Related Experiment Video
Updated: Jan 28, 2026
Studying Triple Negative Breast Cancer Using Orthotopic Breast Cancer Model
Published on: March 20, 2020
From Diagnostic-Therapeutic Pathways to Real-World Data: A Multicenter Prospective Study on Upfront Treatment for
Giulia Pasello1, Giovanni Vicario2, Fable Zustovich3
1Medical Oncology 2, Istituto Oncologico Veneto (IOV) Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Padova, Italy giulia.pasello@iov.veneto.it.
Introduction:
Gefitinib, erlotinib, and afatinib represent the approved first-line options for epidermal growth factor receptor (EGFR)-mutant non-small cell lung cancer (NSCLC). Because pivotal trials frequently lack external validity, real-world data may help to depict the diagnostic-therapeutic pathway and treatment outcome in clinical practice.
Methods:
MOST is a multicenter observational study promoted by the Veneto Oncology Network, aiming at monitoring the diagnostic-therapeutic pathway of patients with nonsquamous EGFR-mutant NSCLC. We reported treatment outcome in terms of median time to treatment failure (mTTF) and assessed the impact of each agent on the expense of the regional health system, comparing it with a prediction based on the pivotal trials.
Results:
An EGFR mutation test was performed in 447 enrolled patients, of whom 124 had EGFR mutation and who received gefitinib (n = 69, 55%), erlotinib (n = 33, 27%), or afatinib (n = 22, 18%) as first-line treatment. Because erlotinib was administered within a clinical trial to 15 patients, final analysis was limited to 109 patients. mTTF was 15.3 months, regardless of the type of tyrosine kinase inhibitor (TKI) used. In the MOST study, the budget impact analysis showed a total expense of €3,238,602.17, whereas the cost estimation according to median progression-free survival from pivotal phase III trials was €1,813,557.88.
Conclusion:
Good regional adherence and compliance to the diagnostic-therapeutic pathway defined for patients with nonsquamous NSCLC was shown. mTTF did not significantly differ among the three targeted TKIs. Our budget impact analysis suggests the potential application of real-world data in the process of drug price negotiation.
Implications For Practice:
The MOST study is a real-world data collection reporting a multicenter adherence and compliance to diagnostic-therapeutic pathways defined for patients with epidermal growth factor receptor-mutant non-small cell lung cancer. This represents an essential element of evidence-based medicine, providing information on patients and situations that may be challenging to assess using only data from randomized controlled trials, e.g., turn-around time of diagnostic tests, treatment compliance and persistence, guideline adherence, challenging-to-treat populations, drug safety, comparative effectiveness, and cost effectiveness. This study may be of interest to various stakeholders (patients, clinicians, and payers), providing a meaningful picture of the value of a given therapy in routine clinical practice.
Insights
Real-world data from the MOST study show that first-line epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) for non-small cell lung cancer (NSCLC) have similar treatment failure times. Budget impact analysis highlights the value of real-world data in drug price negotiations.
Area of Science:
- Oncology
- Pharmacoeconomics
- Real-world evidence
Background:
- Gefitinib, erlotinib, and afatinib are approved first-line treatments for EGFR-mutant NSCLC.
- Pivotal trials often lack external validity, necessitating real-world data to understand clinical practice outcomes.
Purpose of the Study:
- To monitor the diagnostic-therapeutic pathway for patients with nonsquamous EGFR-mutant NSCLC.
- To report treatment outcomes, including median time to treatment failure (mTTF).
- To assess the cost impact of first-line TKIs on the regional health system.
Main Methods:
- The MOST study is a multicenter observational study.
- 124 patients with EGFR-mutant NSCLC received first-line gefitinib, erlotinib, or afatinib.
- Median time to treatment failure (mTTF) and budget impact analysis were performed.
Main Results:
- Median time to treatment failure (mTTF) was 15.3 months, irrespective of the TKI used.
- The real-world study's total expense was €3,238,602.17, compared to a predicted cost of €1,813,557.88 based on pivotal trials.
- Good regional adherence to diagnostic-therapeutic pathways was observed.
Conclusions:
- Real-world data demonstrate comparable mTTF across the three targeted TKIs for EGFR-mutant NSCLC.
- Budget impact analysis suggests real-world data can inform drug price negotiations.
- The study underscores the value of real-world evidence in understanding therapy value in routine clinical practice.
Related Concept Videos
01:29Appendicitis-II: Diagnostic Studies and Management
Diagnosing Appendicitis
It requires a multifaceted approach, starting with a detailed physical examination to pinpoint the location and nature of the pain and identify any associated symptoms. Laboratory tests play a crucial role. A complete Blood Count (CBC) typically reveals leukocytosis (an increased number of...
01:21Aneurysm II: Clinical Manifestations and Diagnostic Studies
01:28Acute Pyelonephritis II: Diagnostic Studies and Management
01:22Case Studies
01:30Hypertension III: Clinical Manifestations and Diagnostic Studies
01:20Venous Thrombosis II: Clinical Manifestations and Diagnostic Studies