Use of Claims to Assess Outcomes and Treatment Effects in the Evolut Low Risk Trial.

Background: Food and Drug Administration-mandated postmarket studies for transcatheter aortic valve replacement in low-risk populations plan to use passively collected registry data linked to claims for long-term follow-up out to 10 years. Therefore, it is critically important to understand the validity of these claims-based end points. We sought to evaluate the ability of administrative claims with () codes to identify trial-adjudicated end points and reproduce treatment comparisons of aortic valve replacement in the Evolut Low Risk Trial.

Methods: We linked Evolut Low Risk trial patients to the Medicare Provider Analysis and Review database. We calculated sensitivity, specificity, positive predictive value, negative predictive value, and agreement statistic of claims to detect clinical end points through 2 years in trial patients. We additionally compared end points across treatment arms using trial-adjudicated outcomes versus claims-based outcomes.

Results: Trial-adjudicated deaths were perfectly identified by claims. Claims had good performance in identifying trial-adjudicated disabling stroke (sensitivity 68.8%, specificity 99.0%, positive predictive value 64.7%, negative predictive value 99.1%, =0.66) and pacemaker placement (sensitivity 85.2%, specificity 98.4%, positive predictive value 90.4%, negative predictive value 97.5%, =0.86), but more modest performance in identifying trial-adjudicated myocardial infarction (=0.46) and vascular complications (=0.45). There was no difference between treatment arms for the primary end point of death or disabling stroke using trial data (hazard ratio, 0.83 [95% CI, 0.41-1.68]) or claims data (hazard ratio, 0.89 [95% CI, 0.43-1.81]; interaction =0.71).

Conclusions: Claims-based end points performed well in ascertaining death, disabling stroke, and pacemaker placement and were able to reproduce principal trial findings. These results support the selective use of claims-based end points for transcatheter aortic valve replacement postmarketing surveillance.

Registration: URL: https://www.clinicaltrials.gov; Unique identifier: NCT02701283.