Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence

被引:153
|
作者
Bartlett, Victoria L. [3 ]
Dhruva, Sanket S. [4 ,5 ]
Shah, Nilay D. [6 ]
Ryan, Patrick [7 ,8 ]
Ross, Joseph S. [1 ,2 ,9 ,10 ]
机构
[1] Yale Sch Med, Sect Gen Internal Med, POB 208093, New Haven, CT 06520 USA
[2] Yale Sch Med, Natl Clinician Scholars Program, POB 208093, New Haven, CT 06520 USA
[3] Yale Sch Med, New Haven, CT 06520 USA
[4] Univ Calif San Francisco, San Francisco Sch Med, Dept Med, San Francisco, CA 94143 USA
[5] San Francisco Vet Affairs Hlth Care Syst, Sect Cardiol, San Francisco, CA USA
[6] Mayo Clin, Div Hlth Care Policy & Res, Rochester, MN USA
[7] Janssen Res & Dev, Epidemiol Analyt, Titusville, NJ USA
[8] Columbia Univ, Dept Biomed Informat, OHDSI, Med Ctr, New York, NY USA
[9] Yale Sch Publ Hlth, Dept Hlth Policy & Management, New Haven, CT 06520 USA
[10] Yale New Haven Med Ctr, 20 York St, New Haven, CT 06504 USA
关键词
MYOCARDIAL-INFARCTION; ACCURACY; EVENTS; CLAIMS; US;
D O I
10.1001/jamanetworkopen.2019.12869
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
IMPORTANCE Although randomized clinical trials are considered to be the criterion standard for generating clinical evidence, the use of real-world evidence to evaluate the efficacy and safety of medical interventions is gaining interest. Whether observational data can be used to address the same clinical questions being answered by traditional clinical trials is still unclear. OBJECTIVE To identify the number of clinical trials published in high-impact journals in 2017 that could be feasibly replicated using observational data from insurance claims and/or electronic health records (EHRs). DESIGN, SETTING, AND PARTICIPANTS In this cross-sectional analysis, PubMed was searched to identify all US-based clinical trials, regardless of randomization, published between January 1, 2017, and December 31, 2017, in the top 7 highest-impact general medical journals of 2017. Trials were excluded if they did not involve human participants, did not use end points that represented clinical outcomes among patients, were not characterized as clinical trials, and had no recruitment sites in the United States. MAIN OUTCOMES AND MEASURES The primary outcomes were the number and percentage of trials for which the intervention, indication, trial inclusion and exclusion criteria, and primary end points could be ascertained from insurance claims and/or EHR data. RESULTS Of the 220 US-based trials analyzed, 33 (15.0%) could be replicated using observational data because their intervention, indication, inclusion and exclusion criteria, and primary end points could be routinely ascertained from insurance claims and/or EHR data. Of the 220 trials, 86 (39.1%) had an intervention that could be ascertained from insurance claims and/or EHR data. Among the 86 trials, 62 (72.1%) had an indication that could be ascertained. Forty-five (72.6%) of 62 trials had at least 80% of inclusion and exclusion criteria data that could be ascertained. Of these 45 studies, 33 (73.3%) had at least 1 primary end point that could be ascertained. CONCLUSIONS AND RELEVANCE This study found that only 15% of the US-based clinical trials published in high-impact journals in 2017 could be feasibly replicated through analysis of administrative claims or EHR data. This finding suggests the potential for real-world evidence to complement clinical trials, both by examining the concordance between randomized experiments and observational studies and by comparing the generalizability of the trial population with the real-world population of interest.
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页数:11
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