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
Grand Rounds June 29: Policy & Priorities: Rethinking University Research with State Data (Aaron McKethan, PhD)June 29 @ 1:00 pm - 2:00 pm
- July 6 @ 1:00 pm - 2:00 pm
- NIH Course on Biostatistics of Pragmatic and Group-Randomized Trials - Free 7-part online course
- June 14, 2018: NIH Collaboratory Members to Host Session on Embedded PCTs at AcademyHealth Annual Research Meeting The upcoming AcademyHealth Annual Research Meeting, June 24-26, 2018, will feature a special session on Embedding Pragmatic Clinical Trials in Health Care Systems: Trials and Tribulations, Monday, June 25, at 5:15 pm, hosted by members of the Health Care Systems Interactions Core Working Group: Chair: Cathy Meyers, MD, NIH/NICCH Discussant: Eric B. Larson, MD, MPH, Kaiser Permanente ...
- June 7, 2018: NIH Releases First Strategic Plan for Data Science On June 4, the National Institutes of Health (NIH) released its first Strategic Plan for Data Science. The plan outlines steps the agency will take to modernize research data infrastructure and resources and to maximize the value of data generated by NIH-supported research. Data science challenges for NIH have evolved and grown rapidly since the launch ...
- June 4, 2018: New Article Explores Misleading Use of the Label “Pragmatic” for Some Randomized Clinical Trials A recent study published in BMC Medicine found that many randomized controlled trials (RCTs) self-labeled as “pragmatic” were actually explanatory in nature, in that they assessed investigational medicines compared with placebo to test efficacy before licensing. Of the RCTs studied, one-third were pre-licensing, single-center, or placebo-controlled trials and thus not appropriately described as pragmatic. Appropriately describing ...
- New Grand Rounds Podcasts
- Podcast April 9, 2018: Data Science in the Era of Data Ubiquity (Robert Califf, MD)
- Considerations for the Return of Genomic Results (Andrew Faucett, MS, LGC)
- IMPACT-AFib: An 80,000 Person Randomized Trial Using the Sentinel Initiative Platform (Richard Platt, MD, MS; Christopher Granger, MD, FACC, FAHA)
- Data and Safety Monitoring in Pragmatic Clinical Trials (Greg Simon, MD, MPH, and Susan Ellenberg, PhD)
- Moderators’ Edition: Discussion of the Future of the NIH Collaboratory (Moderators' Edition)
- NIH Collaboratory policies and guidance documents
- NIH Collaboratory milestone timeline
- Demonstration Projects: Regulatory and Ethics Discussions
- Stakeholder Interviews with Collaboratory Cores and Demonstration Projects