Real-world costs, treatment patterns, and clinical outcomes associated with treatments for advanced anaplastic lymphoma kinase-positive non-small cell lung cancer.

Abstract

Background: Alectinib, brigatinib, and lorlatinib are all preferred first-line (1L) therapies for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) in National Comprehensive Cancer Network (NCCN) guidelines. Although clinical trials have demonstrated their efficacy, real-world evidence on treatment patterns, costs, and outcomes may help differentiate these therapies and inform optimal 1L treatment selection in the absence of head-to-head comparisons.

Objective: To evaluate real-world outcomes for patients with ALK+ NSCLC receiving 1L ALK tyrosine kinase inhibitors (TKIs), focusing on drug acquisition costs, health care utilization, and clinical outcomes.

Methods: This retrospective observational cohort study used data from Optum's deidentified Clinformatics Data Mart Database (2016-2021). Patients were identified using International Classification of Diseases, Tenth Revision codes for lung cancer and ALK TKI pharmacy claims. Eligible patients were aged 18 years, with at least 6 months of continuous enrollment prior to the index date and at least 1 ALK TKI prescription fill. Health care resource utilization (proxied by claim counts) and associated costs (2024 US dollars) were measured per-patient-per-month (PPPM). Time to treatment discontinuation or death (TTD) and overall survival (OS) were assessed using the Kaplan-Meier method.

Results: Among 696 patients, the 1L therapy distribution was crizotinib (n = 366), alectinib (n = 267), brigatinib (n = 22), ceritinib (n = 25), and lorlatinib (n = 16). Total PPPM costs were $28,216 (SD: $29,017). Average 30-day supply costs for ALK TKIs were $17,766 (SD: $2,797). Median OS was 25.5 months (95% CI: 21.1-32.5), and median TTD for 1L therapy was 8.0 months (95% CI: 6.4-9.6). Only 24.3% of patients transitioned to another ALK TKI in a second-line (2L) setting, highlighting high discontinuation rates. Alectinib and lorlatinib were the most common 2L therapies.

Conclusions: This study highlights the economic burden and variable clinical outcomes among patients with advanced ALK+ NSCLC. These real-world estimates inform cost-effectiveness analyses and clinical decision-making regarding treatment sequencing, particularly given uncertainty surrounding multiple preferred 1L options in clinical guidelines.