JAMA Netw Open. 2025 Jun 2;8(6):e2514519. doi: 10.1001/jamanetworkopen.2025.14519.
ABSTRACT
IMPORTANCE: Targeted therapies and immunotherapies prolong survival but are associated with high costs for patients with advanced non-small cell lung cancer (aNSCLC). To date, little is known about survival and medication cost by biomarker status in the US.
OBJECTIVE: To estimate survival and medication cost by aNSCLC biomarker status.
DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study using Flatiron Health data identified patients diagnosed with aNSCLC from January 1, 2016, through December 31, 2022. Each patient had received at least 1 biomarker test and 1 documented line of therapy; follow-up was through September 31, 2023. Patients were categorized based on the presence of driver alterations, including ALK rearrangement, BRAF variation, or EGFR variation. Patients without driver alterations were divided into 3 groups based on their programmed cell death 1 ligand 1 (PD-L1) expression (<1%, 1%-49%, or ≥50%).
MAIN OUTCOMES AND MEASURES: The primary outcome was medication costs, which were a function of survival probability and monthly medication costs. The secondary outcome was medication costs per survivor, defined as the mean aggregate medication costs within each patient cohort for each 1- or 2-year survivor.
RESULTS: The study cohort consisted of 26 635 patients with aNSCLC (mean [SD] age at diagnosis, 68.9 [10.0] years; 13 750 [52%] male; 2610 [10%] African American, 687 [3%] Asian, 18 352 [69%] White, and 4986 [19%] other race, including any race other than African American, Asian, or White). The median overall survival was 39.9 (95% CI, 33.9-48.5) months for patients with ALK rearrangement, 27.0 (95% CI, 24.8-28.8) months for EGFR variation, 18.7 (95% CI, 16.0-20.6) months for BRAF variation, 12.3 (95% CI, 12.0-12.7) months for PD-L1 less than 1%, 13.7 (95% CI, 13.1-14.3) months for PD-L1 of 1% to 49%, and 16.2 (95% CI, 15.3-17.0) months for PD-L1 of 50% or greater. The 1- and 2-year medication costs per patient for the overall cohort were $120 420 (95% CI, $115 540-$126 470) and $182 560 (95% CI, $172 900-$196 040), respectively. Patients with EGFR variation or PD-L1 of 50% or greater incurred relatively higher 1-year medication cost ($131 700 [95% CI, $125 340-$138 280] and $123 590 [95% CI, $115 970-$130 840], respectively) compared with patients with PD-L1 less than 1% ($110 350 [95% CI, $101 680-$120 040]). Patients with ALK rearrangement or EGFR variation incurred the highest 2-year medication cost ($242 130 [95% CI, $206 220-$267 330] and $241 940 [95% CI, $230 840-$254 730], respectively), whereas patients with PD-L1 less than 1% and PD-L1 of 1% to 49% had the lowest 2-year medication cost ($156 340 [95% CI, $142 450-$172 800] and $163 410 [95% CI, $152 410-$174 180], respectively). The medication costs per 1-year survivor were $152 370 (95% CI, $133 550-$178 080) for patients with ALK rearrangement, $175 720 (95% CI, $167 330-$185 390) for EGFR variation, $211 100 (95% CI, $195 030-$229 400) for PD-L1 less than 1%, $210 260 (95% CI, $193 190-$226 580) for PD-L1 of 1% to 49%, and $211 630 (95% CI, $198 670-$224 210) for PD-L1 of 50% or greater, whereas the costs per 2-year survivor were $363 480 (95% CI, $314 710-$401 320) for patients with ALK rearrangement, $468 400 (95% CI, $441 340-$497 860) for patients with PD-L1 of 50% or greater, $460 790 (95% CI, $427 340-$494 080) for patients with PD-L1 of 1% to 49%, and $500 220 (95% CI, $456 900-$556 730) for patients with PD-L1 less than 1%.
CONCLUSIONS AND RELEVANCE: In this cohort study, patients with aNSCLC with driver alterations experienced better survival and incurred lower medication costs per survivor than those without driver variation, indicating the need to develop more affordable and effective medications for patients without driver alterations.
PMID:40493365 | DOI:10.1001/jamanetworkopen.2025.14519
