Some challenges in the analysis and interpretation of large pragmatic (and possibly “simple”) safety studies
View Presentation *Jesse A Berlin, Janssen Research & Development, LLC Yiting Wang, Janssen Research & Development Marsha Wilcox, Janssen Research & Development Keywords: safety, pragmatic trials, causal inference, large simple studies Understanding the potential increase in the risk of uncommon adverse events associated with exposure to medical products requires large sample sizes, only seldom achieved in typical development programs. Applicability of most programs to real world use can also be limited. The use of large pragmatic trials can potentially answer these questions in a less biased way than observational studies, however, challenges can arise from treatment discontinuation or switching. The ITT analysis addresses the effects of offering treatment. Per-protocol analysis may introduce selection bias. Applying methods for causal inference might address questions such as whether the relative risk is less than a specified value among compliers. This talk will outline these issues, present examples, and introduce (at a high level) some potential approaches to analyses that might be helpful in these situations.
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Key Dates
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April 30 - May 22, 2013
Invited Abstract Submission Open -
June 4, 2013
Online Registration Opens -
August 9 - August 23, 2013
Invited Abstract Editing -
August 23, 2013
Short Course materials due from Instructors -
August 26, 2013
Housing Deadline -
September 9, 2013
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 16 - September 18, 2013
Marriott Wardman Park, Washington, DC