Real‐world effectiveness of pembrolizumab in previously treated non‐small cell lung cancer: A population‐based cohort study

Abstract

Purpose: Immunotherapy is promising for lung cancer treatment, although at significant financial impact. The aim of this study was to evaluate the effectiveness and the efficacy-effectiveness gap of pembrolizumab in previously treated non-small cell lung cancer (NSCLC). Methods: A population-based ambispective cohort study was conducted. Cases of interest were identified through the National Cancer Registry database and additional data sources. Patients aged ≥18 years, diagnosed with NSCLC and exposed to pembrolizumab, between 23 June 2016 and 31 October 2018, as second or later lines of treatment for advanced disease were included. Patients were followed-up until death or cutoff date (30 April 2019). Primary outcome was overall survival (OS). Secondary outcomes were progression-free survival (PFS), event-free survival (EFS), and adverse events (AEs) leading to treatment discontinuation. The efficacyeffectiveness gap was evaluated comparing results with clinical trial data. Results: A total of 181 patients were included. Median age was 63 years (range 33-94); 74.6% were male. Median treatment duration was 5.6 months (interquartile range: 1.4-10.4) and, at cutoff date, treatment had been discontinued in 141 patients, mainly due to disease progression. Median OS was 13.0 months (95% confidence interval [CI] 9.3-15.9) and 1-year OS was 53.1% (95% CI 45.2%-60.3%). Median PFS was 5.6 months (95% CI 4.6-7.2), median EFS was 4.7 months (95% CI 3.2-6.0), and treatment was discontinued due to AE in 8.3% of cases (n = 15). The efficacy-effectiveness gap seems to favor pembrolizumab use in clinical practice. Conclusion: Real-world data suggest the performance of pembrolizumab to reflect the clinical trial outcomes in previously treated NSCLC.

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study

“…In the case study of TA531, EHR-derived data available at a recent data cut-off and at NICE’s final decision time point were used to characterise OS and time on treatment. The evidence generated in the case study was consistent with other RWD data studies of broad populations 17–20. At the recent data cut-off, the available sample size was large enough to explore several subgroups that could be of interest to HTA stakeholders, for example, the investigation of a relatively healthy cohort that consists of patients with ECOG PS 0/1 at treatment initiation 21.…”

“…The evidence generated in the case study was consistent with other RWD data studies of broad populations. [17][18][19][20] At the recent data cut-off, the available sample size was large enough to explore several subgroups that could be of interest to HTA stakeholders, for example, the investigation of a relatively healthy cohort that consists of patients with ECOG PS 0/1 at treatment initiation. 21 In contrast, at TA reappraisal, such subgroup analyses were limited by the small sample size.…”

Real-world effectiveness of palbociclib plus fulvestrant in advanced breast cancer: Results from a population-based cohort study

“…This study used data from the Portuguese National Cancer Registry (Registo Oncológico Nacional, RON)'s database, which has been described in detail elsewhere [ [12] , [13] , [14] ]. Briefly, RON is a nationwide population-based cancer registry and its database comprises international recommendations stated by the International Agency for Research on Cancer [ 15 ] and collects relevant information since cancer diagnosis until patient's death.…”