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
March 19: OHDSI: FDA Workshop: Developing & Submitting Proposed Draft Guidance on Patient Experience DataMarch 19 @ 1:00 pm - 5:00 pm
- March 23 @ 1:00 pm - 2:00 pm
- NIH Course on Biostatistics of Pragmatic and Group-Randomized Trials - Free 7-part online course
- March 15, 2018: New Resource for Understanding Ethical and Regulatory Architecture of Patient-Centered Outcomes Research Data Using patient-centered outcomes research (PCOR) data requires balancing the need for sufficient private health information to support meaningful research with the need to protect patient privacy and autonomy. In support of this dual goal, The Office of the National Coordinator for Health Information Technology (ONC) has just released a document that provides a collection of ...
- March 14, 2018: Public Input on Patient-Reported Outcome Measures Due April 1 The Agency for Healthcare Research and Quality (AHRQ) is requesting information to help inform a competition in the fall to develop and implement user-friendly technical tools for the collection of patient-reported outcome (PRO) data. The AHRQ is interested in learning about experiences with physical function PRO measures that are currently in use in ambulatory care ...
- March 7, 2018: FDA Offers Workshop on Submitting Draft Guidance on Patient Experience Data The FDA is conducting a public workshop on Monday, March 19, to obtain input from stakeholders—including patients, patient advocates, academic and medical researchers, expert practitioners, drug developers, and other interested persons—to inform the drafting of a patient-focused drug development guidance as required by the 21st Century Cures Act. Workshop attendees will discuss considerations for development ...
- New Grand Rounds Podcasts
- 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