PCORI at Age Four: What We've Achieved and Where We're Headed

It’s been four years since the US Government Accountability Office appointed PCORI’s founding Board of Governors. As we recognize this anniversary, we look back on a series of accomplishments that provide a solid platform for looking ahead to fulfilling the mandate given to us in the Patient Protection and Affordable Care Act: to conduct and disseminate comparative clinical effectiveness research (CER) that helps patients and those who care for them make informed health decisions.

As I told Board members when they met in mid-September, I’m particularly proud of these signature achievements:

A strong foundation for “research done differently.” Anchored in our National Priorities for Research and supported by our Methodology Standards, we’ve established innovative processes for engaging our stakeholder communities to identify the questions and studies most likely to produce useful information that can change health practices, behaviors, care, and outcomes important to patients. This approach, known as patient-centered outcomes research (PCOR), requires involving relevant stakeholders—patients, caregivers, clinicians, payers, and others—throughout the research process. They help participate in determining the research questions, reviewing and ranking research funding applications, partnering on funded research teams, making study results available, and championing their implementation.

A growing research portfolio based on that foundation. To date, we’ve approved $671 million in funding for 360 patient-centered CER projects (see infographic). Most of these projects are studying high-impact questions that matter to patients and those who care for them; others are further developing the methods used in PCOR.

PCORnet. This potentially transformative initiative aims to improve the nation’s ability to conduct PCOR. It is designed to enable practice-changing research by harnessing the vast data locked within health systems and clinical settings, as well as information and experiences reported by patients themselves. PCORnet is unprecedented in its scope and its commitment to seeing that patients help set the rules by which the network operates.

A clear and ambitious plan for future research. As our research portfolio evolves, it is becoming more focused on high-impact research topics that our stakeholders tell us are important to them and that address critical evidence gaps. We have a new emphasis on pragmatic studies, which are large projects usually conducted in routine clinical settings and intended to provide definitive information that can be directly adopted by healthcare providers.

We’re proud of our rapid growth from a start-up to a national research institute that is a leader in patient-centered CER as well as dedicated to influencing others to make clinical research more patient-centered. And we're pleased that we'll soon be able to share the findings of our initial rounds of funded projects to share.

What We’ve Done

Since 2012, when we began building our research portfolio, our multi-stakeholder review teams have helped us to select projects designed to answer important patient-centered questions, such as:

• A Kansas study comparing approaches to helping smokers with chronic obstructive pulmonary disease give up cigarettes.
• A Massachusetts study comparing ways to treat degenerative cervical spine disease, or neck arthritis, one of the most common indications for spinal surgery in the United States.
• A Washington State study comparing different surveillance imaging methods in women who have had breast cancer.

Analysis of our portfolio is revealing themes that might not have been identified as important research topics without the input of patients and other stakeholders. Examples, which cut across our priority areas and the conditions and populations studied, include self-care and self-management, palliative care, collaborative care, transitions in care, telemedicine, patient navigators, community health workers, cultural/language training, decision support and shared decision-making, and, in pediatric illness, parental support.

We’re seeing that our engagement requirement is also making a difference in how researchers conceive of their projects. For example, our reviewers found that a scientifically strong application was lacking a community-awareness component. In response to reviewer guidance, the researchers added city and county officials as partners and also decided to make the intervention under consideration available in Spanish and accessible on mobile devices. This greatly strengthened the proposal.

Where We’re Going

As part of our increased focus on specific priority areas highlighted by our healthcare stakeholders, we’ll expand the number of targeted funding opportunities we offer. We already have groups of studies on treating severe asthma in African Americans and Hispanics/Latinos and on preventing injuries due to falls by older adults. And our Board of Governors has just approved two projects studying comparative effectiveness of obesity treatment options; one focuses on rural primary care settings in the Midwest, and the other looks at clinics serving African-American and low-income patients in Louisiana.

In addition, the Board has approved a study of ways to improve transitions in care as patients return home from the hospital. In partnership with the Agency for Healthcare Research and Quality (AHRQ), we are funding a study across nine clinical centers on treatment options for uterine fibroids.  And we are partnering with the National Institutes of Health to support research on treatments for hypertension and look forward to funding other high-impact topics.

I’m particularly excited by the centerpiece of our ongoing move to more focused funding—our pragmatic studies initiative. This will represent the greatest allocation of our research funding in 2015 and beyond, providing the opportunity to support dozens of high-impact CER projects that could significantly enhance patient care. Topics for these funding announcements are identified through the work of our multi-stakeholder advisory panels, which help us prioritize research questions suggested by the public, research organizations, and the healthcare community. Our Board will consider the first of these proposals for approval in early 2015.

This shift from broad calls for relatively small projects to more-targeted calls for large studies is a natural progression that reflects the guidance we’ve received from the healthcare community, as well as our ever-sharpening focus on defining what makes an effective CER question. We know that studies asking the right questions are likely to yield findings that patients and those who care for them will put to use. I’ll be talking more about that in a future blog post.

This coming year will see us start to harness the potential of PCORnet, our collaboration of research networks based in both large health systems and patient groups. Our goal is to use the power of large sets of healthcare data, under policies developed with the help of patients, to enable more rapid and cost-effective clinical research. We’ve already identified the first clinical trial we plan to conduct using this still-developing network—a test to determine what dose of aspirin will best treat patients who have cardiovascular disease. We expect this study to get under way in the spring.

Keeping Our Commitment to Patients

The most valuable lesson I learned during more than three decades as a family physician is to always focus on doing what’s best for patients. It’s a lesson I took with me during my years as a researcher and now do my best to follow at PCORI. From our list of achievements in our still-short history and our plans for the future, I think we’re doing a good job of keeping that objective in mind.

Thanks to all of you who have played a part in our success. We look forward to working with you as we continue down the path of improving patient care and outcomes through patient-centered research.