#TBT: Pragmatic Clinical Trials

After deciding that we didn’t want to post embarrassing photos on Instagram, we opted instead to bring the “Throwback Thursday” concept to our blog. Starting today, on every other Thursday we’re planning to take a look at a research topic that’s currently in the news and tag it with previous research, videos or commentaries in a relevant way. As the saying goes, “what’s old is new again” – and we hope you enjoy our wonky twist on #tbt.

Pragmatic clinical trials (PCTs) popped back on our radar recently, with the Patient-Centered Outcomes Research Institute’s (PCORI) announcement that they are planning to fund this type of research in 2014. “We hope to fund a greater number of larger, longer studies, including large pragmatic trials, to answer critical questions that will help patients and those who care for them make better-informed health and healthcare decisions,” said PCORI Executive Director Dr. Joe Selby.

PCTs are randomized trials that are designed to determine the risks, benefits, and costs of an intervention as they would occur in routine clinical practice and whether a treatment will work under real-life conditions in a way that matters to patients. They can provide a middle ground between a randomized controlled trial, which looks at whether a clinical intervention can work in a highly regulated, carefully chosen, treatment-adherent patient population, and an observational study, which studies the effectiveness of the intervention in the real world. PCTs are being used more often today because of the kind of information that they provide to health care decision-makers.

PCORI’s Pragmatic Clinical Trials Initiative will allow for longer study durations and emphasize high-priority questions that cannot be answered by more traditional study types in other research areas. Some of this research will be initiated as a PCT under PCORI’s national Clinical Data Research Networks (CDRN), Patient Powered Research Networks (PPRN) or targeted funding announcements, such as such as how to diagnose and manage bipolar disorder. PCORI also is initiating a funding announcement dedicated solely to PCTs and Large Simple Trials to evaluate patient-centered outcomes, with letters of intent due by March 7.

In addition, the National Institutes of Health, through its Clinical and Translational Science Awards Consortium, published a paper this week that outlined the need for a pragmatic clinical trial infrastructure.

Since it’s clear we’ll be hearing a lot more on this topic, we decided to bring back a video explaining PCTs in simple terms. Dr. Marc Berger, now vice president of real world data and analytics at Pfizer Inc, spoke with us in 2011 about the concept as well as the importance of using different types of comparative effectiveness research study designs to answer research questions.