Patient-Centered Outcomes Research: Early Evidence From A Burgeoning Field.

Abstract

Report From The Field Health AffairsVol. 35, No. 4: Patients’ & Consumers’ Use Of Evidence Patient-Centered Outcomes Research: Early Evidence From A Burgeoning FieldBara Vaida Affiliations Bara Vaida ( [email protected] ) is an independent journalist in Washington, D.C. PUBLISHED:April 2016Free Accesshttps://doi.org/10.1377/hlthaff.2016.0239AboutSectionsView PDFPermissions ShareShare onFacebookTwitterLinked InRedditEmail ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsDownload Exhibits TOPICSPatient engagementPharmaceuticalsAffordable Care ActSystems of careComparative effectivenessMedical researchOutcomes researchPatient careResearchersCosts and spendingOn a chilly, rain-soaked January morning, Kevin Paul, a fifty-nine-year-old deliveryman with a snowy white beard reminiscent of Santa Claus’s, waited in a hospital gown in St. Luke’s Mid America Heart Institute in Kansas City, Missouri. He was anxious. Early that morning, he had gone to his doctor’s office after feeling short of breath when lifting equipment at work.Paul’s doctor had sent him to St. Luke’s for a coronary angiography. For the test, a physician snakes a catheter through a groin or neck artery toward the heart, injects contrast dye, and looks for blockages. If one is found, several treatments may follow, including the insertion of a stent—a medical device that looks like a tube made of chicken mesh and that props open a narrowed artery.Katie Jaschke, a twenty-nine-year-old nurse with a warm demeanor and short blonde hair, stopped in Paul’s room before Paul was wheeled away for the test. She was there to talk about the procedure and help him answer a question.“Do you know the difference between stents?” she asked, handing him a piece of paper called a decision aid—a tool St. Luke’s is developing to help patients make decisions. Using graphics and text, Jaschke detailed the differences between two stents: One would require him to take a drug for a year, and the other would require him to take a drug for just a month. The conversation eased Paul’s anxiety.“It was just really great that they explained I had options instead of just saying, you have to do it this way,” he said, after recovering from receiving four stents. The money for the development of St. Luke’s decision aid was provided by the Patient-Centered Outcomes Research Institute (PCORI), a nonprofit created in 2010 by the Affordable Care Act (ACA). Through hundreds of research contracts, PCORI hopes to drive an evolution in health care, getting patients more engaged in their health care decisions and in shaping the process of medical research. According to Christine Stencel of PCORI, 1 the institute has a nine-year budget of about $3 billion and is also working to fill the yawning evidence gap in medicine. Too often screenings, tests, and treatments don’t provide definitive results to help patients and doctors make decisions. Evidence from medical studies is often based on averages, which means that some patients don’t respond at all during a study, but doctors don’t know which ones. Research frequently lacks demographic diversity or takes place in a controlled environment that doesn’t reflect the reality of daily life. Often doctors make decisions based on medical culture and their personal experience of what works, and patients don’t have enough information to decide for themselves. The gap in evidence has resulted in Americans’ wasting billions of dollars annually on unnecessary tests and treatments. The US health care system remains the worst performing—in terms of efficiency, equity, and outcomes—of the systems of the world’s wealthiest eleven nations, according to a Commonwealth Fund analysis of its own surveys and data collected by the Organization for Economic Cooperation and Development and the World Health Organization. 2“PCORI has long been needed in research,” says Lisa Simpson, president and CEO of AcademyHealth, a research advocacy organization.That is why many health care stakeholders, including consumers, patients, payers, physicians, policy makers, researchers, and hospitals, welcomed the creation of PCORI. The institute’s legislative mandate is to finance comparative effectiveness studies—research that compares one treatment for a condition against another to determine what works best for different individuals. Since its launch in September 2010, PCORI has spent $1.25 billion 3 on research projects, and it has another $1.3 billion to spend on additional research. 1 The institute has laid the foundation for the production of significant evidence and a process for delivering it to clinicians, health care systems, and patients. Its work has been promising, as the enthusiastic involvement of patients, researchers, and clinicians demonstrates. But at least one powerful stakeholder group—health insurers—has grown impatient and wants to see more evidence now. If insurers’ dissatisfaction persists, they may raise the volume of their complaints to get the attention of Congress, which must reauthorize the institute’s funding before it runs out in September 2019. If Congress doesn’t act, PCORI may find itself with no budget, and its work may not continue.Building A FrameworkIn the years leading up to the ACA, lawmakers debated the need for a research organization to provide evidence of effectiveness as health care costs escalated. Sen. Max Baucus (D-MT), then chair of the Senate Finance Committee and an architect of the ACA, became a proponent of comparative effectiveness research because it would provide a mechanism for enabling a shift in health care financing from fee-for-service to value-based care, thus rewarding health systems for patient outcomes instead of for the volume of tests and services they provide. But the idea of comparative effectiveness research stirred up intense fears of health care rationing. The anxiety stemmed from historical practices in other countries, such as the United Kingdom—where the National Institute for Health and Care Excellence (NICE) considers evidence of clinical effectiveness and cost and advises the government on which drugs and treatments should be covered. 4“There was a lot of debate on what PCORI should be and how it should be structured,” says Steven Pearson, founder and president of the Institute for Clinical and Economic Review, a Boston-based nonprofit that evaluates the value of tests, treatments, and health system approaches. “Comparative effectiveness doesn’t mean the same thing to people. Some felt [PCORI] was about addressing rising health care costs, and others felt it was about getting patients into the center [of research]. Like all new ventures, especially in the political realm, it had to be explained and sold to people.”In June 2009 Baucus acknowledged the need to dampen the controversy about comparative effectiveness and came up with a politically palatable name for PCORI that lives on today. “Senators on my committee on both sides of the [aisle] suggested that we stop using the name, ‘comparative effectiveness research,’” Baucus said at a Brookings Institution gathering to discuss health care reform. 5(pp12–13) “They suggested that we switch to something else that is a little less controversial. So we talked about this one day…and I said…, a name we could use is Patient-Centered Outcomes Research. We could call it P-COR. At least, it would reflect the intent of the research.” Baucus went on to describe the framework of PCORI. “Patients must be actively involved in studying the research priorities and in designing the research study,” he said. 5(pp13–14) “Patients must be at the center of the questions about medical care that we want answered. Practicing physicians need to be at the table[, too]. They know what questions to ask, and they are key to making research meaningful for decisions that we make with patients.” He also said that the institute needed to be independent of the government so that it wouldn’t be subject to the vagaries of the annual budget process. And all of its proceedings had to be public to assuage fears that PCORI would be an entity that rationed health care services.Indeed, when the language authorizing PCORI was drafted as part of the ACA, the institute was funded through a trust fund separate from annual appropriations, with money from the federal government and a tax on health insurers. The legislation required PCORI’s proceedings to be open to the public and made clear that results would neither consider cost nor mandate reimbursement for any public or private payer.A Cultural ShiftSeven years after Baucus’s remarks at Brookings, PCORI has fulfilled many of his goals. According to Stencel, the institute had funded 476 research and infrastructure projects as of January 2016. Patients have been elevated to the level of partners with the academics and physicians leading the research, which has resulted in studies about questions of value to patients.PCORI has also funded the development of strategies for helping patients and doctors communicate better with each other and for helping patients make decisions for themselves. The approach has had a ripple effect on health systems, drug companies, federal agencies, and universities. All of them are looking for new ways to engage with patients.“By virtue of existing, PCORI has created a cultural awareness [of patient engagement] in these other sectors, and that is driving a paradigm shift in the health ecosystem,” says Marc Boutin, CEO of the National Health Council, a patient advocacy organization. “Researchers are helping families and caregivers identify health goals…and clinical outcomes that matter to them, drug companies over time will produce products that are more useful to consumers, and for the payers, there is [already] evidence that with patient engagement, adherence goes up and health care costs go down.”PCORI is the third piece of the nation’s research foundation. The National Institutes of Health (NIH) funds the discovery of new treatments, and the Agency for Healthcare Research and Quality (AHRQ) funds studies of improving health systems and care delivery. PCORI provides the evidence on “what is the most effective treatment for people who are living in the real world and are not an average person in a clinical trial,” says AcademyHealth’s Simpson. Later this year PCORI expects to publish comparative effectiveness studies on controversial questions that affect thousands of Americans, such as the following: What are among the most helpful drug treatments for stroke patients—antidepressants or cholesterol-lowering drugs? 6 What is the best nonsurgical treatment for lumbar spinal stenosis (a narrowing of the spine that can cause debilitating back pain)—an epidural injection or rehabilitation exercises? 7 Dozens more studies in future years will look at managing treatments for conditions from diabetes 8 to uterine fibroids. 9 But the work that PCORI has been doing may have been done too slowly and too quietly. The results from some of its largest clinical studies—those that could have the biggest impact on the health care system—won’t come until 2019 or 2020, after PCORI would need to be reauthorized. 10,11 “I worry that in their worry to be so patient focused…they spent a lot of time garnering and maintaining support among patient groups” but took a while before getting to large-scale clinical effectiveness studies, says Gail Wilensky, a health economist and senior fellow at Project HOPE, which publishes Health Affairs . Wilensky, who directed the Medicare and Medicaid programs from 1990 to 1992, was an early champion of the creation of PCORI. “They are going to be in a period soon where [Congress] will be involved, and it would behoove them to make clear what contribution PCORI makes,” she says. Joe Selby, PCORI’s executive director, has acknowledged such concerns and maintains that PCORI will stay focused on determining which research projects deserve its remaining $1.3 billion.“The main thing that we have to do is keep doing what we are doing now,” says Selby, a tall, avuncular family doctor from Fulton, Missouri. “Funding important research, mak[ing] sure the research is done well, gets published, and that we disseminate it.”Laying The Foundation The political turmoil about comparative effectiveness and the ACA itself led PCORI’s twenty-one-member Board of Governors to proceed carefully. As required by the ACA, PCORI’s board includes three patient and health care consumer representatives and four practicing physicians, including one surgeon. The other members are a nurse; a provider of integrated health care; a hospital leader; three payers, two of whom are health insurers and one of whom is a self-insured employer; one leader each from the drug, medical device, and diagnostic industries; a quality improvement researcher; and four representatives from state and government agencies, including the directors or their designees of the NIH and ARHQ. 12 To fulfill the ACA’s requirement of transparency, transcripts from PCORI’s board proceedings are recorded and posted on its website. The comptroller general of the United States appoints all members of the board except the two from the NIH and AHRQ. Those appointments weren’t made until September 2010, and the institute didn’t have an executive director until July 2011—a year after the ACA became law. 13 The board members methodically debated their mandate and the definition and design of patient-centered research, says Sarah Kuehl Egge, a former Senate Budget Committee aide who worked on the language in the ACA that authorized PCORI. “I think that it took a lot longer than anyone anticipated,” says Egge, who is now a senior manager at the consulting firm Washington Council Ernst & Young. Some health industry professionals such as Mark Helfand—a professor of medicine, medical informatics, and clinical epidemiology at Oregon Health and Science University—initially wanted PCORI to begin by funding a few of the hundred comparative effectiveness priorities on a list that Congress had commissioned from the Institute of Medicine (IOM) in 2009. 14 The list had been created with extensive input from the public and included studies where there is no clinical clarity for the treatment of chronic diseases such as atrial fibrillation (an abnormal heartbeat) and ductal carcinoma in situ (a noninvasive form of breast cancer). “I was an advocate early on for [PCORI] to pick a few of the really high-stakes research questions and just get going on them,” says Helfand, a member of PCORI’s Methodology Committee. “I was disappointed, at the time, that they didn’t do that right away.”Instead, PCORI spent its initial years commissioning studies on methods used in patient-centered outcomes research and projects for improving communication between patients and doctors, such as the decision aid being developed at St. Luke’s. PCORI funded research to collect data on how to engage patients and other health care stakeholders. It invited members of the public to weigh in on what they thought the definition of PCORI and the research priorities should be. PCORI built a priorities and review process and decided that the research it funded should fall into one or more of the following five broad areas: assessment of prevention, diagnosis, and treatment options; improving health care systems; communication and dissemination research; addressing disparities; and accelerating patient-centered and methodological outcomes research. 15 Advisory committees for each area were created to help PCORI prioritize research and reach consensus on what should be funded. To ensure that it could clearly demonstrate to Congress and health care stakeholders that it was meeting its legislative mandate, PCORI created a multistep merit review process and a committee for choosing the research to fund and for monitoring the progress of research already under way. The NIH and AHRQ were consulted to avoid duplicating research that was already being conducted. PCORI recruited patients, health care experts, and other stakeholders to join the advisory committees and participate in the merit review process. In addition, it instituted a peer review process to assess study findings. The whole process was modeled after those of the NIH and AHRQ, but PCORI was the first to include patients. 16(p11)PCORI also had to develop a strategy for disseminating its results so that they would have an impact on care. Clinicians are notoriously slow to adopt new ways of practicing medicine, even if new evidence is presented to demonstrate that there is a better way.“A lot of decision making in medicine is based on tradition and culture,” says Ron Keren, a pediatric hospitalist at Children’s Hospital of Philadelphia who has been a recipient of PCORI funding. “It’s ‘this is what we have done, and it’s worked pretty well, so we aren’t going to change it.’” The ACA directed PCORI to partner with AHRQ and disseminate research findings within ninety days after a study is completed. 16(p5,p189) Toward that end, PCORI invested in research to develop learning collaboratives to assist clinicians with adopting patient-centered research in practice. The institute alerted medical societies to its work; created training programs to teach researchers how to incorporate patient partners into their studies; and set up a PCORI “ambassador” program to engage community, patient, and clinician organizations in the institute’s work. “We were busy setting up the apparatus for [research],” Selby says. “For getting, in a fair way, input from all sources.” In 2013 PCORI announced that it would create a national data network for research, called PCORnet. 13,17 The idea, which had been championed by the IOM, 18 was to link large health systems and patient groups through a common data model that uses standardized electronic health records (EHRs). The records contain detailed patient information about medical history, outcomes, laboratory results, age, sex, and race or ethnicity. Each organization would maintain control of its own EHRs to maintain privacy and security, but all would collaborate to help one another enroll patients in studies on various topics as well as obtain their most up-to-date medical information. Eventually, Selby says, the network could work closely with the Food and Drug Administration’s (FDA’s), Sentinel Initiative, which is a national electronic network that tracks the safety of drugs and devices after they have reached the market.“PCORnet is like a moon shot initiative,” says Sean Tunis, a member of one of PCORI’s clinical trials advisory panels and president and CEO of the Center for Medical Technology Policy, an organization that focuses on improving the quality of patient care. In mid-2013 PCORI also announced a process of funding clinical effectiveness studies that were large scale and pragmatic. As of January 2016 it had funded nineteen projects that aim to produce results derived from everyday health care, instead of from controlled and specialized research settings. Among the studies is a head-to-head comparison of two drugs to treat chronic obstructive pulmonary disease. Another will look at whether proton or photon radiation works better for treating nonmetastatic breast cancer. 10Of the $1.25 billion that PCORI has spent on research projects, 73 percent went to comparative effectiveness research studies, 19 percent to building PCORnet, and 8 percent to methodology research projects to ensure the integrity of PCORI’s work, according to Stencel.Looking back, Helfand now says that he thinks PCORI made the right choice in focusing on building a foundation and creating PCORnet. “They were brilliant really, …to say, let’s get the infrastructure right,” he says. “It was courageous because they had to take a longer-term view.”Skeptics And AdvocatesIn early October 2015, about a thousand people with an interest in health care research gathered in the cavernous ballroom of a Marriott hotel in Crystal City, Virginia, to attend PCORI’s first annual meeting. Selby addressed the pace of the institute’s work up front. “The board has been saying to us, since 2012, that you should have an annual meeting,” Selby said in his opening statement to the audience. “The staff said: ‘We will. When we have findings.’ Well, we have findings, and this next day and a half are a celebration of findings from PCORI-funded studies. Here is your first annual meeting, dear board members.” 19 Among the findings highlighted during the meeting were results from a few of PCORI’s first comparative effectiveness and patient engagement studies. One from 2012 found that treating osteomyelitis (a severe bone infection) in children with oral antibiotics worked just as well as the standard practice of giving the children antibiotics for weeks through a catheter in the arm. 20 Another was a study of stroke patients with atrial fibrillation that was funded in 2013. It found that prescribing the blood thinner warfarin for the patients upon discharge from the hospital resulted in lower mortality, fewer adverse heart events, fewer strokes, and more quality time at home over two years, compared to not using warfarin. 21 Most importantly, the researchers talked about how patients’ involvement in their research changed the course of their studies and the potential positive impact of increased patient engagement on the health system. Adnan Chhatriwalla, medical director of structural intervention at St. Luke’s Mid America Heart Institute, presented his work on the decision aid (funded in 2013) 22 that was used to help Kevin Paul with his stent procedure. His research showed that there was “a disconnect between patient values and what actual treatments were,” he said at an October 7 panel on results of PCORI-supported research. Given a choice, some patient might choose a less expensive stent, he said. Indeed, a 2014 Cochrane Collaboration review of 115 studies on decision aids showed that patients using them felt more informed and clearer about what was important to them. 23 The use of the aids also reduced the number of people choosing an invasive and expensive surgical treatment option. PCORI’s leaders emphasized the promise of PCORnet and introduced its first studies. The network includes thirteen large health systems (such as the Kaiser Foundation Research Institute, Mayo Clinic, and Weill Cornell Medical College) and twenty patient and caregiver networks run by patient organizations (such as the Genetic Alliance and the Immune Deficiency Foundation). 24 The first clinical trial will be a three-year $18.6 million study of as many as 20,000 patients to assess the appropriate dosage of aspirin for patients with heart disease, which is the leading killer of Americans. 25 Two other studies will examine approaches for treating obesity. 26,27Yet one health care leader expressed skepticism.“I feel like PCORI from a payer point of view was slow to start, slow to get focused,” says Lewis Sandy, senior vice president for clinical advancement at UnitedHealth Group, an insurer. “People in my organization that are knowledgeable about PCORI said, ‘What are the top three things that PCORI can show demonstrating…that it ought to continue?’ I’m sure they’re there. I don’t know what they are.” That matters, because when the ACA was debated, insurers agreed to pay a tax to help fund PCORI. Payers routinely look at evidence on medical treatments to consider which ones provide the most value for the cost. According to Stencel, as of fiscal year 2014, through a trust fund created by the ACA, insurers had contributed $299 million to PCORI’s budget, while the federal government had provided about $537 million. 28–31“The question will be, what is the ultimate goal and the value of PCORI moving forward?” says Clare Krusing, communications director for America’s Health Insurance Plans, the chief lobbying organization for the nation’s health insurers. “There is a lot of funding going into [PCORI], and we aren’t seeing the level of research and results yet to inform a lot of the coverage decisions that plans are weighing at this point.”A New LandscapeSelby has three answers for Sandy, Krusing, and other skeptics. He says that PCORI has already had an impact on researchers’ collaboration with patients; PCORI’s formation of PCORnet has provided a long-lasting resource for faster collaborative research; and PCORI has built a growing portfolio of large, meaningful studies that no other organization would likely have funded. As evidence of PCORI’s influence, Selby points to a number of organizations that now engage patients in research. Geisinger Health System, a physician-owned hospital group, revamped its research process to include patients after it received PCORI funding. 32 The University of Arkansas 33 and the University of Pittsburgh 34 established patient-centered research centers. In 2013 the FDA launched efforts to add input from patients to the drug and medical device regulatory process. 35 The drug industry’s advocacy organization, Pharmaceutical Research and Manufacturers of America (PhRMA), has asked the FDA to develop regulations to guide meaningful patient engagement in clinical trials and drug labeling. 36The current congressional debate over the 21st Century Cures Act—legislation to accelerate US biomedical research that is now pending in the Senate—calls for including patient representatives on almost every advisory board, body, council, or panel to be created by the act. Drug companies such as Pfizer, 37 Sanofi, 38 and UCB 39 have created offices of patient engagement. Furthermore, there are now thousands of patients who are passionate about patient-centered outcomes research. John Walsh, cofounder and president of the COPD Foundation—an advocacy group in Miami, Florida, for the roughly fifteen million Americans living with chronic lung diseases—describes PCORI as “one of the most productive assets created from the ACA.” And PCORnet has been created in such a way that it could endure even if PCORI doesn’t. On its website, PCORI says it anticipates that the NIH, the FDA, and private industry will step in with resources for PCORnet in the future. 17“There is a lot more of this research that needs to be done than PCORI could ever fund,” Selby says. “So we wanted to set up an infrastructure that could even outlive PCORI possibly, but it would be a legacy that would facilitate this kind of research regardless of who the funder is.” Results from at least twenty-four comparative effectiveness studies should be available by the end of 2017—just before Congress begins to seriously consider PCORI’s reauthorization. 11“The results will speak for themselves,” says Debra Barksdale, a nurse practitioner, member of PCORI’s Board of Governors, and a professor and associate dean of academic programs at Virginia Commonwealth University’s School of Nursing. “I think PCORI is at a turning point, in that it has really gotten into its stride.”NOTES1 Christine Stencel, communications director, PCORI, personal communication, January 20, 2016. 2 Davis K , Stremikis K , Squires D , Schoen C . Mirror, mirror on the wall, 2014 update: how the U.S. health care system compares internationally [Internet]. New York (NY) : Commonwealth Fund ; 2014 Jun [cited 2016 Feb 25 ]. Available from: http://www.commonwealthfund.org/publications/fund-reports/2014/jun/mirror-mirror Google Scholar 3 Christine Stencel, communications director, PCORI, personal communication, January 26, 2016. 4 Vize R . Rationing care is a fact of life for the NHS . Guardian [serial on the Internet]. 2015 Apr 24 [cited 2016 Feb 25 ]. Available from: http://www.theguardian.com/healthcare-network/2015/apr/24/rationing-care-fact-of-life-nhs Google Scholar 5 Baucus M . Testimony in: Brookings Institution. Implementing comparative effectiveness research: priorities, methods, and impact [Internet]. Washington (DC) : Brookings Institution ; 2009 Jun 9 [cited 2016 Feb 25 ]. p. 11 – 19 . Available from: http://www.brookings.edu/~/media/events/2009/6/09-health-care-cer/20090609_health_care_cer.pdf Google Scholar 6 Hernandez AF . Patient-Centered Research into Outcomes Stroke Patients Prefer and Effectiveness Research (PROSPER) [Internet]. Washington (DC) : Patient-Centered Outcomes Research Institute ; [cited 2016 Mar 4 ]. Available from: http://www.pcori.org/research-results/2013/patient-centered-research-outcomes-stroke-patients-prefer-and-effectiveness