PCORI Established to Fund Patient-Centered Research

Tim Casey

Since the mid-1990s, Jonathan Tobin, PhD, and his colleagues have conducted research studies focused on increasing cancer screening among minority women. Three times, they received funding from the National Cancer Institute to assess patient navigators who provide assistance to people in need of help to access care.

During the course of their work, they noticed that many of the women also suffered from depression, which they believed decreased the screening rates. Last August, they submitted a grant proposal for a randomized controlled study to test whether integrating mental health services with cancer screening is better than providing cancer screening alone.

In early May, Dr. Tobin received an e-mail telling him the application had been approved by the Patient-Centered Outcomes Research Institute (PCORI), a government-authorized, private, nonprofit health research organization created in March 2010 as part of the Patient Protection and Affordable Care Act.

Dr. Tobin, president and chief executive officer of Clinical Directors Network, Inc. (CDN), will serve as the coprincipal investigator of the trial with Elisa Weiss, PhD, director of evaluation at the Leukemia and Lymphoma Society. CDN, a primary care, practice-based research network, will partner with community health centers in the Bronx, New York, and recruit approximately 700 women 50 to 64 years of age who have depression and are nonadherent with recommended cervical, breast, and/or colorectal cancer screenings. The first patients will enroll this fall and will be assigned a patient navigator to remind them to schedule appointments and deal with any barriers faced in accessing care. The $2 million grant began on July 1 and runs through June 30, 2016.

CDN was founded in 1985 to expand research engagement beyond academic institutions and bring in practicing clinicians as researchers. The organization has been successful at making clinicians working in community health centers partners in research, according to Dr. Tobin.

“PCORI takes it a step further and says that we need the voice of the patient at the table as well,” Dr. Tobin said in an interview with First Report Managed Care (FRMC). “Our partnership really represents not only a community/academic partnership, but a very highly engaged set of stakeholders in social service organizations, medical care organizations, academic institutions, practice-based research networks, and, most important, the patients.”

This year, PCORI will award an estimated $350 million to fund patient-centered comparative clinical effectiveness research projects. Funding will increase to approximately $500 million in 2014.

PCORI receives most of its funding through user fees from health plan sponsors and issuers of group and individual health insurance policies. The fee is $1 per plan participant for the first plan year ending after September 30, 2013, and $2 per plan participant in succeeding years plus any potential costs associated with medical inflation.

The federal government has authorized and committed to funding PCORI through 2019. In 2017 or later, Congress will decide if PCORI will continue to exist.

“I am sure they will be looking at a number of factors, not least of which is are we having an impact and are we, in fact, improving decision-making that is being made by patients,” PCORI deputy executive director Anne Beal, MD, said in an interview with FRMC.

Dr. Beal, a pediatrician and former head of Aetna’s charitable organization, was the second employee hired at PCORI after executive director Joe Selby, MD, a physician who joined following 13 years as the director of research at Kaiser Permanente. PCORI now has more than 90 employees and plans to expand in the next few years.

The organization is overseen by a 21-person board of governors that includes National Institutes of Health (NIH) director Francis S. Collins, MD, and Carolyn Clancy, MD, former director of the Agency for Healthcare Research and Quality (AHRQ). The board, which was appointed by the US Government Accountability Office, has members with experience as nurses, doctors, researchers, payers, business people, and pharmaceutical and biotechnology executives.

In May 2012, after seeking public comments and reviewing previous attempts to prioritize comparative effectiveness research, the board released PCORI’s 5 research priorities: (1) assessment of prevention, diagnosis, and treatment options; (2) improving healthcare systems; (3) communication and dissemination research; (4) addressing disparities; and (5) accelerating patient-centered outcomes research and methodological research.

PCORI’s main goal is funding research that provides patients with information they need to make informed healthcare decisions. As such, Dr. Beal serves as PCORI’s chief officer for engagement, a role in which she travels and speaks with patient groups, physician groups, health plans, and other stakeholders to identify research questions that are most relevant to them. In addition, patients can serve on advisory panels along with clinicians and healthcare professionals to help PCORI prioritize its research questions and offer input on proposals.

“Patients really want the kind of research that applies to their specific needs and conditions,” Dr. Beal said. “We wanted to do work that does not just sit on the shelf, but is utilized and put into practice. By engaging with these different groups and making sure we ask the right questions, and working with them to help get it out to their members and to the folks who rely on them for information, we are able to make sure that the research has an impact.”

With more treatment options available to them, from new drugs to surgical techniques to physical therapy, patients want to be more active partners in their care, according to Dr. Beal. She added that numerous conditions such as cancer, heart disease, and diabetes require patient self-management, which can sometimes be overwhelming for patients due to all of the choices on how to best treat their conditions.

Although Dr. Beal referred to the randomized controlled trial as the “gold standard” in comparative effectiveness research, she said PCORI realizes the studies may have limitations in terms of the patients enrolled and the outcomes measured. For example, most patients in randomized trials are tested for a single condition, but Dr. Beal understands that patients with cardiovascular disease have other conditions such as high cholesterol, diabetes, asthma, and/or arthritis. She also noted that when her 83-year-old aunt was diagnosed with cancer, she was primarily concerned with good quality of life and not as much with morbidity or mortality, which are common outcomes measured in the medical literature.

After spending the first few years of PCORI’s existence building infrastructure and developing a strategic plan and research priorities, the organization has recently started funding research. In the past 6 months, PCORI has announced numerous grant awards, revealed infrastructure investments in expanding the nation’s health information technology through the adoption of electronic health records, and sought proposals for patient-centered outcomes research projects that address disparities in controlling asthma among blacks and Hispanics/Latinos in the United States.

PCORI’s launch has been welcomed by Dr. Tobin and others interested in eliminating healthcare disparities, in which people have differences in clinical status or access to care based on their race, ethnicity, or socioeconomic status. PCORI will work closely with the NIH and AHRQ to disseminate and implement patient-centered outcomes research, some of which will address health equity.

Dr. Tobin and his staff at CDN have been busy working on grant proposals they plan to submit to PCORI in August. One of the initiatives is focused on community-acquired methicillin-resistant Staphylococcus aureus (MRSA) infections. In 2010, CDN and the Rockefeller University Center for Clinical and Translational Science received a small pilot grant to examine community-acquired MRSA infections in 6 primary care practices in New York City. They then obtained funding from the NIH to examine additional practices in New York, Chicago, and San Antonio. Based on the positive findings from that observational study, CDN and Rockefeller are designing a trial with community health partners to test whether home-based interventions can prevent transmission of the infections to family and household members and reduce the re-infection rates.

As part of the PCORI grant proposals, researchers must identify how they plan to publish the results in peer-reviewed medical journals as well as in local newspapers and trade publications and through presentations at scientific conferences. PCORI is interested in providing the general public with as much information as possible so people can make the best healthcare decisions, a policy that Dr. Tobin embraces.

“We have an obligation to the taxpayers who have supported the research,” Dr. Tobin said. “We have an obligation to the scientific and clinical community to share with them whether we found our approach to be effective or not effective. And then, finally, we have an obligation not only to bring the results back to the patients who were a part of this study, but to other people in the community who could benefit.”