Emulation Differences vs. Biases When Calibrating Real-World Evidence Findings Against Randomized Controlled Trials

被引:39
作者
Franklin, Jessica M. [1 ]
Glynn, Robert J. [1 ]
Suissa, Samy [2 ,3 ,4 ]
Schneeweiss, Sebastian [1 ]
机构
[1] Harvard Med Sch, Brigham & Womens Hosp, Dept Med, Div Pharmacoepidemiol & Pharmacoecon, Boston, MA 02115 USA
[2] McGill Univ, Dept Epidemiol, Montreal, PQ, Canada
[3] McGill Univ, Dept Biostat, Montreal, PQ, Canada
[4] McGill Univ, Dept Occupat Hlth, Montreal, PQ, Canada
关键词
D O I
10.1002/cpt.1793
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Actionable real-world evidence (RWE) requires accurate estimation of causal treatment effects. Calibration of RWE against randomized controlled trials (RCTs) is sometimes done to demonstrate that RWE can support the same causal conclusion as RCTs. Disagreements can occur when studies in each pair asked different questions in different populations or due to the presence of residual bias. Distinguishing among reasons for differences will impact the level of confidence in RWE.
引用
收藏
页码:735 / 737
页数:3
相关论文
共 10 条
[1]   Using Electronic Health Records to Derive Control Arms for Early Phase Single-Arm Lung Cancer Trials: Proof-of-Concept in Randomized Controlled Trials [J].
Carrigan, Gillis ;
Whipple, Samuel ;
Capra, William B. ;
Taylor, Michael D. ;
Brown, Jeffrey S. ;
Lu, Michael ;
Arnieri, Brandon ;
Copping, Ryan ;
Rothman, Kenneth J. .
CLINICAL PHARMACOLOGY & THERAPEUTICS, 2020, 107 (02) :369-377
[2]   Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation [J].
Eichler, Hans-Georg ;
Koenig, Franz ;
Arlett, Peter ;
Enzmann, Harald ;
Humphreys, Anthony ;
Petavy, Frank ;
Schwarzer-Daum, Brigitte ;
Sepodes, Bruno ;
Vamvakas, Spiros ;
Rasi, Guido .
CLINICAL PHARMACOLOGY & THERAPEUTICS, 2020, 107 (04) :773-779
[3]   Nonrandomized Real-World Evidence to Support Regulatory Decision Making: Process for a Randomized Trial Replication Project [J].
Franklin, Jessica M. ;
Pawar, Ajinkya ;
Martin, David ;
Glynn, Robert J. ;
Levenson, Mark ;
Temple, Robert ;
Schneeweiss, Sebastian .
CLINICAL PHARMACOLOGY & THERAPEUTICS, 2020, 107 (04) :817-826
[4]   Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available [J].
Hernan, Miguel A. ;
Robins, James M. .
AMERICAN JOURNAL OF EPIDEMIOLOGY, 2016, 183 (08) :758-764
[5]   Using Real-World Data to Predict Findings of an Ongoing Phase IV Cardiovascular Outcome Trial: Cardiovascular Safety of Linagliptin Versus Glimepiride [J].
Patorno, Elisabetta ;
Schneeweiss, Sebastian ;
Gopalakrishnan, Chandrasekar ;
Martin, David ;
Franklin, Jessica M. .
DIABETES CARE, 2019, 42 (12) :2204-2210
[6]   Claims-based studies of oral glucose-lowering medications can achieve balance in critical clinical variables only observed in electronic health records [J].
Patorno, Elisabetta ;
Gopalakrishnan, Chandrasekar ;
Franklin, Jessica M. ;
Brodovicz, Kimberly G. ;
Masso-Gonzalez, Elvira ;
Bartels, Dorothee B. ;
Liu, Jun ;
Schneeweiss, Sebastian .
DIABETES OBESITY & METABOLISM, 2018, 20 (04) :974-984
[7]   Effect of Linagliptin vs Glimepiride on Major Adverse Cardiovascular Outcomes in Patients With Type 2 Diabetes: The CAROLINA Randomized Clinical Trial [J].
Rosenstock, Julio ;
Kahn, Steven E. ;
Johansen, Odd Erik ;
Zinman, Bernard ;
Espeland, Mark A. ;
Woerle, Hans J. ;
Pfarr, Egon ;
Keller, Annett ;
Mattheus, Michaela ;
Baanstra, David ;
Meinicke, Thomas ;
George, Jyothis T. ;
von Eynatten, Maximilian ;
McGuire, Darren K. ;
Marx, Nikolaus .
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2019, 322 (12) :1155-1166
[8]   ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions [J].
Sterne, Jonathan A. C. ;
Hernan, Miguel A. ;
Reeves, Barnaby C. ;
Savovic, Jelena ;
Berkman, Nancy D. ;
Viswanathan, Meera ;
Henry, David ;
Altman, Douglas G. ;
Ansari, Mohammed T. ;
Boutron, Isabelle ;
Carpenter, James R. ;
Chan, An-Wen ;
Churchill, Rachel ;
Deeks, Jonathan J. ;
Hrobjartsson, Asbjorn ;
Kirkham, Jamie ;
Juni, Peter ;
Loke, Yoon K. ;
Pigott, Theresa D. ;
Ramsay, Craig R. ;
Regidor, Deborah ;
Rothstein, Hannah R. ;
Sandhu, Lakhbir ;
Santaguida, Pasqualina L. ;
Schunemann, Holger J. ;
Shea, Beverly ;
Shrier, Ian ;
Tugwell, Peter ;
Turner, Lucy ;
Valentine, Jeffrey C. ;
Waddington, Hugh ;
Waters, Elizabeth ;
Wells, George A. ;
Whiting, Penny F. ;
Higgins, Julian P. T. .
BMJ-BRITISH MEDICAL JOURNAL, 2016, 355
[9]  
US Food and Drug Administration (FDA), 2018, FRAM FDAS REAL WORLD
[10]   Participation in a Clinical Trial Enhances Adherence and Persistence to Treatment A Retrospective Cohort Study [J].
van Onzenoort, Hein A. W. ;
Menger, Frederique E. ;
Neef, Cees ;
Verberk, Willem J. ;
Kroon, Abraham A. ;
de Leeuw, Peter W. ;
van der Kuy, Paul-Hugo M. .
HYPERTENSION, 2011, 58 (04) :573-578