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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25.	國家藥品監督管理局藥品審評中心. 《以患者為中心的藥物臨床試驗實施技術指導原則(試行)》. 2023.
26.	國家藥品監督管理局藥品審評中心. 《以患者為中心的藥物獲益-風險評估技術指導原則(試行)》. 2023.
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31.	孫昱, 文海若, 汪祺. 以患者為中心的藥物研發策略. 藥物評價研究, 2020, 43(9): 1719-1727.
32.	尹立強, 趙楊升, 馬偉. "以患者為中心"在醫療器械研發中的價值和應用. 中國醫療器械信息, 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.
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.	崔鴻雁, 呂蘭青, 邵明義等. 以患者為中心進行中醫藥臨床研究的思考與探討. 中華中醫藥雜志, 2022, 37(12): 7224-7227.
40.	FDA. Clinical outcome assessment (COA) compendium.

1. 張俊華, 鄭文科, 張伯禮, 等. 真實世界研究. 世界中醫藥, 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. 謝靜, 王煥玲. 以患者為中心的臨床試驗: 醫藥研發新模式. 協和醫學雜志, 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. 鄭航. 臨床試驗行業的基本特征與發展趨勢. 2018年中國藥學會藥物臨床評價研究專業委員會學術年會暨臨床研究高峰論壇論文集, 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. 美國食品藥品監督管理局以患者為中心的藥物開發系列指導原則1-《綜合和有代表性的信息收集指導原則》介紹. 中國臨床藥理學雜志, 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. 國家藥品監督管理局藥品審評中心. 《以患者為中心的臨床試驗設計技術指導原則(征求意見稿)》. 2022.
21. 國家藥品監督管理局藥品審評中心. 《以患者為中心的臨床試驗獲益-風險評估技術指導原則(征求意見稿)》. 2022.
22. 國家藥品監督管理局藥品審評中心. 《以患者為中心的臨床試驗實施技術指導原則(征求意見稿)》. 2022.
23. 國家藥品監督管理局藥品審評中心. 《組織患者參與藥物研發的一般考慮指導原則(試行)》. 2022.
24. 國家藥品監督管理局藥品審評中心. 《以患者為中心的藥物臨床試驗設計技術指導原則(試行)》. 2023.
25. 國家藥品監督管理局藥品審評中心. 《以患者為中心的藥物臨床試驗實施技術指導原則(試行)》. 2023.
26. 國家藥品監督管理局藥品審評中心. 《以患者為中心的藥物獲益-風險評估技術指導原則(試行)》. 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, 張新宇, 張清, 等. 關于以患者為中心藥物開發的見解—現有藥物評價體系的重要補充. 中國食品藥品監管, 2021, (4): 44-51.
31. 孫昱, 文海若, 汪祺. 以患者為中心的藥物研發策略. 藥物評價研究, 2020, 43(9): 1719-1727.
32. 尹立強, 趙楊升, 馬偉. "以患者為中心"在醫療器械研發中的價值和應用. 中國醫療器械信息, 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. 崔鴻雁, 呂蘭青, 邵明義等. 以患者為中心進行中醫藥臨床研究的思考與探討. 中華中醫藥雜志, 2022, 37(12): 7224-7227.
40. FDA. Clinical outcome assessment (COA) compendium.

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