CCHE Seminar Series: Transportability of overall survival estimates from US to Canadian patients with advanced non–small cell lung cancer
Transportability of overall survival estimates from US to Canadian patients with advanced non–small cell lung cancer with implications for regulatory and health technology assessment
Paul Arora
University of Toronto
Friday September 29, 2023, 10am-12pm, HS Room 412 and Zoom
Abstract: Importance: The applicability of survival outcomes from US clinical practice data for patients with advanced non–small cell lung cancer (NSCLC) to non-US contexts, which can influence regulatory decisions and health technology evaluations outside the US, is unclear. Objective: To ascertain if overall survival (OS) estimates from a large US database for patients with advanced NSCLC are comparable to those for Canadian patients undergoing similar treatments. Design, Setting, and Participants: This retrospective study assessed the transportability of OS estimates from US-based samples to Canadian populations by adjusting for pretreatment characteristics. It included 17,432 patients diagnosed with advanced NSCLC between January 1, 2011, and September 30, 2020. Data on race and ethnicity, present in the US but not Canadian database, were excluded from analysis. Exposures: First-line treatments for advanced NSCLC included platinum-doublet chemotherapy or pembrolizumab monotherapy. Main Outcomes and Measures: OS was determined from the commencement of treatment. Results: 15,669 US and 1,763 Canadian patients were analyzed. Adjusted OS estimates for the Canadian cohort showed a minimal mean absolute difference from the observed OS (0.56% over 60 months for chemotherapy and 4.54% over 30 months for pembrolizumab). A mismatched outcome model indicated a 6.64% discrepancy. Conclusions and Relevance: OS estimates from US data, when adjusted for baseline clinical features, can closely mirror OS in certain Canadian advanced NSCLC patient groups. This has potential implications for regulatory and health technology decisions beyond the US.
Paul is an epidemiologist and status assistant professor at the University of Toronto in the Dalla Lana School of Public Health. He has over 15 years of experience in conducting and leading evaluations in public health and clinical epidemiology. He holds PhD in epidemiology and has a strong background in biostatistics and scientific writing, which he applies to generate real-world evidence for health outcomes and policy decisions. He was a co-founder and CEO of Lighthouse Outcomes, a consulting firm that specialized in advanced analytics for clinical epidemiology research, which was acquired by Cytel in 2020. At Cytel, he served as a vice president and led the advanced analytics group in the real-world evidence division, where he worked on the application of cutting-edge epidemiologic methods and machine learning to health outcomes problems. He also continues to teach and mentor students and researchers in the Division of Epidemiology, where he has published more than 50 peer-reviewed scientific articles and developed software for Bayesian network meta-analysis of clinical trials. He is passionate about advancing the field of epidemiology and improving health outcomes through rigorous and innovative research.
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