Clinical Management and Outcomes of Patients in England With eGFR Mutation-Positive Non-Small Cell Lung Cancer: A Retrospective Observational Study and Its Impact on UK Health Technology Assessments
Author(s)
Elise R. Bell, MSc1, Craig S. Knott, PhD2, Sasha D. Borges, MIPH1, Mark L. James, MSc1, Maximiliaan A. Nievaart, Pharmacy1, Cristina Martin-Fernández, BSc, MRes, PhD1.
1Johnson & Johnson, High Wycombe, United Kingdom, 2HDI, Cambridge, United Kingdom.
1Johnson & Johnson, High Wycombe, United Kingdom, 2HDI, Cambridge, United Kingdom.
OBJECTIVES: The management of patients with epidermal growth factor receptor mutation positive (EGFRm) non-small cell lung cancer (NSCLC) poses significant challenges, particularly for those with Exon 20 insertion mutations (Exon20ins) compared to those with common EGFR mutations (cEGFR). This study aims to evaluate the clinical management and outcomes for patients, and the associated implications for UK Health Technology Assessments.
METHODS: A retrospective observational study was conducted utilising data from the National Disease Registration Service (NDRS) for patients diagnosed with NSCLC in England between 2016 and 2021 with follow up until March 2023. Patients were categorised into two cohorts based on their EGFR mutation subtype: Exon20ins and cEGFR. Key clinical outcomes, including overall survival (OS) and time to next treatment (TTNT), were assessed, alongside the calculation of proportional quality-adjusted life year (QALY) shortfalls.
RESULTS: Of the 103,441 patients diagnosed, 11.0% were found to be EGFRm, with a median OS of 19.1 months for Exon20ins patients, significantly lower than the 24.6 months observed in the cEGFR cohort. The introduction of osimertinib benefited cEGFR patients OS and TTNT, with 90.5% receiving this targeted therapy post its reimbursement decision. Nearly half of the Exon20ins patients did not receive subsequent treatment, compared to 32% in the cEGFR group. Proportional QALY shortfall estimates indicated a justification for a NICE severity modifier of 1.2 for Exon20ins patients, whilst for cEGFR patients the proportional shortfall approached the 0.85 threshold, indicating a significant unmet need with the current standard of care.
CONCLUSIONS: This study underscores the urgent need for improved treatment options for both Exon20ins and cEGFR patients in England, emphasising the necessity of access to effective therapies. Additionally, it highlights the importance of utilising national real-world data to support decision making in health technology assessments, thereby facilitating future improvements in patient outcomes and access to new treatments.
METHODS: A retrospective observational study was conducted utilising data from the National Disease Registration Service (NDRS) for patients diagnosed with NSCLC in England between 2016 and 2021 with follow up until March 2023. Patients were categorised into two cohorts based on their EGFR mutation subtype: Exon20ins and cEGFR. Key clinical outcomes, including overall survival (OS) and time to next treatment (TTNT), were assessed, alongside the calculation of proportional quality-adjusted life year (QALY) shortfalls.
RESULTS: Of the 103,441 patients diagnosed, 11.0% were found to be EGFRm, with a median OS of 19.1 months for Exon20ins patients, significantly lower than the 24.6 months observed in the cEGFR cohort. The introduction of osimertinib benefited cEGFR patients OS and TTNT, with 90.5% receiving this targeted therapy post its reimbursement decision. Nearly half of the Exon20ins patients did not receive subsequent treatment, compared to 32% in the cEGFR group. Proportional QALY shortfall estimates indicated a justification for a NICE severity modifier of 1.2 for Exon20ins patients, whilst for cEGFR patients the proportional shortfall approached the 0.85 threshold, indicating a significant unmet need with the current standard of care.
CONCLUSIONS: This study underscores the urgent need for improved treatment options for both Exon20ins and cEGFR patients in England, emphasising the necessity of access to effective therapies. Additionally, it highlights the importance of utilising national real-world data to support decision making in health technology assessments, thereby facilitating future improvements in patient outcomes and access to new treatments.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE133
Topic
Clinical Outcomes, Economic Evaluation, Health Technology Assessment
Topic Subcategory
Thresholds & Opportunity Cost
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology