Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer

Sanjay Popat, Stephen V. Liu, Nicolas Scheuer, Grace G. Hsu, Alexandre Lockhart, Sreeram V. Ramagopalan (), Frank Griesinger and Vivek Subbiah ()
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Sanjay Popat: Royal Marsden Hospital and Institute of Cancer Research
Stephen V. Liu: Georgetown University
Nicolas Scheuer: Roche Products Ltd
Grace G. Hsu: Cytel, Inc
Alexandre Lockhart: Cytel, Inc
Sreeram V. Ramagopalan: Global Access, F. Hoffmann-La Roche
Frank Griesinger: Pius-Hospital Oldenburg
Vivek Subbiah: The University of Texas MD Anderson Cancer Center

Nature Communications, 2022, vol. 13, issue 1, 1-10

Abstract: Abstract As advanced non-small cell lung cancer (aNSCLC) is being increasingly divided into rare oncogene-driven subsets, conducting randomised trials becomes challenging. Using real-world data (RWD) to construct control arms for single-arm trials provides an option for comparative data. However, non-randomised treatment comparisons have the potential to be biased and cause concern for decision-makers. Using the example of pralsetinib from a RET fusion-positive aNSCLC single-arm trial (NCT03037385), we demonstrate a relative survival benefit when compared to pembrolizumab monotherapy and pembrolizumab with chemotherapy RWD cohorts. Quantitative bias analyses show that results for the RWD-trial comparisons are robust to data missingness, potential poorer outcomes in RWD and residual confounding. Overall, the study provides evidence in favour of pralsetinib as a first-line treatment for RET fusion-positive aNSCLC. The quantification of potential bias performed in this study can be used as a template for future studies of this nature.

Date: 2022
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DOI: 10.1038/s41467-022-30908-1

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