Importance: Nonrandomized studies using insurance claims databases can be analyzed to produce real-world evidence on the effectiveness of medical products. Given the lack of baseline randomization and measurement issues, concerns exist about whether such studies produce unbiased treatment effect estimates.
Objective: To emulate the design of 30 completed and 2 ongoing randomized clinical trials (RCTs) of medications with database studies using observational analogues of the RCT design parameters (population, intervention, comparator, outcome, time [PICOT]) and to quantify agreement in RCT-database study pairs.
Design, Setting, And Participants: New-user cohort studies with propensity score matching using 3 US claims databases (Optum Clinformatics, MarketScan, and Medicare). Inclusion-exclusion criteria for each database study were prespecified to emulate the corresponding RCT. RCTs were explicitly selected based on feasibility, including power, key confounders, and end points more likely to be emulated with real-world data. All 32 protocols were registered on ClinicalTrials.gov before conducting analyses. Emulations were conducted from 2017 through 2022.
Exposures: Therapies for multiple clinical conditions were included.
Main Outcomes And Measures: Database study emulations focused on the primary outcome of the corresponding RCT. Findings of database studies were compared with RCTs using predefined metrics, including Pearson correlation coefficients and binary metrics based on statistical significance agreement, estimate agreement, and standardized difference.
Results: In these highly selected RCTs, the overall observed agreement between the RCT and the database emulation results was a Pearson correlation of 0.82 (95% CI, 0.64-0.91), with 75% meeting statistical significance, 66% estimate agreement, and 75% standardized difference agreement. In a post hoc analysis limited to 16 RCTs with closer emulation of trial design and measurements, concordance was higher (Pearson r, 0.93; 95% CI, 0.79-0.97; 94% meeting statistical significance, 88% estimate agreement, 88% standardized difference agreement). Weaker concordance occurred among 16 RCTs for which close emulation of certain design elements that define the research question (PICOT) with data from insurance claims was not possible (Pearson r, 0.53; 95% CI, 0.00-0.83; 56% meeting statistical significance, 50% estimate agreement, 69% standardized difference agreement).
Conclusions And Relevance: Real-world evidence studies can reach similar conclusions as RCTs when design and measurements can be closely emulated, but this may be difficult to achieve. Concordance in results varied depending on the agreement metric. Emulation differences, chance, and residual confounding can contribute to divergence in results and are difficult to disentangle.
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http://dx.doi.org/10.1001/jama.2023.4221 | DOI Listing |
Elife
December 2024
Institute of Cognitive and Clinical Neuroscience, Central Institute of Mental Health, Medical Faculty Mannheim, Heidelberg University, Mannheim, Germany.
Chronic back pain (CBP) is a global health concern with significant societal and economic burden. While various predictors of back pain chronicity have been proposed, including demographic and psychosocial factors, neuroimaging studies have pointed to brain characteristics as predictors of CBP. However, large-scale, multisite validation of these predictors is currently lacking.
View Article and Find Full Text PDFEur J Ophthalmol
December 2024
Department of Ophthalmology, Centro Hospitalar e Universitário de Coimbra (CHUC), Coimbra, Portugal.
Introduction: Keratoconus patients turn to the internet for answers to their disease expectations. Webpages are not filtered or submitted to evaluation before getting published. We aim to evaluate the quality and readability of the online information regarding keratoconus in Portugal and Brazil.
View Article and Find Full Text PDFMed Decis Making
December 2024
Department of Social & Behavioral Sciences, College of Public Health, Temple University, Philadelphia, PA, USA.
Background: In the United States, human papillomavirus (HPV) vaccination among 27- to 45-y-olds (mid-adults) is recommended based on shared clinical decision making with a health care provider. We developed a patient decision aid tool to support the implementation of this mid-adult HPV vaccination guideline. The purpose of this study was to evaluate the effect of a patient decision aid tool for HPV vaccination, HPV DECIDE, compared with an information fact sheet among mid-adults who have not received the HPV vaccine.
View Article and Find Full Text PDFPacing Clin Electrophysiol
December 2024
Department of Cardiology, University Hospitals of Leicester NHS Trust, Glenfield Hospital, Leicester, UK.
Background: Pulmonary vein isolation (PVI) has been established as an effective management option for symptomatic paroxysmal atrial fibrillation (PAF). We aimed to explore the role of P-wave parameters in a 12-lead electrocardiogram (ECG) in predicting the success of repeat PAF ablation.
Methods: We enrolled consecutive patients who underwent a second AF ablation procedure for PAF in a UK tertiary center after an index ablation conducted between 2018 and 2019 and a repeat ablation up to 2021.
Pacing Clin Electrophysiol
December 2024
Department of Cardiology, Lady Davis Carmel Medical Center, Haifa, Israel.
Background: Pacemaker recipients demonstrate a higher prevalence of atrial fibrillation (AF), yet the regular ventricular activation in pacemaker-dependent patients with AF presents a substantial diagnostic challenge.
Methods: A total of 310 medical practitioners completed a brief, validated survey consisting of three electrocardiograms displaying AF with ventricular pacing. Participants were instructed to identify the underlying rhythm.
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