| Added by | ircm doc |
|---|---|
| Group name | EquipeCTCS |
| Item Type | Journal Article |
| Title | Assessing the real-world effectiveness of 8 major metastatic breast cancer drugs using target trial emulation |
| Creator | Antoine et al. |
| Author | Alison Antoine |
| Author | David Pérol |
| Author | Mathieu Robain |
| Author | Thomas Bachelot |
| Author | Rémy Choquet |
| Author | William Jacot |
| Author | Béchir Ben Hadj Yahia |
| Author | Thomas Grinda |
| Author | Suzette Delaloge |
| Author | Christine Lasset |
| Author | Youenn Drouet |
| Abstract | BACKGROUND: Demonstration of trial emulation ability to benchmark randomised controlled trials (RCTs) from real-world data (RWD) is required to increase confidence in the use of routinely collected data for decision making in oncology. METHODS: To assess the frequency with which emulation findings align with RCTs regarding effect size on overall survival (OS) in metastatic breast cancer (MBC), 8 of 13 pre-selected pivotal RCTs in MBC were emulated using data from 32,598 patients enrolled in the French ESME-MBC cohort between January 1, 2008 and December 31, 2021. Adjustment methods and confounders were selected a priori for each emulation; stabilized weight was the reference method to mitigate confounding. Concordance in OS hazard ratios with associated 95 % confidence intervals between RCTs and emulations were assessed used predefined metrics based on statistical significance, estimates, and standardized differences. RESULTS: The effect sizes were consistent with RCT results in 7 out of the 8 emulations; 4 emulations achieved full statistical significance agreement; 5 emulations had a point estimate included in the RCT CI (estimate agreement); 6 emulations reported no significant differences between RCT and emulation (standardized difference agreement). Discrepancies related to residual confounders and significant shifts in prescription practices post-drug approval may arise in some cases. CONCLUSION: Target trial emulation from RWD combined with appropriate adjustment can provide conclusions similar to RCTs in MBC. In oncology, this methodology offers opportunities for confirming the impact on long-term survival, for expanding indications in patients excluded from RCTs and for comparative effectiveness in single-arm trials using external control arms. |
| Publication | European Journal of Cancer (Oxford, England: 1990) |
| Volume | 213 |
| Pages | 115072 |
| Date | 2024-12 |
| Journal Abbr | Eur J Cancer |
| Language | eng |
| DOI | 10.1016/j.ejca.2024.115072 |
| ISSN | 1879-0852 |
| Library Catalog | PubMed |
| Extra | PMID: 39476445 |
| Tags | Aged, Antineoplastic Agents, Breast Neoplasms, Causal inference, clinic, Comparative effectiveness, Female, Humans, Middle Aged, Neoplasm Metastasis, Randomized Controlled Trials as Topic, Real-world data, Target trial emulation |
| Date Added | 2025/01/16 - 12:04:45 |
| Date Modified | 2025/01/16 - 12:07:29 |
| Notes and Attachments | PubMed entry (Attachment) Texte intégral (Attachment) |
Institut de Recherche en Cancérologie de
