In 2019, NIH Health Care Systems Research Collaboratory held a comprehensive workshop to explore and discuss statistical issues encountered with embedded pragmatic clinical trials (ePCTs). The new Workshop Summary describes panel discussions with the principal investigators and statisticians of NIH Collaboratory Demonstration Projects and the challenges and solutions encountered during the design and analysis of their trials.
The 4 panel discussions covered the following topics:
- Measurement and Data: Outcomes, Exposures, and Subgroups Based on EHR Data
- To Cluster or Not to Cluster?
- Choosing a Parallel Group or Stepped-Wedge Design
- Unique Complications
This Workshop Summary also provides lessons learned and recommends tools to help others design and analyze future ePCTs. For more on the design and analysis of pragmatic clinical trials, see the tools provided by the Biostatistics and Study Design Core and Living Textbook chapters on Experimental Designs and Randomization Schemes and Analysis Plans.