Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer.
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Embargo End Date
ICR Authors
Authors
Popat, S
Liu, SV
Scheuer, N
Hsu, GG
Lockhart, A
Ramagopalan, SV
Griesinger, F
Subbiah, V
Liu, SV
Scheuer, N
Hsu, GG
Lockhart, A
Ramagopalan, SV
Griesinger, F
Subbiah, V
Document Type
Journal Article
Date
2022-06-17
Date Accepted
2022-05-25
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.
Citation
Nature Communications, 2022, 13 (1), pp. 3500 -
Source Title
Nature Communications
Publisher
NATURE PORTFOLIO
ISSN
2041-1723
eISSN
2041-1723
2041-1723
2041-1723