The New England Journal of Medicine November 23, 2023

Osimertinib with or without Chemotherapy in EGFR-Mutated Advanced NSCLC

David Planchard, Pasi A. Jänne, Ying Cheng, James C-H Yang, Noriko Yanagitani, Sang-We Kim, Shunichi Sugawara, Yan Yu, Yun Fan, Sarayut L Geater, Konstantin Laktionov, Chee K Lee, et al.

Bottom Line

First-line treatment with osimertinib combined with platinum-based chemotherapy significantly prolonged progression-free survival compared to osimertinib monotherapy in patients with EGFR-mutated advanced non-small-cell lung cancer.

Key Findings

1. First-line combination therapy with osimertinib and chemotherapy significantly improved median progression-free survival (PFS) to 25.5 months, compared with 16.7 months for osimertinib monotherapy (HR 0.62; 95% CI, 0.49 to 0.79; P<0.001), representing an absolute median PFS improvement of 8.8 months.
2. The objective response rate (ORR) was nominally higher in the combination group, achieving 83% compared to 76% in the osimertinib monotherapy group.
3. The addition of chemotherapy resulted in a higher incidence of Grade 3 or higher adverse events, largely driven by expected chemotherapy-related toxicities such as cytopenias (e.g., neutropenia and anemia).

Study Design

Design
Phase 3 RCT
Open-Label
Sample
557
Patients
Duration
19.5 mo
Median
Setting
Multicenter, global
Population Adults with previously untreated, locally advanced or metastatic non-squamous NSCLC harboring an EGFR exon 19 deletion or L858R mutation.
Intervention Osimertinib (80 mg daily) plus pemetrexed and platinum-based chemotherapy (cisplatin or carboplatin) for 4 cycles, followed by maintenance osimertinib plus pemetrexed.
Comparator Osimertinib (80 mg daily) monotherapy.
Outcome Progression-free survival (PFS) by investigator assessment.

Study Limitations

The trial utilized an open-label design, which inherently carries a risk of bias in investigator-assessed endpoints, though this was mitigated by concordance with a blinded independent central review.
At the time of primary publication, overall survival (OS) data remained immature, leaving it uncertain whether the 8.8-month progression-free survival advantage will definitively translate into a long-term overall survival benefit.
The combination regimen necessitates intravenous chemotherapy infusions and incurs higher rates of Grade 3 or greater toxicities compared to oral osimertinib alone, impacting patient quality of life and healthcare resource utilization.

Clinical Significance

The FLAURA2 trial established the upfront addition of platinum-pemetrexed chemotherapy to osimertinib as a new, highly efficacious standard-of-care first-line option for EGFR-mutated advanced NSCLC. While it offers a profound delay in disease progression, clinicians must weigh the extended progression-free survival against the additive toxicities of cytotoxic chemotherapy. This regimen is particularly compelling for patients with a high disease burden or symptomatic central nervous system metastases where maximizing early disease control is paramount.

Historical Context

The third-generation EGFR tyrosine kinase inhibitor (TKI) osimertinib redefined the first-line treatment landscape for advanced EGFR-mutated NSCLC after the landmark FLAURA trial demonstrated its superiority over first-generation TKIs in both progression-free and overall survival. Nevertheless, resistance inevitably develops. Drawing on prior studies that showed survival benefits when first-generation TKIs (such as gefitinib) were combined with chemotherapy, the FLAURA2 trial was designed to evaluate whether preemptively combining osimertinib with standard chemotherapy could delay the emergence of resistance and deepen responses in the frontline setting.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the mechanism of action of osimertinib differ from first- and second-generation EGFR TKIs, and why is it effective in both first-line and subsequent-line therapy for EGFR-mutated NSCLC?

Key Response

Osimertinib is a third-generation, irreversible EGFR TKI that selectively targets both canonical EGFR-sensitizing mutations (Exon 19 deletion, L858R) and the T790M resistance mutation, while sparing wild-type EGFR. This dual action explains its superiority over older TKIs and its role in overriding the most common mechanism of acquired resistance.

Resident
Resident

Given the results of FLAURA2, how do you weigh the progression-free survival benefit of adding chemotherapy to osimertinib against the increased toxicity profile in a newly diagnosed, symptomatic patient versus an asymptomatic patient with low tumor burden?

Key Response

While FLAURA2 shows improved PFS, the combination arm had significantly higher rates of grade 3 or higher adverse events. For a highly symptomatic patient, the rapid and deeper response of the combination might outweigh the toxicity. For an asymptomatic patient, osimertinib alone remains highly effective and much better tolerated, making shared decision-making essential.

Fellow
Fellow

How does the addition of platinum-pemetrexed to osimertinib impact the development of specific mechanisms of acquired resistance compared to osimertinib monotherapy, and how might this influence post-progression sequencing?

Key Response

Combining chemotherapy with osimertinib upfront may eradicate diverse sub-clonal populations early, potentially altering dominant acquired resistance mechanisms (like MET amplification or C797S). Furthermore, if a patient progresses on the combination, standard platinum-doublet chemo is no longer an option, leaving a gap in second-line treatments and increasing reliance on novel targeted therapies or clinical trials.

Attending
Attending

With overall survival data still maturing in the FLAURA2 trial, to what extent should a PFS benefit alone drive a paradigm shift to upfront combination therapy, considering the high financial toxicity, clinical burden of intravenous treatments, and diminished quality of life?

Key Response

Historically, sequential therapy (TKI followed by chemo upon progression) has yielded excellent overall survival. Without a proven OS benefit for concurrent administration, the routine use of upfront chemo-osimertinib risks over-treating a subset of patients who would have enjoyed years of progression-free, pill-only treatment with high quality of life. Attendings must balance trial endpoints with real-world patient outcomes.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

In evaluating the FLAURA2 trial design, how does the use of a proportional hazards model account for the potential violation of the proportional hazards assumption over time, particularly when considering the delayed separation of curves often seen in diverse clonal dynamic models?

Key Response

In combination targeted-chemo trials, toxicity-related discontinuations can cause early curve crossing, or benefits may only manifest after the emergence of resistant clones, violating proportional hazards. Analyzing the Restricted Mean Survival Time (RMST) or using piecewise Cox models might provide a more robust statistical interpretation of the durability of the combination's effect compared to a standard hazard ratio.

Journal Editor
Journal Editor

Considering the open-label nature of the FLAURA2 study regarding the chemotherapy arm, how robust is the progression-free survival endpoint given the potential for investigator bias in radiographic assessment and the imbalance in toxicity-related dose modifications?

Key Response

While blinded independent central review (BICR) mitigates bias for the primary endpoint, open-label administration means both patients and investigators knew who received chemotherapy. This can influence toxicity reporting, dose delays, and the timing of clinical progression declarations. Reviewers must scrutinize concordance rates between investigator-assessed and BICR-assessed PFS to ensure the benefit was not artificially inflated.

Guideline Committee
Guideline Committee

Based on the FLAURA2 data, should NCCN and ASCO guidelines formally upgrade osimertinib plus chemotherapy to a Category 1 Preferred recommendation over osimertinib monotherapy for all patients, or should it be restricted to specific high-risk subgroups?

Key Response

Current NCCN guidelines strongly recommend osimertinib monotherapy (Category 1) for first-line EGFR-mutated NSCLC. While FLAURA2 demonstrates a clear PFS advantage, the lack of mature OS data and increased toxicity suggest the combination might be better positioned as an Alternative Preferred option or recommended specifically for high-risk subgroups (e.g., L858R mutations or CNS disease) rather than a blanket replacement of the monotherapy standard.

Clinical Landscape

Noteworthy Related Trials

2017

AURA3 Trial

n = 419 · NEJM

Tested

Osimertinib 80mg daily

Population

Patients with T790M-positive advanced NSCLC progressing after first-line EGFR-TKI therapy

Comparator

Platinum-based therapy plus pemetrexed

Endpoint

Progression-free survival (PFS)

Key result: Osimertinib demonstrated significantly greater efficacy than standard platinum-pemetrexed chemotherapy, with a median PFS of 10.1 months versus 4.4 months.
2018

FLAURA Trial

n = 556 · NEJM

Tested

Osimertinib 80mg daily

Population

Treatment-naive patients with EGFR-mutated advanced NSCLC

Comparator

Standard EGFR-TKIs (gefitinib or erlotinib)

Endpoint

Progression-free survival (PFS)

Key result: Osimertinib significantly improved median PFS compared to standard EGFR-TKIs (18.9 months vs 10.2 months) and prolonged overall survival.
2018

NEJ009 Trial

n = 345 · J Clin Oncol

Tested

Gefitinib plus carboplatin and pemetrexed

Population

Treatment-naive patients with advanced EGFR-mutated NSCLC

Comparator

Gefitinib monotherapy

Endpoint

Progression-free survival (PFS) and overall survival (OS)

Key result: The combination of gefitinib and chemotherapy significantly improved both PFS and OS compared to gefitinib alone, despite increased toxicity.

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