I keep a close eye on PCORI’s research investments to make sure we do everything we can to fund studies that will help patients and those who care for them make better-informed healthcare decisions. So I’d like to share three things I find particularly impressive—including the dramatic impact PCORI is starting to have on the culture of research in the United States.

First, I am pleased to see just how robust a portfolio we have of comparative clinical effectiveness research, or CER, projects, studying which of two or more care options work best, for whom, under which circumstances, based on outcomes most important to patients. After all, that’s why Congress authorized our creation six years ago.

With our Board of Governors' approval* of our latest round of studies, we have awarded more than $1.38 billion since 2012 for research and related projects. Some $995 million—about 72 percent of the total—goes to more than 300 patient-centered CER studies, and $391 million supports 193 other projects that directly advance our mission. This includes $109 million in research to improve the science and methods of CER, as Congress intended us to do, and $282 million for the National Patient-Centered Clinical Research Network, or PCORnet, our unique effort to harness the power of data and partnerships to conduct critical patient-centered outcomes research faster, more effectively, and at lower cost than is possible now.

Focus, Focus, Focus

Second, I am impressed by how much our portfolio has evolved in the past couple of years to include more and larger studies targeting conditions that impose a high burden on patients, their families, and the healthcare system as a whole. These include numerous studies of new and older drugs, medical procedures, and other approaches to prevention, diagnosis, and treatment. The range of CER studies we’ve funded reflects the interests of the patients, caregivers, clinicians, payers, and other healthcare stakeholders we serve—and the expanse of care delivered in diverse, real-world settings.

We’d planned from early on to complement our broad calls for researchers’ best ideas for CER studies of any kind with larger funding announcements addressing specific high-impact topics and conditions identified by our stakeholders through our research prioritization process. The pace of this shift has picked up sharply of late based on stakeholder input, especially in the area of direct comparisons of healthcare interventions, such as one drug to another or surgical to nonsurgical procedures.

The proportion of our portfolio invested in such projects to date has jumped from 17 percent in 2013 to more than 40 percent of our research funding through 2015, and we project it will be 60 percent or more in the next few years. Much of this focused research is being conducted in the form of pragmatic clinical studies, which usually are set in real-life practice situations, such as typical hospital and outpatient care settings, and involve more diverse patient groups than traditional trials.

To date, our Board has approved about $459 million for 49 targeted and pragmatic studies. These projects include numerous studies involving drugs and surgical procedures and address a wide variety of concerns (see box). On the horizon: potential funding announcements on new oral blood thinners, disease-modifying therapies for multiple sclerosis, and new ways to reduce long-term opioid use in managing chronic pain.

Changing the Conversation

When I stepped back for a moment and considered both the portfolio we’re building and the input we’re getting from the healthcare community, something else struck me. PCORI, which was little more than a concept in 2010, is now changing the conversation about how health research is conducted in this country. And in the process, I firmly believe, our leadership is helping to change the culture of research itself.

We’ve done this through our commitment to focusing on the issues and outcomes most important to patients. We work to make good on that commitment every day by engaging patients and their families, along with researchers, clinicians, insurers, health systems, and others across the healthcare community in all aspects of our work.

Every day, we see examples of how we’re helping to change the conversation about research. It’s a theme threaded through the growing body of papers our awardees are publishing in top scientific journals, including those in a new theme issue of Health Affairs on patients’ and consumers’ use of evidence. One of those authors, Steven H. Woolf, MD, MPH, Virginia Commonwealth University Center on Society and Health, has said that “engaging patients as equal partners creates a respectful, trusting relationship that improves the quality of research.” Another awardee, cancer researcher Supriya Mohile, MD, MS, of the University of Rochester, says she’ll “never again be able to conceptualize or implement a research project without the input of these important stakeholders.” And Andrea Jensen, a caregiver who has worked with us, called her experience “a unique opportunity ... to help researchers understand what it’s really like to live with [a condition] day in and day out.”

We’re also seeing our approach have an impact on many leading research and healthcare institutions. For example, the Geisinger Health System cites our work as an influence in its developing a strategic vision for patient engagement in research. Our emphasis on engagement is informing training programs and research efforts at a half-dozen or more academic medical centers, including the University of Texas San Antonio, Tulane University, and the Universities of Arkansas, Pennsylvania, Pittsburgh, and Wisconsin. And the American Heart Association cites PCORI as inspiration for the creation of its Aspire Alliance.

PCORI was not the first to engage patients in the research process, nor are we alone in changing the conversation about health research to become more patient-centered. Congress, federal health agencies, health systems, and industry are increasingly taking similar approaches. But we’d like to think our leadership is one reason why this trend is accelerating and why patient-centered research is more than a trend and instead the future of health research and care delivery.

Looking Ahead

In the coming months and years, we look forward to ongoing opportunities to work with our stakeholders—patients, clinicians, insurers, the life sciences industry, policymakers, and others—to build on the progress we’ve made to date in identifying the questions and in funding the studies that matter most to them and will help them make better healthcare decisions.

In doing so, we’ll continue to place a premium on identifying the gaps in our knowledge about how to best prevent, diagnose, and treat some of the most challenging diseases and conditions patients face, always focused on the outcomes they care about most. And we’ll do so with the commitment we’ve had since our creation to be good stewards of the funds and public trust invested in us.

*Note: This blog post, which was originally published on April 26, was updated on May 24 to reflect the Board of Governors' approval of our latest round of studies.