1373P - Measuring PFS in clinical trials and observational studies of patients with NSCLC: A scoping review

Background

Real-world data is increasingly used to complement clinical trial data. However, comparing progression-free survival (PFS) across studies is challenging due to differences in definition and response assessment. This scoping review assessed the characteristics of PFS reported for randomized clinical trials (RCTs) and observational studies of patients with advanced non-small cell lung cancer (NSCLC).

Methods

This study included RCTs and observational studies that compared the PFS of immunotherapy-based regimens in patients with advanced NSCLC with other treatments or trial data. A search was conducted in PubMed and Embase for 2012-2023. Two reviewers screened all records with an AI-assisted review tool (ASReview) and extracted information on study characteristics, treatment regimens, and PFS characteristics (definition of events, follow-up periods, radiological criteria, and assessment schedules.

Results

A total of 40 RCTs and 144 observational studies were included. Most RCTs were conducted in multiple continents (70%), while most observational studies were conducted in Asia (62%). Most RCTs and observational studies used disease progression and death as events for PFS definition. However, the start and end date for follow-up varied, and many observational studies did not report information on these PFS characteristics (Table). In addition, in most RCTs and observational studies, RECIST v1.1 was reported for the evaluation of progression. All RCTs reported highly detailed assessment schedules, whereas most observational studies did not report any information on assessment schedules. Table: 1373P

PFS measurement characteristics in RCTs versus observational studies

Conclusions

Observational studies often fail to report on PFS characteristics and, if reported, often differ from the characteristics reported for RCTs. Better reporting and documentation of PFS characteristics is needed to improve the quality and usability of real-world PFS.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

Authors.

Funding

Has not received any funding.

Disclosure

M. Koopman: Financial Interests, Institutional, Advisory Board, advisory board and speaker: Pierre Fabre; Financial Interests, Institutional, Advisory Board: MSD, Bayer; Financial Interests, Institutional, Advisory Board, Advisory Board, speaker: Servier; Financial Interests, Institutional, Invited Speaker: Merck, BMS; Financial Interests, Institutional, Trial Chair: Servier; Financial Interests, Institutional, Research Grant: Servier, Roche, Bayer, Bristol Myers Squibb, Merck, Personal Genomics Diagnostics, Sirtex, Pierre Fabre; Financial Interests, Institutional, Funding: Pierre Fabre, Amgen, Nordic Farma, Novartis, Merck, Servier, BMS; Non-Financial Interests, Leadership Role, vice-chair of DCCG: Dutch Colorectal Cancer Group; Non-Financial Interests, Other, ESMO faculty member for the Gastro-Intestinal Tumours – colorectal cancer: ESMO; Non-Financial Interests, Advisory Role, expert member of committee “regie op registers dure geneesmiddelen” ZINNL: ZiNNL; Non-Financial Interests, Advisory Role, CRC expert on Kanker.nl platform for answering online CRC questions of CRC (non) patients: Patient respresentative organisation (Kanker.nl); Non-Financial Interests, Leadership Role, chair of RWD & DH working group: ESMO; Other, PI of the Dutch Prospective Colorectal Cancer Cohort study: PLCRC project. All other authors have declared no conflicts of interest.