Background
Real-world (rw) evidence derived from electronic health records (EHRs) is being explored for use as a comparator to single arm clinical trials in oncology. While RECIST is a standard for assessing tumor response in trials, alternative approaches are needed in rw cohorts. Our objective was to examine whether rwRR derived from abstracted “real-world tumor response” (rwTR) might perform similarly to RECIST-based response rates in a variety of settings to support assessments of drug efficacy.
Methods
We focused on randomized clinical trials supporting FDA approvals in aNSCLC between 2015-2017 (4 trials, 5 arms). We selected rw cohorts of aNSCLC patients from de-identified Flatiron Health EHR-derived database by applying the eligibility criteria of each trial as feasible, following an established internal process; trials were considered infeasible for comparison and not reported when fewer than 30 eligible rw patients were identified at the time of analyses. rwTR was abstracted from unstructured clinical notes and was based on the treating oncologist’s assessment of disease change (e.g., partial response) indicated by imaging at each point of radiographic disease evaluation. We compared the rwRRs observed in rw cohorts to objective response rates (ORRs) from trials.
Results
Table1581P
| Trial | Trial arm | ORR | rwRR |
|---|---|---|---|
| CheckMate-017 | E: Nivolumab / 2L+ squamous | 20% (27/135) | 25% (17/68) |
| CheckMate-057 | E: Nivolumab / 2L+ nonsquamous | 19% (56/292) | 17% (51/294) |
| KEYNOTE-021 | E: Carboplatin, Pembrolizumab, Pemetrexed/ 1L nonsquamous | 55% (33/60) | 42% (37/88) |
| KEYNOTE-021 | C: Carboplatin and Pemetrexed/ 1L nonsquamous | 29% (18/63) | 34% (101/294) |
| ALEX | C: Crizotinib/ 1L | 75% (114/151) | 68% (80/118) |
Conclusions
Data curated from EHRs of rw patients with aNSCLC can provide estimates of response rates that are comparable to RECIST-based response rates observed in similar patient populations studied in clinical trial settings. These findings demonstrate the potential of leveraging rw cohorts as a comparator to single arm clinical trials in oncology.
Authors:
Ma, X, Nussbaum, NC, Magee, K, Bourla, AB, Tucker, MG, Bellomo, L, Bennette, CS
Sources:
ESMO Annual Congress