Optimized Treatment Sequences for ALK+ Stage IV NSCLC: Cost-Effectiveness Analysis From RCT data and Real-World Evidence
Author(s)
Rahyssa R. Sales, PhD1, Bruna Carvalho Silva, MD2, Rafael Duarte Paes, BSc2, Rodrigo Dienstmann, PhD, MD2, Deborah Marta dos Santos Oliveira, MSc2, Mariana Tosello Laloni, PhD, MD2, Ana Caroline Zimmer Gelatti, PhD, MD2, William Nassib William Junior, PhD, MD2, Carlos Gil Moreira Ferreira Gil, PhD, MD2, Pedro Nazareth Aguiar Junior, PhD, MD2.
1Biologist, Oncoclínicas&Co/MedSir, São Paulo, Brazil, 2Oncoclínicas&Co/MedSir, São Paulo, Brazil.
1Biologist, Oncoclínicas&Co/MedSir, São Paulo, Brazil, 2Oncoclínicas&Co/MedSir, São Paulo, Brazil.
Presentation Documents
OBJECTIVES: The rising costs associated with new cancer treatment technologies have become a significant concern. Patients with ALK-mutated non-small cell lung cancer (NSCLC) experience long survival rates, yet there is no established standard treatment. The lack of cost-effectiveness studies adds to the uncertainty surrounding pharmacoeconomic models. This study aims to analyze the cost-effectiveness relationship between randomized clinical trials (RCT) and real-world evidence (RWE) involving 126 patients with ALK-mutated stage IV NSCLC from Oncoclínicas&Co/MedSir, Brazil, between 2007 and 2024.
METHODS: An analytical decision model was developed, considering transitions between six health states: first-line progression-free survival (PFS) with tyrosine kinase inhibitors (TKIs; lorlatinib, alectinib, brigatinib), first-line post-progression survival (PPS), second-line PFS with TKIs, third-line PFS with chemotherapy, PPS, and death. The duration of each state was determined by the median PFS and the area under the PFS and overall survival (OS) curves, estimated over 20 and 30 years using an exponential distribution. Costs were limited to drug purchases, as toxicity management costs were negligible.
RESULTS: The QALY for brigatinib was 6.01 in RCT versus 5.10 in RWE, while the LYS was 7.73 RCT versus 7.48 RWE. For alectinib, QALY was 6.96 RCT versus 6.60 RWE, and LYS was 8.42 RCT versus 9.40 RWE. Lorlatinib had QALY and LYS of 7.71 and 9.40, respectively, in RCT. The first-line TKI costs in RCT (972,105 BRL) were higher than in RWE (644,360 BRL), while second-line TKI costs in RWE (710,014 BRL) exceeded RCT costs (140,854 BRL).
CONCLUSIONS: RWE clarified some uncertainties in the model, but the cost-effectiveness ratio remains unfavorable across both models, likely due to the high costs of the evaluated technologies.
METHODS: An analytical decision model was developed, considering transitions between six health states: first-line progression-free survival (PFS) with tyrosine kinase inhibitors (TKIs; lorlatinib, alectinib, brigatinib), first-line post-progression survival (PPS), second-line PFS with TKIs, third-line PFS with chemotherapy, PPS, and death. The duration of each state was determined by the median PFS and the area under the PFS and overall survival (OS) curves, estimated over 20 and 30 years using an exponential distribution. Costs were limited to drug purchases, as toxicity management costs were negligible.
RESULTS: The QALY for brigatinib was 6.01 in RCT versus 5.10 in RWE, while the LYS was 7.73 RCT versus 7.48 RWE. For alectinib, QALY was 6.96 RCT versus 6.60 RWE, and LYS was 8.42 RCT versus 9.40 RWE. Lorlatinib had QALY and LYS of 7.71 and 9.40, respectively, in RCT. The first-line TKI costs in RCT (972,105 BRL) were higher than in RWE (644,360 BRL), while second-line TKI costs in RWE (710,014 BRL) exceeded RCT costs (140,854 BRL).
CONCLUSIONS: RWE clarified some uncertainties in the model, but the cost-effectiveness ratio remains unfavorable across both models, likely due to the high costs of the evaluated technologies.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE298
Topic
Economic Evaluation
Disease
SDC: Oncology