Alternative approaches for evaluating the comparative effectiveness of cancer therapies using real world data
3655 Promenade Sir William Osler 鈥� Meakins 鈥� Rm 521 聽 ALL ARE WELCOME!
SYNOPSIS: Well-designed randomized controlled trials (RCTs) represent the gold standard for establishing the efficacy of new cancer therapies because they can generate internally valid results. However, barriers to trial enrollment can lead to underrepresentation of particular patient subgroups (e.g., older adults, women, racial/ethnic minorities) and limit the external validity of trial findings. In contrast, observational data sources and epidemiologic methods can be used to estimate the effectiveness of cancer treatments in more generalizable populations treated in routine care settings. Yet, these studies are subject to unmeasured confounding by indication and frailty, which may ultimately threaten internal validity. This talk will provide an overview of various epidemiologic approaches employed to assess cancer therapy effectiveness in real world populations and highlight a novel, hybrid approach that draws upon both RCT and observational data sources.
As a motivating clinical example, the talk will focus on evaluating the comparative effectiveness of alternative adjuvant chemotherapies for the treatment of stage III colon cancer. OBJECTIVES: At the end of this seminar, participants will be able to: 1) define internal and external validity and discuss the relevance of these concepts to the interpretation of findings from RCTs and observational studies of cancer therapies 2) describe the strengths and limitations of alternative epidemiologic approaches for evaluating the comparative effectiveness of cancer therapies using observational data sources 3) explain at a conceptual level how RCT and observational data can be combined to effectively transport RCTs findings to target populations of patients in routine care聽
