Volume 14 Issue 4
Jul.  2023
Turn off MathJax
Article Contents
SHI Xiuqing, YAN Siyu, HUANG Qiao, LI Xuhui, WANG Yongbo, MA Wenhao, SHANG Hongcai, JIN Yinghui. Real World Research: Bridging the Gap Between Clinical Practice Guidelines and Clinical Decision Making[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(4): 859-867. doi: 10.12290/xhyxzz.2022-0217
Citation: SHI Xiuqing, YAN Siyu, HUANG Qiao, LI Xuhui, WANG Yongbo, MA Wenhao, SHANG Hongcai, JIN Yinghui. Real World Research: Bridging the Gap Between Clinical Practice Guidelines and Clinical Decision Making[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(4): 859-867. doi: 10.12290/xhyxzz.2022-0217

Real World Research: Bridging the Gap Between Clinical Practice Guidelines and Clinical Decision Making

doi: 10.12290/xhyxzz.2022-0217

National Natural Science Foundation of China 82174230

More Information
  • Corresponding author: SHANG Hongcai, E-mail: shanghongcai@126.com; JIN Yinghui, E-mail: jinyinghuiebm@163.com
  • Received Date: 2022-04-20
  • Accepted Date: 2022-06-13
  • Available Online: 2022-10-10
  • Publish Date: 2023-07-30
  • Clinical practice guidelines are an important basis for clinical decision-making, but there are still many factors that prevent physicians from following the guidelines. One of the problems, the limited external validity of the guidelines, ultimately prevents the guidelines from providing an adequate basis for clinical decision-making, thus forming the gap between the guidelines and clinical decisions. Real-world research can enhance the external validity of the guidelines, provide a basis for clinical decision-making, and offer localized evidence for the guidelines, evidence of cost-effectiveness and other evidence for the guidelines. It can also monitor the implementation of the guidelines and help to make up for the lack of evidence in the formulation of traditional guidelines. Therefore, high-quality real-world studies and real-world evidence may be considered in the guideline development process to facilitate guideline optimization and serve as an aid to guideline quality improvement.
  • loading
  • [1] Institute of Medicine. Clinical Practice Guidelines We Can Trust[M]. Washington DC: National Academies Press, 2011.
    [2] U.S. Food and Drug Administration. Framework for FDA's Real-World Evidence Program[EB/OL]. (2018-12)[2022-04-15]. https://www.fda.gov/media/120060/download.
    [3] Concato J, Corrigan-Curay J. Real-World Evidence-Where Are We Now?[J]. N Engl J Med, 2022, 386: 1680-1682. doi:  10.1056/NEJMp2200089
    [4] U.S. Food and Drug Administration. Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices-Guidance for Industry and Food and Drug Administration Staff[EB/OL]. (2017-08)[2022-04-15]. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-real-world-evidence-support-regulatory-decision-making-medical-devices.
    [5] 国家药品监督管理局. 真实世界数据用于医疗器械临床评价技术指导原则(征求意见稿)[EB/OL]. (2019-12-13)[2022-04-15]. https://www.cmde.org.cn//zhuanti/zqyj/20191213150200919.html.
    [6] 国家药品监督管理局. 真实世界证据支持药物研发与审评的指导原则(试行)[EB/OL]. (2020-01-07)[2022-04-15]. https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200107151901190.html.
    [7] U.S. Food and Drug Administration. 21st Century Cure Act[EB/OL]. (2016-12-13)[2022-04-15]. https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act.
    [8] U.S. Food and Drug Administration. Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics Guidance for Industry[EB/OL]. (2019-05-09)[2022-04-15]. https://www.fda.gov/news-events/fda-brief/fda-brief-fda-issues-draft-guidance-industry-submitting-real-world-evidence-new-drug-and-biologic.
    [9] 国家药品监督管理局. 国家药监局药审中心关于发布《真实世界研究支持儿童药物研发与审评的技术指导原则(试行)》的通告(2020年第22号)[EB/OL]. (2020-09-01)[2022-04-15]. https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200901104448101.html.
    [10] Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-World Evidence-What Is It and What Can It Tell Us?[J]. N Engl J Med, 2016, 375: 2293-2297. doi:  10.1056/NEJMsb1609216
    [11] New JP, Bakerly ND, Leather D, et al. Obtaining real-world evidence: the Salford Lung Study[J]. Thorax, 2014, 69: 1152-1154. doi:  10.1136/thoraxjnl-2014-205259
    [12] Alonso-Coello P, Irfan A, Solà I, et al. The quality of clinical practice guidelines over the last two decades: a systematic review of guideline appraisal studies[J]. Qual Saf Health Care, 2010, 19: e58.
    [13] Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer (Ta, T1, and Carcinoma in Situ)[J]. Eur Urol, 2022, 81: 75-94. doi:  10.1016/j.eururo.2021.08.010
    [14] Chang SS, Boorjian SA, Chou R, et al. Diagnosis and Treatment of Non-Muscle Invasive Bladder Cancer: AUA/SUO Guideline[J]. J Urol, 2016, 196: 1021-1029. doi:  10.1016/j.juro.2016.06.049
    [15] Mori K, Miura N, Babjuk M, et al. Low compliance to guidelines in nonmuscle-invasive bladder carcinoma: A systematic review[J]. Urol Oncol, 2020, 38: 774-782. doi:  10.1016/j.urolonc.2020.06.013
    [16] Chamie K, Saigal CS, Lai J, et al. Compliance with guidelines for patients with bladder cancer: variation in the delivery of care[J]. Cancer, 2011, 117: 5392-5401. doi:  10.1002/cncr.26198
    [17] Posen A, Benken S, Kaluzna SD, et al. Poor guideline adherence in a real-world evaluation of hypertensive emergency management[J]. Am J Emerg Med, 2022, 51: 46-52. doi:  10.1016/j.ajem.2021.09.073
    [18] Cabana MD, Rand CS, Powe NR, et al. Why don't physicians follow clinical practice guidelines? A framework for improvement[J]. JAMA, 1999, 282: 1458-1465. doi:  10.1001/jama.282.15.1458
    [19] Jin YH, Tan LM, Khan KS, et al. Determinants of successful guideline implementation: a national cross-sectional survey[J]. BMC Med Inform Decis Mak, 2021, 21: 19. doi:  10.1186/s12911-020-01382-w
    [20] Wong GW, Miravitlles M, Chisholm A, et al. Respiratory guidelines——which real world?[J]. Ann Am Thorac Soc, 2014, 11: S85-S91. doi:  10.1513/AnnalsATS.201309-298RM
    [21] Guyatt GH, Oxman AD, Vist GE, et al. GRADE: an emerging consensus on rating quality of evidence and strength of recommendations[J]. BMJ, 2008, 336: 924-926. doi:  10.1136/bmj.39489.470347.AD
    [22] Herland K, Akselsen JP, Skjønsberg OH, et al. How representative are clinical study patients with asthma or COPD for a larger "real life" population of patients with obstructive lung disease?[J]. Respir Med, 2005, 99: 11-19. doi:  10.1016/j.rmed.2004.03.026
    [23] Hoffman SN, TenBrook JA, Wolf MP, et al. A meta-analysis of randomized controlled trials comparing coronary artery bypass graft with percutaneous transluminal coronary angioplasty: one-to eight-year outcomes[J]. J Am Coll Cardiol, 2003, 41: 1293-1304. doi:  10.1016/S0735-1097(03)00157-8
    [24] Brett W, Hirschmann MT, Guller U, et al. CABG vs. PCI in coronary artery disease: what is the evidence?[J]. Card Surg Today, 2006, 2: 43-55.
    [25] Grapow MT, von Wattenwyl R, Guller U, et al. Rando-mized controlled trials do not reflect reality: real-world analyses are critical for treatment guidelines![J]. J Thorac Cardiovasc Surg, 2006, 132: 5-7. doi:  10.1016/j.jtcvs.2006.03.035
    [26] Kennedy-Martin T, Curtis S, Faries D, et al. A literature review on the representativeness of randomized controlled trial samples and implications for the external validity of trial results[J]. Trials, 2015, 16: 495. doi:  10.1186/s13063-015-1023-4
    [27] Chew SY, Koh MS, Loo CM, et al. Making Clinical Practice Guidelines Pragmatic: How Big Data and Real World Evidence Can Close the Gap[J]. Ann Acad Med Singap, 2018, 47: 523-527. doi:  10.47102/annals-acadmedsg.V47N12p523
    [28] Hayward RS. Clinical practice guidelines on trial[J]. CMAJ, 1997, 156: 1725-1727.
    [29] 周奇, 董冲亚, 王业明, 等. 循证医学理念在指导临床研究与实践中的作用: 基于对抗病毒药物治疗新型冠状病毒肺炎的思考[J]. 中国循证医学杂志, 2022, 22: 373-379. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ202204001.htm
    [30] Schneeweiss S, Patorno E. Conducting Real-world Evidence Studies on the Clinical Outcomes of Diabetes Treatments[J]. Endocr Rev, 2021, 42: 658-690. doi:  10.1210/endrev/bnab007
    [31] Wedam S, Fashoyin-Aje L, Bloomquist E, et al. FDA Approval Summary: Palbociclib for Male Patients with Metastatic Breast Cancer[J]. Clin Cancer Res, 2020, 26: 1208-1212. doi:  10.1158/1078-0432.CCR-19-2580
    [32] Khan O, Williams MJ, Amezcua L, et al. Multiple sclerosis in US minority populations: Clinical practice insights[J]. Neurol Clin Pract, 2015, 5: 132-142. doi:  10.1212/CPJ.0000000000000112
    [33] Williams MJ, Amezcua L, Okai A, et al. Real-World Safety and Effectiveness of Dimethyl Fumarate in Black or African American Patients with Multiple Sclerosis: 3-Year Results from ESTEEM[J]. Neurol Ther, 2020, 9: 483-493. doi:  10.1007/s40120-020-00193-5
    [34] Kleeberg UR, Linde H, Günther G, et al. Bendamustin-Rituximab Combination Is a Safe and Effective, Ambulatory Treatment for Elderly Patients with Chronic Lymphocytic Leukemia: Retrospective Real-world Analysis by Age from a German Registry and Review of the Literature[J]. Anticancer Res, 2016, 36: 2827-2838.
    [35] Beasley R, Holliday M, Reddel HK, et al. Controlled Trial of Budesonide-Formoterol as Needed for Mild Asthma[J]. N Engl J Med, 2019, 380: 2020-2030. doi:  10.1056/NEJMoa1901963
    [36] Albertson TE, Murin S, Sutter ME, et al. The Salford Lung Study: a pioneering comparative effectiveness approach to COPD and asthma in clinical trials[J]. Pragmat Obs Res, 2017, 8: 175-181.
    [37] Magnussen H, Disse B, Rodriguez-Roisin R, et al. Withdrawal of inhaled glucocorticoids and exacerbations of COPD[J]. N Engl J Med, 2014, 371: 1285-1294. doi:  10.1056/NEJMoa1407154
    [38] Chen Y, Wang C, Shang H, et al. Clinical practice guidelines in China[J]. BMJ, 2018, 360: j5158.
    [39] Kearon C, Akl EA, Comerota AJ, et al. Antithrombotic therapy for VTE disease: Antithrombotic Therapy and Prevention of Thrombosis, 9th ed: American College of Chest Physicians Evidence-Based Clinical Practice Guidelines[J]. Chest, 2012, 141: e419S-e496S. doi:  10.1378/chest.11-2301
    [40] Konstantinides SV, Torbicki A, Agnelli G, et al. 2014 ESC guidelines on the diagnosis and management of acute pulmonary embolism[J]. Eur Heart J, 2014, 35: 3033-3069. doi:  10.1093/eurheartj/ehu283
    [41] Wang C, Zhai Z, Yang Y, et al. Efficacy and safety of low dose recombinant tissue-type plasminogen activator for the treatment of acute pulmonary thromboembolism: a randomized, multicenter, controlled trial[J]. Chest, 2010, 137: 254-262. doi:  10.1378/chest.09-0765
    [42] Franklin JM, Glynn RJ, Martin D, et al. Evaluating the Use of Nonrandomized Real-World Data Analyses for Regulatory Decision Making[J]. Clin Pharmacol Ther, 2019, 105: 867-877. doi:  10.1002/cpt.1351
    [43] 彭晓霞, 舒啸尘, 谭婧, 等. 基于真实世界数据评价治疗结局的观察性研究设计技术规范[J]. 中国循证医学杂志, 2019, 19: 779-786. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201907004.htm
    [44] Cortese S, Panei P, Arcieri R, et al. Safety of Methylphenidate and Atomoxetine in Children with Attention-Deficit/Hyperactivity Disorder (ADHD): Data from the Italian National ADHD Registry[J]. CNS Drugs, 2015, 29: 865-877. doi:  10.1007/s40263-015-0266-7
    [45] 谭婧, 刘兴会, 孙鑫. 基于真实世界数据的疾病管理研究[J]. 协和医学杂志, 2019, 10: 284-288. doi:  10.3969/j.issn.1674-9081.2019.03.017
    [46] Camm AJ, Lip GY, De Caterina R, et al. 2012 focused update of the ESC Guidelines for the management of atrial fibrillation: an update of the 2010 ESC Guidelines for the management of atrial fibrillation. Developed with the special contribution of the European Heart Rhythm Association[J]. Eur Heart J, 2012, 33: 2719-2747. doi:  10.1093/eurheartj/ehs253
    [47] Andrade AA, Li J, Radford MJ, et al. Clinical Benefit of American College of Chest Physicians versus European Society of Cardiology Guidelines for Stroke Prophylaxis in Atrial Fibrillation[J]. J Gen Intern Med, 2015, 30: 777-782. doi:  10.1007/s11606-015-3201-1
    [48] Quanstrum KH, Hayward RA. Lessons from the mammography wars[J]. N Engl J Med, 2010, 363: 1076-1079. doi:  10.1056/NEJMsb1002538
    [49] Shaughnessy AF, Cosgrove L, Lexchin JR. The Need to Systematically Evaluate Clinical Practice Guidelines[J]. J Am Board Fam Med, 2016, 29: 644-648. doi:  10.3122/jabfm.2016.06.160115
    [50] Steels S, van der Zande M, van Staa TP. The role of real-world data in the development of treatment guidelines: a case study on guideline developers' opinions about using observational data on antibiotic prescribing in primary care[J]. BMC Health Serv Res, 2019, 19: 942. doi:  10.1186/s12913-019-4787-5
    [51] National Institue for Health and Care Excellence. NICE strategy 2021 to 2026-Dynamic, Collaborative, Excellent[EB/OL]. (2021-04)[2022-04-15]. https://www.nice.org.uk/about/who-we-are/corporate-publications/the-nice-strategy-2021-to-2026.
    [52] Collins R, Bowman L, Landray M, et al. The Magic of Randomization versus the Myth of Real-World Evidence[J]. N Engl J Med, 2020, 382: 674-678. doi:  10.1056/NEJMsb1901642
    [53] Levy RI. Currently available lipid-lowering agents[J]. Hosp Pract (Off Ed), 1988, 23: 14-21. doi:  10.1080/21548331.1988.11703634
    [54] Collins R, Reith C, Emberson J, et al. Interpretation of the evidence for the efficacy and safety of statin therapy[J]. Lancet, 2016, 388: 2532-2561. doi:  10.1016/S0140-6736(16)31357-5
    [55] Rimm EB, Stampfer MJ, Ascherio A, et al. Vitamin E consumption and the risk of coronary heart disease in men[J]. N Engl J Med, 1993, 328: 1450-1456. doi:  10.1056/NEJM199305203282004
    [56] Stampfer MJ, Hennekens CH, Manson JE, et al. Vitamin E consumption and the risk of coronary disease in women[J]. N Engl J Med, 1993, 328: 1444-1449. doi:  10.1056/NEJM199305203282003
    [57] Heart Outcomes Prevention Evaluation Study Investigators, Yusuf S, Dagenais G, et al. Vitamin E supplementation and cardiovascular events in high-risk patients[J]. N Engl J Med, 2000, 342: 154-160. doi:  10.1056/NEJM200001203420302
    [58] 刘晓清, 孙晓川. 真实世界证据[J]. 协和医学杂志, 2017, 8: 305-310. doi:  10.3969/j.issn.1674-9081.2017.05.021
    [59] Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy[J]. JAMA, 2003, 290: 1624-1632.
    [60] 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[J]. 中国循证医学杂志, 2018, 18: 277-283. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201804003.htm
    [61] 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[J]. 中国循证医学杂志, 2017, 17: 1365-1372. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201712001.htm
    [62] 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[J]. 中国循证医学杂志, 2017, 17: 999-1004. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201709002.htm
    [63] Booth CM, Karim S, Mackillop WJ. Real-world data: towards achieving the achievable in cancer care[J]. Nat Rev Clin Oncol, 2019, 16: 312-325.
    [64] 徐浩, 史大卓, 刘保延, 等. 以临床实践数据为导向构建中西医结合临床指南的设想[J]. 中国中西医结合杂志, 2009, 29: 544-547. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXJ200906023.htm
    [65] 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[J]. 中国循证医学杂志, 2018, 18: 230-237. https://www.cnki.com.cn/Article/CJFDTOTAL-ZZXZ201802016.htm
  • 加载中


    通讯作者: 陈斌, bchen63@163.com
    • 1. 

      沈阳化工大学材料科学与工程学院 沈阳 110142

    1. 本站搜索
    2. 百度学术搜索
    3. 万方数据库搜索
    4. CNKI搜索

    Figures(1)  / Tables(1)

    Article Metrics

    Article views (449) PDF downloads(100) Cited by()
    Proportional views


    DownLoad:  Full-Size Img  PowerPoint