Introduction of patient-focused drug development related concepts_Chinese Journal of Evidence-Based Medicine

Authors：

WU Xiaolei ¹ , SHI Menglong ¹ , ZHANG Chenyao ¹ , MA Yucong ¹ , DU Liang ² , LIU Binglin ³ ,  CAO Lujia ¹ ,  ZHANG Junhua ¹

1. Evidence-based Medicine Center, Tianjin University of Traditional Chinese Medicine, Tianjin 301617, P. R. China;
2. Chinese Evidence-based Medicine Center, West China Hospital of Sichuan University, Chengdu 610041, P. R. China;
3. Center for Drug Evaluation, China Food and Drug Administration, Beijing 10002, P. R. China;

Corresponding author：

CAO Lujia, Email: caolujia0825@163.com; ZHANG Junhua, Email: zjhtcm@foxmail.com

Keywords：

Patient-focused drug development (PFDD); Clinical outcome assessment; Patient experience data

DOI：

10.7507/1672-2531.202307066

Video：

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The patient, as the person who experiences the disease first-hand, has the most direct and accurate experience of the pain of the disease and the most accurate need for health products. Although there is a vast array of technological means to combat disease and maintain health, the human burden of disease has not been reduced and the health needs of patients have not been fully met. Therefore, "patient-focused drug development" is imperative. Gathering comprehensive information from patients through multiple channels and incorporating this information into the entire drug development process can help ensure that patients’ experiences, perspectives, needs and priorities are taken into account and valued. This article will introduce the concept, development process and the specific problems it faces in patient-focused drug development.

Citation： WU Xiaolei, SHI Menglong, ZHANG Chenyao, MA Yucong, DU Liang, LIU Binglin, CAO Lujia, ZHANG Junhua. Introduction of patient-focused drug development related concepts. Chinese Journal of Evidence-Based Medicine, 2023, 23(12): 1472-1477. doi: 10.7507/1672-2531.202307066 Copy

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32.	尹立强, 赵杨升, 马伟. "以患者为中心"在医疗器械研发中的价值和应用. 中国医疗器械信息, 2021, 27(1): 36-38.
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34.	Benjamin K, Vernon MK, Patrick DL, et al. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA emerging good practices task force report. Value Health, 2017, 20(7): 838-855.
35.	EMA. Draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies.
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37.	Timmermans S. The engaged patient: the relevance of patient-physician communication for twenty-first-century health. J Health Soc Behav, 2020, 61(3): 259-273.
38.	Greenhalgh T. Narrative based medicine: narrative based medicine in an evidence based world. BMJ, 1999, 318(7179): 323-325.
39.	崔鸿雁, 吕兰青, 邵明义等. 以患者为中心进行中医药临床研究的思考与探讨. 中华中医药杂志, 2022, 37(12): 7224-7227.
40.	FDA. Clinical outcome assessment (COA) compendium.

1. 张俊华, 郑文科, 张伯礼, 等. 真实世界研究. 世界中医药, 2019, 14(12): 3101-3105.
2. Evangelidis N, Tong A, Howell M, et al. International survey to establish prioritized outcomes for trials in people with coronavirus disease 2019. Crit Care Med, 2020, 48(11): 1612-1621.
3. 谢静, 王焕玲. 以患者为中心的临床试验: 医药研发新模式. 协和医学杂志, 2021, 12(6): 849-853.
4. Sharma NS. Patient centric approach for clinical trials: current trend and new opportunities. Perspect Clin Res, 2015, 6(3): 134-138.
5. 郑航. 临床试验行业的基本特征与发展趋势. 2018年中国药学会药物临床评价研究专业委员会学术年会暨临床研究高峰论坛论文集, 2018: 107-110.
6. Patalano F, Gutzwiller FS, Shah B, et al. Gathering structured patient insight to drive the pro strategy in COPD: patient-centric drug development from theory to practice. Adv Ther, 2020, 37(1): 17-26.
7. FDA. Decentralized clinical trials for drugs, biological products, and devices. 2023.
8. Perfetto EM, Burke L, Oehrlein EM, et al. Patient-focused drug development: a new direction for collaboration. Med Care, 2015, 53(1): 9-17.
9. FDA. Food and Drug Administration safety and innovation act, pub l no. 122-144, 126 stat 993. 2012.
10. FDA. PDUFA reauthorization performance goals and procedures fiscal years 2013-2017.
11. FDA. Plan for issuance of patient focused drug development guidance.
12. Warner K, See W, Haerry D, et al. EUPATI guidance for patient involvement in medicines research and development (R&D); guidance for pharmaceutical industry-led medicines R&D. Front Med (Lausanne), 2018, 5: 270.
13. FDA. Patient-focused drug development: collecting comprehensive and representative input guidance for industry, Food and Drug Administration staff, and other stakeholders.
14. FDA. Patient-focused drug development: methods to identify what is important to patients guidance for industry, Food and Drug Administration staff, and other stakeholders.
15. FDA. Methods to identify what is important to patients & select, develop or modify fit- for-purpose clinical outcomes assessments.
16. 宋彩梅. 美国食品药品监督管理局以患者为中心的药物开发系列指导原则1-《综合和有代表性的信息收集指导原则》介绍. 中国临床药理学杂志, 2021, 37(17): 2381-2384.
17. FDA. Patient-focused drug development: incorporating clinical outcome assessments into endpoints for regulatory decision-making.
18. ICH. Reflection paper on patient-focused drug development for public consultation.
19. EMA. Multi-stakeholder workshop: patient experience data in medicines development and regulatory decision-making.
20. 国家药品监督管理局药品审评中心. 《以患者为中心的临床试验设计技术指导原则(征求意见稿)》. 2022.
21. 国家药品监督管理局药品审评中心. 《以患者为中心的临床试验获益-风险评估技术指导原则(征求意见稿)》. 2022.
22. 国家药品监督管理局药品审评中心. 《以患者为中心的临床试验实施技术指导原则(征求意见稿)》. 2022.
23. 国家药品监督管理局药品审评中心. 《组织患者参与药物研发的一般考虑指导原则(试行)》. 2022.
24. 国家药品监督管理局药品审评中心. 《以患者为中心的药物临床试验设计技术指导原则(试行)》. 2023.
25. 国家药品监督管理局药品审评中心. 《以患者为中心的药物临床试验实施技术指导原则(试行)》. 2023.
26. 国家药品监督管理局药品审评中心. 《以患者为中心的药物获益-风险评估技术指导原则(试行)》. 2023.
27. FDA. CDER patient-focused drug development.
28. FDA. CDER & CBER drug development tool qualification project search.
29. EMA. EMA 25th anniversary symposium: new approaches in patient-focused cancer drug development.
30. 周孟, 张新宇, 张清, 等. 关于以患者为中心药物开发的见解—现有药物评价体系的重要补充. 中国食品药品监管, 2021, (4): 44-51.
31. 孙昱, 文海若, 汪祺. 以患者为中心的药物研发策略. 药物评价研究, 2020, 43(9): 1719-1727.
32. 尹立强, 赵杨升, 马伟. "以患者为中心"在医疗器械研发中的价值和应用. 中国医疗器械信息, 2021, 27(1): 36-38.
33. FDA. Patient-focused drug development glossary.
34. Benjamin K, Vernon MK, Patrick DL, et al. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA emerging good practices task force report. Value Health, 2017, 20(7): 838-855.
35. EMA. Draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies.
36. Basch E, Bennett AV. Patient-reported outcomes in clinical trials of rare diseases. J Gen Intern Med, 2014, 29(Suppl 3): S801-S803.
37. Timmermans S. The engaged patient: the relevance of patient-physician communication for twenty-first-century health. J Health Soc Behav, 2020, 61(3): 259-273.
38. Greenhalgh T. Narrative based medicine: narrative based medicine in an evidence based world. BMJ, 1999, 318(7179): 323-325.
39. 崔鸿雁, 吕兰青, 邵明义等. 以患者为中心进行中医药临床研究的思考与探讨. 中华中医药杂志, 2022, 37(12): 7224-7227.
40. FDA. Clinical outcome assessment (COA) compendium.

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