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

rw cohorts included 862 patients, and clinical trial cohorts (2 control arms and 3 experimental arms) included 701 patients, see response rates in the table (all except ALEX were confirmed RRs).Table:

1581P

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.

Legal entity responsible for the study

Flatiron Health, Inc.

Funding

Flatiron Health Inc., independent subsidiary of the Roche group.

Disclosure

X. Ma: Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group.: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. N.C. Nussbaum: Shareholder / Stockholder / Stock options, Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group.: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. K. Magee: Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. A.B. Bourla: Shareholder / Stockholder / Stock options, Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. M. Tucker: Shareholder / Stockholder / Stock options, Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group.: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. L. Bellomo: Shareholder / Stockholder / Stock options, Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche. C. Bennette: Shareholder / Stockholder / Stock options, Full / Part-time employment, Flatiron Health, Inc. is an independent subsidiary of the Roche Group.: Flatiron Health, Inc.; Shareholder / Stockholder / Stock options: Roche.