PCORI Invites Full Proposals Under First Round of Pragmatic Studies Initiative

When we announced our new pragmatic clinical studies and large simple trials initiative, we were optimistic that the research community would embrace our effort to build a new portfolio of larger, longer PCORI-funded studies focused on high-impact research topics. And we weren’t disappointed.

Instead of soliciting a wide range of the research community’s best ideas, as our broad funding announcements do, our call for pragmatic clinical studies focuses on a series of topics identified by our own advisory panels, the Institute of Medicine (IOM), and the Agency for Healthcare Research and Quality (AHRQ) because of the topic’s heavy burden on patients, families, and the healthcare system. The contracts under this program—up to five years in duration with up to $10 million in total direct costs—are longer and larger than those we’ve previously awarded.

To be successful, it’s essential that applicants partner with relevant patient, clinician, and other stakeholder organizations. These organizations must strongly endorse the proposed study and plan to be involved with research teams throughout the conduct of the study, so that research questions and outcomes are more relevant and findings more likely to be disseminated and implemented.

With a larger investment and longer study duration, we expect projects in this portfolio to produce findings that can fundamentally shift our understanding of which options are most effective for individual patients. In sum, the pragmatic trials funding program is designed to hit home runs.

An important first step

We were pleased to see a robust response to our call for competitive Letters of Intent (LOIs) for this opportunity, and we have now invited 40 research teams to submit full applications. These teams have proposed research on 27 topics, 13 of which are on our list of topics, while 19 are found on the IOM list, and four are in AHRQ’s catalog of critical topics; many topics addressed in these LOIs are on more than one list.

The most commonly addressed topic among the approved LOIs is cancers, followed by mental health conditions, cardiovascular disease, and other chronic conditions that impose a substantial burden on patients, those who care for them, and the healthcare system overall. But those LOIs are complemented by a diverse range of proposals that study topics such as rare disease, pain, and substance abuse, as I described in my presentation to our Board of Governors on May 5.

The study designs also vary. Sixty percent are trials with randomization of individuals, 27 percent are cluster randomized trials, and 13 percent are large-scale observational studies.

Next steps, including round two

Full applications for this first round of pragmatic clinical studies are due August 8. Applications will go through our scientifically rigorous merit review process in November, after which we expect six to nine to be approved for funding by our Board of Governors in January 2015.

But this is just the inaugural cycle of the initiative. We’ll announce a second round of pragmatic clinical studies funding on May 19 and plan additional cycles beyond that. Watch the Funding Opportunities section of PCORI's website for more information. If you’d like to be sure to be alerted to future funding opportunities, be sure to sign up for our e-mail alerts.

We created this funding category in response to stakeholder feedback and our understanding that many important health research questions require a greater investment and longer timeline than our broad funding announcements permit. As compared to our previously funded randomized controlled trials, studies in the new category include broader and more diverse populations, are conducted in routine clinical care settings, are often simpler and larger, and are intended to provide information that can be directly adopted by healthcare providers.

As the first round of our pragmatic clinical studies funding progresses, we are eager to learn from the process and find opportunities to refine the program in future funding cycles. But most of all, we look forward to funding research teams that will knock the ball out of the park on critical health issues.