Materials and video recordings are now available from the NIH Pragmatic Trials Collaboratory’s recent workshop, “Endpoint Strategy in Pragmatic Trials: From Big Questions to Meaningful Outcomes.“
The workshop shared evidence and best practices in thoughtfully selecting endpoints and interpreting treatment effects in pragmatic clinical trials embedded in healthcare systems to best inform decision-making.
The first day of the workshop focused on selecting the endpoint strategy, introduced the foundational connections between endpoints and meaningfulness, and examined endpoint complexities encountered in the design and analysis of pragmatic trials. The second day of the workshop focused on the meaningfulness of treatment effects, outlined key concepts in interpreting treatment effects, and introduced statistical analysis considerations for handling complex endpoint scenarios in pragmatic trials.
Access the complete workshop materials, including slides and videocast recordings.
- Session 1: Aiming for Impact: What is the Right Concept to Target?
- Steven George, PT, PhD (moderator); Michael Ho, MD, PhD; Frederick Masoudi, MD, MSPH; Miguel Vazquez, MD; Tracy Wang, MD, MHS, MSc; Wendy Weber, ND, PhD, MPH
- Session 2: The Estimands Framework: Aligning Objectives, Endpoints, and Analyses
- Liz Turner, PhD (moderator); Elizabeth Colantuoni, PhD, ScM; Fan Li, PhD; Richard Skolasky, Jr, MA, ScD; Hulin Wu, PhD
- Session 3: Working With Multiple Outcomes: Multicomponent Endpoints and Beyond
- Emily O’Brien, PhD (moderator); Julie Fritz, PhD, PT; Patrick Heagerty, PhD; Eric Roseen, DC, PhD; Pamela Shaw, PhD, MS; Qilu Yu, PhD
- Session 4: Unpacking Uncertainty, Meaningfulness, and Magnitude
- Greg Simon, MD, MPH (moderator); Devon Check, PhD; Lynn DeBar, PhD, MPH; Juan Marcos Gonzalez, PhD; Laura Lee Johnson, PhD; Natalia Morone, MD, MS
- Session 5: Toward Best Practices for Interpretation of Endpoints
- Adrian Hernandez, MD, MHS (comoderator); Kevin Weinfurt, PhD (comoderator); Andrea Cook, PhD; Corita Grudzen, MD, MSHS; Liz Turner, PhD; Kari Stephens, PhD; Christy Zigler, PhD, MSEd