the FDA Patient Representative Program［EB/OL］.（2024-04-23）［2024-12-09］.https：//www.fda.gov/patients/learn-about-fda-patient-engagement/about-fda-patient-representative-program.

［10］ Food & Drug Administration.CDER Patient-Focused Drug Development［EB/OL］.（2024-08-14）［2024-12-09］.https：//www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development.

［11］ Food & Drug Administration.FDA-led Patient-Focused Drug Development （PFDD） Public Meetings［EB/OL］.（2024-02-14）［2024-12-09］.https：//www.fda.gov/industry/prescription-drug-user-fee-amendments/fda-led-patient-focused-drug-development-pfdd-public-meetings.

［12］ Food & Drug Administration. Externally-led Patient-Focused Drug Development Meetings［EB/OL］.（2022-07-29）［2024-12-09］.https：//www.fda.gov/industry/prescription-drug-user-fee-amendments/externally-led-patient-focused-drug-development-meetings.

［13］ 21st Century Cures Act［EB/OL］.（2016-12-13）［2024-12-09］.https：//www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf.

［14］ Crossnohere NL， Fischer R， Crossley E， et al. The evolution of patient-focused drug development and Duchenne muscular dystrophy［J］.Expert Rev Pharmacoecon Outcomes Res，2020，20（1）：57-68.

［15］ Mease C， Fermaglich LJ， Jackler K， et al. Determining commonalities in the experiences of patients with rare diseases：a qualitative analysis of US Food and Drug Administration Patient Engagement Sessions［J］.Patient，2024，17（1）：25-37.

［16］ Food & Drug Administration. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient's Voice in Medical Product Development and Regulatory Decision Making［EB/OL］.（2024-02-14）［2024-12-09］.https：//www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical.

［17］ Food & Drug Administration. Patient Engagement Advisory Committee［EB/OL］.（2020-10-13）［2024-12-09］.https：//www.fda.gov/advisory-committees/committees-and-meeting-materials/patient-engagement-advisory-committee.

［18］ Food & Drug Administration. Patient Engagement Cluster［EB/OL］.（2022-10-25）［2024-12-09］.https：//www.fda.gov/patients/learn-about-fda-patient-engagement/patient-engagement-cluster.

［19］ Food & Drug Administration. Memorandum of Understanding Between the U.S. Department of Health and Human Services， Food and Drug Administration， and the National Organization For Rare Disorders［EB/OL］.（2018-02-22）［2024-12-09］.https：//www.fda.gov/about-fda/non-profit-and-other-mous/mou-225-18-008.

［20］ Food & Drug Administration. Patient Engagement Collaborative［EB/OL］.（2024-11-01）［2024-12-09］.https：//www.fda.gov/patients/learn-about-fda-patient-engagement/patient-engagement-collaborative.

［21］ Food & Drug Administration. CDRH Patient and Caregiver Connection［EB/OL］.（2024-06-24）［2024-12-09］.https：//www.fda.gov/about-fda/division-patient-centered-development/cdrh-patient-and-caregiver-connection.

［22］ Food & Drug Administration. Patients Ask FDA［EB/OL］.（2024-11-21）［2024-12-09］.https：//www.fda.gov/patients/learn-about-fda-patient-engagement/patients-ask-fda.

［23］ Food & Drug Administration.Enhancing FDA's approach to Patient Engagement［EB/OL］.（2017-05-01）［2024-12-09］.https：//fda.report/media/109891/Enhancing-FDA_s-approach-toPatient-Engagement-%28PDF%29.pdf+%2Autf-8%27%27Enhancing-FDA%E2%80%99s-approach-toPatient-Engagement-%28PDF%29.pdf.

［24］ Food & Drug Administration. Patient Affairs Staff Enhancing FDA Patient Engagement［EB/OL］.（2018-04-03）［2024-12-09］.https：//www.fda.gov/media/112220/download.

［25］ Chalasani M， Vaidya P， Mulli T. Enhancing the incorporation of the patient's voice in drug development and evaluation［J］.Res Involv Engagem，2018，（4）：10-16.

［26］ Food & Drug Administration.The Voice of the Patient： A Series of Reports from FDA's Patient-Focused Drug Development Initiative［EB/OL］.（2024-02-14）［2024-12-09］.https：//www.fda.gov/forindustry/userfees/prescriptiondruguserfee/ucm368342.htm.

［27］ European Medicines Agency. Engagement Framework： EMA and patients， consumers and their organisations［EB/OL］.（2022-01-20）［2024-12-09］.https：//www.ema.europa.eu/en/documents/other/engagement-framework-european-medicines-agency-and-patients-consumers-and-their-organisations_en.pdf.

［28］ Murphy A， Bere N， Vamvakas S，et al. The added value of patient engagement in early dialogue at EMA： scientific advice as a case study［J］.Front Med （Lausanne），2022，8：811855.

［29］ The Council for International Organizations of Medical Sciences. Patient involvement in the development， regulation and safe use of medicines［EB/OL］.（2022-11-15）［2024-12-09］.https：//www.eatg.org/wp-content/uploads/2022/09/cioms-wg-xi-patient-involvement-report-06sept2022.pdf.

［30］ Pharmaceuticals and Medical Devices Agency. The outlook for Patient Involvement in Medical Device Development-Japanese Regulatory View［EB/OL］.（2024-11-18）［2024-12-09］.https：//www.pmda.go.jp/files/000245371.pdf.

［31］ Pharmaceuticals and Medical Devices Agency. Pharmaceuticals and Medical Devices Agency Guidance on Patient Participation ［EB/OL］.（2022-09-07）［2024-12-09］.https：//www.pmda.go.jp/files/000243407.pdf.

［32］国务院. 国务院关于改革药品医疗器械审评审批制度的意见（国发〔2015〕44号）［EB/OL］.（2018-08-18）［2024-12-09］.https：//www.gov.cn/zhengce/content/2015-08/18/content_10101.htm.

［33］国家药品监督管理局药品审评中心.国家药监局药审中心关于发布《以临床价值为导向的抗肿瘤药物临床研发指导原则》的通告（2021年第46号）［EB/OL］.（2021-11-15）［2024-12-09］.https：//www.cde.org.cn/main/news/viewInfoCommon/ef7bfde96c769308ad080bb7ab2f538e.

［34］国家药品监督管理局药品审评中心.国家药监局药审中心关于发布《组织患者参与药物研发的一般考虑指导原则（试行）》的通告（2022年第46号）［EB/OL］.（2022-11-21）［2024-12-09］.https：//www.cde.org.cn/main/news/viewInfoCommon/41c7a683e4d0dcca28bccadc47096d2a.

［35］国家药品监督管理局药品审评中心. 国家药监局药审中心关于发布《以患者为中心的药物临床试验设计技术指导原则（试行）》《以患者为中心的药物临床试验实施技术指导原则（试行）》《以患者为中心的药物获益-风险评估技术指导原则（试行）》的通告（2023年第44号）［EB/OL］.（2023-07-27）［2024-12-09］.https：//www.cde.org.cn/main/news/viewInfoCommon/42c008e28f7004cd19b73949142380bd.

［36］ Pitts PJ. Towards meaningful engagement for the patient Voice［J］. Patient，2019，12（4）：361-363.

［37］ Jobson E， Garcia M， Sharek D，et al. Embedding patient engagement in the R&D process of a life sciences company through co-creation with a patient expert R&D board： a case study［J］.Res Involv Engagem，2024，10（1）：116-133.

［38］ Stergiopoulos S， Michaels DL， Kunz BL， et al. Measuring the impact of patient engagement and patient centricity in clinical research and development［J］.Ther Innov Regul Sci， 2020，54（1）：103-116.

［39］ Auwal FI， Copeland C， Clark EJ，et al. A systematic review of models of patient engagement in the development and life cycle management of medicines［J］.Drug Discov Today，2023，28（9）：1-9.

［40］ Levitan B， Getz K， Eisenstein EL， et al. Assessing the fnancial value of patient engagement： a quantitative approach from CTTI's patient groups and clinical trials project［J］.Ther Innov Regul Sci，2018，52：220-229.

［41］ Klein B， Perfetto EM， Oehrlein EM， et al. Measuring and demonstrating the value of patient engagement across the medicines lifecycle：a patient engagement impact measurement framework［J］.Patient. 2025，18（1）：3-18.

[1]	陈锋, 吴欣然. 药品监管领域大语言模型（LLM）一体化建设策略研究 [J]. 中国医药导刊, 2025, 27(1): 1-6.
[2]	赵宝龙, 华坚, 张长风. 基于CRISPR的细胞产品的临床应用及监管挑战 [J]. 中国医药导刊, 2025, 27(1): 101-108.
[3]	徐仁恩, 熊海燕, 宋东杰, 饶龙. 大数据和AI技术在江西省药品智慧监管中的探索和应用 [J]. 中国医药导刊, 2024, 26(7): 637-642.
[4]	唐竹, 刘勤, 缪锦辉, 丛薇, 方东来, 马馥娟, 李劲. 远程监管在药品监管和企业管理的作用与实践 [J]. 中国医药导刊, 2024, 26(7): 643-649.
[5]	刘涛, 张钰, 李钻, 种璟, 靳泽宇, 杨东, 肖楠, 孙玲. 人工智能在药品审评中的应用展望 [J]. 中国医药导刊, 2024, 26(5): 504-510.
[6]	徐子涵a, 赵志霞ab, 刘丽宏ab. 美欧药品智能监管服务平台建设及对我国的启示 [J]. 中国医药导刊, 2024, 26(11): 1065-1070.
[7]	周怡a, 夏天雨b, 尤翀, 周晓华ab, 国家重点研发计划“面向药品现代化监管的智能化服务平台”项目团队. 面向药品现代化监管的智能化服务平台的建设、应用与思考 [J]. 中国医药导刊, 2024, 26(11): 1075-1079.
[8]	冀亦默, 林琳, 徐平洋. “一云多池”架构下药监数据备份方案与流程研究 [J]. 中国医药导刊, 2024, 26(10): 1060-1064.
[9]	田蕊, 郭亮, 徐匡根, . 完善新时代药品执法体系机制思考[J]. 中国医药导刊, 2023, 25(7): 714-722.
[10]	肖牧原, 胡婕, 金永宏. 行政调解在药品监管投诉领域运用情况的研究[J]. 中国医药导刊, 2023, 25(4): 368-374.
[11]	张然, 陶群山, 罗京京. 安徽省药品上市许可持有人质量管理体系调研分析[J]. 中国医药导刊, 2023, 25(12): 1213-1218.
[12]	王木兰, 杨洁, 王欢, 王雅丽, 朱江, 马小燕. 职业化专业化药品检查员队伍建设研究现状和趋势[J]. 中国医药导刊, 2023, 25(11): 1092-1099.
[13]	付美芳, 王劲, 赵丹彤, 牛忻, 王素香. 市售牡丹皮配方颗粒质量评价与分析[J]. 中国医药导刊, 2023, 25(11): 1104-1111.
[14]	胡位歆, 和金周. 国内外制药企业质量管理成熟度现状及对我国药品生产监管的启示[J]. 中国医药导刊, 2022, 24(8): 740-745.
[15]	王广平. 药品全生命周期药物警戒体系研究与思考[J]. 中国医药导刊, 2022, 24(7): 637-642.

快速检索引用检索图表检索高级检索