Rethinking Clinical Trials: A Living Textbook of Pragmatic Clinical Trials
Welcome to the Living Textbook of pragmatic clinical trials, a collection of knowledge from the NIH Health Care Systems Research Collaboratory. Pragmatic clinical trials are performed in real-world clinical settings with highly generalizable populations to generate actionable clinical evidence at a fraction of the typical cost and time needed to conduct a traditional clinical trial. They present an opportunity to efficiently address critical knowledge gaps and generate high-quality evidence to inform medical decision-making. However, these trials pose different challenges than are typically encountered with traditional clinical trials. The Living Textbook reflects a collection of expert consensus regarding special considerations, standard approaches, and best practices in the design, conduct, and reporting of pragmatic clinical trials. Given the rapid pace of change in this field, this electronic textbook will continue to be added to and updated.
By sharing lessons from our experience with pragmatic research, we aim to facilitate large-scale collaborative research studies with healthcare systems. The Living Textbook relies on the following knowledge sources:
DEMONSTRATION PROJECTS: Pragmatic clinical
trials that address questions of major public health importance and provide proof of concept for innovative pragmatic research designs.
CORES: Working groups that support the conduct of Demonstration Projects and generate guidance addressing implementation challenges.
DISTRIBUTED RESEARCH NETWORK: Network enabling investigators to collaborate in the use of electronic health data while safeguarding protected health information.
Upcoming Learning Opportunities
December 13: Research Methods Resources: A New Website from the NIH Office of Disease Prevention (David M. Murray, PhD)December 13 @ 11:00 am - 12:00 pm
Grand Rounds December 15: Does Machine Learning Have a Place in a Learning Health System? (Michael Pencina, PhD)December 15 @ 1:00 pm - 2:00 pm
- NIH Course on Biostatistics of Pragmatic and Group-Randomized Trials - Free 7-part online course
- December 12, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Patient-Reported Outcomes Core We recently asked Dr. Kevin Weinfurt, Chair of the Patient-Reported Outcomes (PRO) Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. He says the biggest impact of the Core has been working with national initiatives to improve inclusion of PROs in the electronic health record (EHR). Further, Core ...
- December 7, 2017: Dr. Greg Simon Explains Individual, Cluster, and Stepped-Wedge Randomization in a New Prop Video In a new video in the Living Textbook, Dr. Greg Simon describes the differences between individual, cluster, and stepped-wedge randomization using props, including marbles, Play-Doh, and glassware. “In the end, it’s all about randomly assigning who gets which treatment, or who gets which treatment when, so that we’re able to make some un-biased judgement about which ...
- December 5, 2017: Dr. Greg Simon Answers the Question: What is a Pragmatic Trial? In a new video for the Living Textbook, Dr. Greg Simon uses props—including Play-Doh and toys—to define pragmatic trials and their importance in clinical research. “Pragmatic clinical trials are about helping doctors make better decisions to take care of their patients.” —Greg Simon, MD
- New Grand Rounds Podcasts
- Moderators’ Edition: Discussion of the Future of the NIH Collaboratory (Moderators' Edition)
- Bringing Machine Learning to the Point of Care to Improve Suicide Prevention (Greg Simon, MD, MPH)
- Health Is More Than Healthcare (Karen DeSalvo, MD, MPH, MSc)
- Preprints: What, Why Not, and How (Harlan Krumholz, MD)
- NIH Collaboratory policies and guidance documents
- NIH Collaboratory milestone timeline
- Demonstration Projects: Regulatory and Ethics Discussions
- Stakeholder Interviews with Collaboratory Cores and Demonstration Projects