真实世界证据助推药械评价与监管决策
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摘要
近年来,真实世界数据与证据受到全球医疗卫生和监管决策部门的重视。尤其是药械监管决策的需求推动了真实世界证据的产生和使用。但在不同药械评价与监管决策环境下,对真实世界数据及证据的应用模式不同。本文探讨不同数据来源的真实世界证据在上市前药械评价、上市后药械监测与评价中的作用及其在监管决策中的价值,为正确生产和使用真实世界证据,推动药械监管决策提供参考。
In recent years, real-world evidence data(RWD) and real-world evidence(RWE) have gained substantial attentions from healthcare practitioners and health authorities worldwide. In particular, the needs from regulatory bodies have promoted the production and use of real-world evidence. In the context of drug and device evaluation and regulation decisions, the pattern for using real world evidence may differ. This article aimed to discuss the potential uses of RWE for pre-approval clinical evaluation, post-approval monitoring and evaluation, and associated regulatory decisions, which may ultimately improve the production and use of RWE for regulatory decisions.
In recent years, real-world evidence data(RWD) and real-world evidence(RWE) have gained substantial attentions from healthcare practitioners and health authorities worldwide. In particular, the needs from regulatory bodies have promoted the production and use of real-world evidence. In the context of drug and device evaluation and regulation decisions, the pattern for using real world evidence may differ. This article aimed to discuss the potential uses of RWE for pre-approval clinical evaluation, post-approval monitoring and evaluation, and associated regulatory decisions, which may ultimately improve the production and use of RWE for regulatory decisions.
引文
1U.S. Food & Drug Administration. FDA's Sentinel Initiative 2016. Available at: https://www.fda.gov/Safety/FDAsSentinelInitiative/ default.htm.
2U.S. Food & Drug Administration. Use of real-world evidence to support regulatory decision- making for medical devices. Available at: https://www.sogou.com/link? url=hedJjaC291MKYTE9c75 enrRl1lre67CaI2smYvubWPDFTrmuzC6_HxuOpabFoY9DDAtF2 Uxnf-_K7H6UfOzzMu_peCDdarnS0mCuc_NSvjlMbO2TiRDw9Lmp63ypS7ArUC4tv2Zulat5pUc3zy0dg.
3Oyinlola, J O, Campbell J, Kousoulis AA. Is real world evidence influencing practice? A systematic review of CPRD research in NICE guidances. BMC Health Serv Res, 2016, 16: 299.
4Suzuki H, Shimada K, Fujiwara K. Antihypertensive effectiveness of combination therapy with losartan/hydrochlorothiazide for 'real world' management of isolated systolic hypertension. Ther Adv Cardiovasc Dis, 2015, 9(1): 10-18.
5Barclay K, Carruthers R, Traboulsee A, et al. Best practices for long-term monitoring and follow-up of alemtuzumab-treated ms patients in real-world clinical settings. Front Neurol, 2019, 10: 253.
6Garrison LP, Jr. Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report. Value Health, 2007, 10(5): 326-35.
7Hiroi S, Sugano K, Tanaka S, et al. Impact of health insurance coverage for Helicobacter pylori gastritis on the trends in eradication therapy in Japan: retrospective observational study and simulation study based on real-world data. BMJ Open, 2017, 7(7): e015855.
8Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Comp Eff Res, 2018, 7(12): 1133-1143.
9Stang PE, Ryan PB, Racoosin JA, et al. Advancing the science for active surveillance: rationale and design for the observational medical outcomes partnership. Ann Intern Med, 2010, 153(9): 600606.
10U.S. Food & Drug Administration. Framework for FDA's real world evidence program. Available at: https://www.fda.gov/downloads/ ScienceResearch/SpecialTopics/RealWorldEvidence/UCM627769.p df.
11U.S. Food & Drug Administration. Best practice for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Available at: https://www.fda.gov/ downloads/Drugs/GuidanceComplianceRegulatoryInformation/G uidances/U CM243537.pdf.
12Sun X, Li Y, Guyatt G. Available at: https://blogs.bmj.com/ bmj/2018/11/06/production-of-trustworthy-real-world-data-andevidence-in-china/.
13贝伐珠单抗又有新适应症获批,真实世界数据为其提供新思路.Availableat:https://www.sohu.com/a/273433437_564028.
14Sun X, Tan J, Tang L, et al. Real world evidence: experience and lessons from China. BMJ, 2018, 360: j5262.
15国家药品监督管理局.关于政协十三届全国委员会第一次会议第2489号(医疗体育类247号)提案答复的函.Availableat:http://www.nmpa.gov.cn/WS04/CL2141/331878.html.
16Han, S, Jeong HS, Kim H, et al. The treatment pattern and adherence to direct oral anticoagulants in patients with atrial fibrillation aged over 65. PLoS One, 2019, 14(4): e0214666.
17U.S. Food & Drug Administration. Guidance for Industry and FDA Staff: Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. Available at: https://www.fda.gov/downloads/ MedicalDevices/DeviceRegulationandGuidance/GuidanceDocu ments/ucm071121.pdf.
18Find Drugs & Conditions. Ibrance approval expanded to include men with breast cancer. Available at: https://www.drugs.com/news/ ibrance-approval-expanded-include-men-breast-cancer81595.html.
19Resnic FS, Majithia A, Marinac-Dabic D, et al. Registry-based prospective, active surveillance of medical-device safety. N Engl J Med, 2017, 376(6): 526-535.
20中华人民共和国卫生部.药品不良反应报告和监测管理办法(卫生部令第81号).Availableat:http://www.sda.gov.cn/WS01/CL0053/62621.html.
21国家食品药品监督管理总局安监司.关于征求《关于推动生产企业开展药品重点监测工作的通知(征求意见稿)》意见的函.Availableat:http://www.cfda.gov.cn/WS01/CL0778/79321.html.
22孙鑫,谭婧,唐立,等.基于真实世界证据的上市后药品评价技术框架体系:思考与建议.中国循证医学杂志,2018,18(4):277-283.
23谭婧,程亮亮,王雯,等.患者登记研究的策划与患者登记数据库构建:基于观察性设计的真实世界研究.中国循证医学杂志,2017,17(12):1-8.
24U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenati de.
25Dore DD, Bloomgren GL, Wenten M, et al. A cohort study of acute pancreatitis in relation to exenatide use. Diabetes Obes Metab, 2011, 13(6): 559-66.
26孙鑫,谭婧,唐立,等.重新认识真实世界研究.中国循证医学杂志,2017,17(2):126-130.
27王雯,刘艳梅,谭婧,等.回顾性数据库研究的概念,策划与研究数据库构建.中国循证医学杂志,2018,18(2):1365-1372.
28唐立,康德英,喻佳洁,等.实效性随机对照试验:真实世界研究的重要设计.中国循证医学杂志,2017,2017(17):999-1004.
29李洪,魏来,郭晓蕙,等.真实世界研究伦理审查初探.中国循证医学杂志,2018,18(11):1198-1202.
30王雯,谭婧,于川,等.基于中国医院电子病历数据的药品安全性评价模式探索.中国药物警戒,2019,16(3):134-138.
2U.S. Food & Drug Administration. Use of real-world evidence to support regulatory decision- making for medical devices. Available at: https://www.sogou.com/link? url=hedJjaC291MKYTE9c75 enrRl1lre67CaI2smYvubWPDFTrmuzC6_HxuOpabFoY9DDAtF2 Uxnf-_K7H6UfOzzMu_peCDdarnS0mCuc_NSvjlMbO2TiRDw9Lmp63ypS7ArUC4tv2Zulat5pUc3zy0dg.
3Oyinlola, J O, Campbell J, Kousoulis AA. Is real world evidence influencing practice? A systematic review of CPRD research in NICE guidances. BMC Health Serv Res, 2016, 16: 299.
4Suzuki H, Shimada K, Fujiwara K. Antihypertensive effectiveness of combination therapy with losartan/hydrochlorothiazide for 'real world' management of isolated systolic hypertension. Ther Adv Cardiovasc Dis, 2015, 9(1): 10-18.
5Barclay K, Carruthers R, Traboulsee A, et al. Best practices for long-term monitoring and follow-up of alemtuzumab-treated ms patients in real-world clinical settings. Front Neurol, 2019, 10: 253.
6Garrison LP, Jr. Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report. Value Health, 2007, 10(5): 326-35.
7Hiroi S, Sugano K, Tanaka S, et al. Impact of health insurance coverage for Helicobacter pylori gastritis on the trends in eradication therapy in Japan: retrospective observational study and simulation study based on real-world data. BMJ Open, 2017, 7(7): e015855.
8Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Comp Eff Res, 2018, 7(12): 1133-1143.
9Stang PE, Ryan PB, Racoosin JA, et al. Advancing the science for active surveillance: rationale and design for the observational medical outcomes partnership. Ann Intern Med, 2010, 153(9): 600606.
10U.S. Food & Drug Administration. Framework for FDA's real world evidence program. Available at: https://www.fda.gov/downloads/ ScienceResearch/SpecialTopics/RealWorldEvidence/UCM627769.p df.
11U.S. Food & Drug Administration. Best practice for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Available at: https://www.fda.gov/ downloads/Drugs/GuidanceComplianceRegulatoryInformation/G uidances/U CM243537.pdf.
12Sun X, Li Y, Guyatt G. Available at: https://blogs.bmj.com/ bmj/2018/11/06/production-of-trustworthy-real-world-data-andevidence-in-china/.
13贝伐珠单抗又有新适应症获批,真实世界数据为其提供新思路.Availableat:https://www.sohu.com/a/273433437_564028.
14Sun X, Tan J, Tang L, et al. Real world evidence: experience and lessons from China. BMJ, 2018, 360: j5262.
15国家药品监督管理局.关于政协十三届全国委员会第一次会议第2489号(医疗体育类247号)提案答复的函.Availableat:http://www.nmpa.gov.cn/WS04/CL2141/331878.html.
16Han, S, Jeong HS, Kim H, et al. The treatment pattern and adherence to direct oral anticoagulants in patients with atrial fibrillation aged over 65. PLoS One, 2019, 14(4): e0214666.
17U.S. Food & Drug Administration. Guidance for Industry and FDA Staff: Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. Available at: https://www.fda.gov/downloads/ MedicalDevices/DeviceRegulationandGuidance/GuidanceDocu ments/ucm071121.pdf.
18Find Drugs & Conditions. Ibrance approval expanded to include men with breast cancer. Available at: https://www.drugs.com/news/ ibrance-approval-expanded-include-men-breast-cancer81595.html.
19Resnic FS, Majithia A, Marinac-Dabic D, et al. Registry-based prospective, active surveillance of medical-device safety. N Engl J Med, 2017, 376(6): 526-535.
20中华人民共和国卫生部.药品不良反应报告和监测管理办法(卫生部令第81号).Availableat:http://www.sda.gov.cn/WS01/CL0053/62621.html.
21国家食品药品监督管理总局安监司.关于征求《关于推动生产企业开展药品重点监测工作的通知(征求意见稿)》意见的函.Availableat:http://www.cfda.gov.cn/WS01/CL0778/79321.html.
22孙鑫,谭婧,唐立,等.基于真实世界证据的上市后药品评价技术框架体系:思考与建议.中国循证医学杂志,2018,18(4):277-283.
23谭婧,程亮亮,王雯,等.患者登记研究的策划与患者登记数据库构建:基于观察性设计的真实世界研究.中国循证医学杂志,2017,17(12):1-8.
24U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenati de.
25Dore DD, Bloomgren GL, Wenten M, et al. A cohort study of acute pancreatitis in relation to exenatide use. Diabetes Obes Metab, 2011, 13(6): 559-66.
26孙鑫,谭婧,唐立,等.重新认识真实世界研究.中国循证医学杂志,2017,17(2):126-130.
27王雯,刘艳梅,谭婧,等.回顾性数据库研究的概念,策划与研究数据库构建.中国循证医学杂志,2018,18(2):1365-1372.
28唐立,康德英,喻佳洁,等.实效性随机对照试验:真实世界研究的重要设计.中国循证医学杂志,2017,2017(17):999-1004.
29李洪,魏来,郭晓蕙,等.真实世界研究伦理审查初探.中国循证医学杂志,2018,18(11):1198-1202.
30王雯,谭婧,于川,等.基于中国医院电子病历数据的药品安全性评价模式探索.中国药物警戒,2019,16(3):134-138.