Characterization of a Real-World Response Variable and Comparison with RECIST-Based Response Rates from Clinical Trials in Advanced NSCLC
| Autor: | Joshua S. Kraut, Ariel Bulua Bourla, Xinran Ma, Nathan C. Nussbaum, Kelly Magee, Bryan Bowser, Lawrence Bellomo, Meghna Samant, Olga Tymejczyk, Caroline S. Bennette, Melisa Tucker |
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| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
Lung Neoplasms Lower risk Correlation RWD RWE Internal medicine Carcinoma Non-Small-Cell Lung Overall survival Medicine Humans Pharmacology (medical) Clinical significance Disease burden Response Evaluation Criteria in Solid Tumors Retrospective Studies Original Research Real-world evidence business.industry Reproducibility of Results Response Retrospective cohort study General Medicine Real-world data Clinical trial RECIST Non small cell business |
| Zdroj: | Advances in Therapy |
| ISSN: | 1865-8652 |
| Popis: | Introduction Effectiveness metrics for real-word research, analogous to clinical trial ones, are needed. This study aimed to develop a real-world response (rwR) variable applicable to solid tumors and to evaluate its clinical relevance and meaningfulness. Methods This retrospective study used patient cohorts with advanced non-small cell lung cancer from a nationwide, de-identified electronic health record (EHR)-derived database. Disease burden information abstracted manually was classified into response categories anchored to discrete therapy lines (per patient-line). In part 1, we quantified the feasibility and reliability of data capture, and estimated the association between rwR status and real-world progression-free survival (rwPFS) and real-world overall survival (rwOS). In part 2, we investigated the correlation between published clinical trial overall response rates (ORRs) and real-world response rates (rwRRs) from corresponding real-world patient cohorts. Results In part 1, 85.4% of patients (N = 3248) had at least one radiographic assessment documented. Median abstraction time per patient-line was 15.0 min (IQR 7.8–28.1). Inter-abstractor agreement on presence/absence of at least one assessment was 0.94 (95% CI 0.92–0.96; n = 503 patient-lines abstracted in duplicate); inter-abstractor agreement on best confirmed response category was 0.82 (95% CI 0.78–0.86; n = 384 with at least one captured assessment). Confirmed responders at a 3-month landmark showed significantly lower risk of death and progression in rwOS and rwPFS analyses across all line settings. In part 2, rwRRs (from 12 rw cohorts) showed a high correlation with trial ORRs (Spearman’s ρ = 0.99). Conclusions We developed a rwR variable generated from clinician assessments documented in EHRs following radiographic evaluations. This variable provides clinically meaningful information and may provide a real-world measure of treatment effectiveness. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01659-0. |
| Databáze: | OpenAIRE |
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