Patients and their doctors face crucial choices every day: Surgically excise precancerous breast cells or watch carefully for growth. Try meditation for anxiety or go with prescription drugs. Stay in the hospital for tests after chest pain or head home and have testing later. To help with such decisions, and to rein in U.S. health care costs, Congress 9 years ago created an independent research institute that would enlist patients as partners in designing studies that compare the benefits of established medical treatments.
Now, after awarding nearly $2.6 billion for research, the nonprofit Patient-Centered Outcomes Research Institute (PCORI) in Washington, D.C., faces a turning point. The agency’s charter was set to expire on 30 September, and it is operating under a temporary extension until 20 December. Congress must decide whether to reauthorize the organization long term, and if it does, whether to fine-tune its mission.
Supporters say it’s obvious that PCORI should continue. With annual incoming funds averaging about $480 million since 2014, the operation has paid for more than 700 projects, more than half completed by now, that are already influencing health care. “We’re starting to see results that really matter,” says Christine Goertz, a health services researcher at Duke University in Durham, North Carolina, who chairs PCORI’s governing board.
In spite of complaints that PCORI was slow to launch large randomized trials that could sway medical practice and cut costs, disease advocacy and research groups back it. “They have picked up speed and set up more effective studies. What they’re doing now is what they should be doing,” says Ross McKinney, chief scientific officer for the Association of American Medical Colleges, based in Washington, D.C.
Lawmakers seem to agree—bipartisan bills in the House of Representatives and Senate would reauthorize PCORI for as long as 10 years. But with a crowded legislative calendar and impeachment taking lawmakers’ attention, it’s not clear when a bill will be passed.
PCORI’s birth took political compromises. Congress created it as part of then-President Barack Obama’s 2010 Affordable Care Act, funding it largely from a tax on health insurance plans that goes into a trust fund. Backers reasoned that “comparative effectiveness research” could help control ballooning medical costs. Conservatives worried, however, that PCORI’s work would result in health care rationing, so lawmakers barred the agency from making cost comparisons while mandating that its board include stakeholders representing industry, insurers, patients, and physicians. They also gave it a name that did not mention comparative research, but instead reflected a less controversial goal: putting the needs and interests of patients first.
Figuring out what “patient-centered research” meant took time. “Nobody really knew what it was or used [the term] in their own way. In fact, PCORI defined it,” says Ellen Sigal, chair of Friends of Cancer Research, a Washington, D.C.–based patient group. PCORI-funded researchers must try to include patients every step of the way, from enlisting them in study design to, at times, including them on the research team; patients also help PCORI review research proposals. The requirements “have been an adjustment” for many scientists, but worthwhile overall, says health services researcher Michael Fischer of the Harvard University–affiliated Brigham & Women’s Hospital in Boston, who has worked on PCORI projects.
PCORI says patients have suggested changes to studies, such as emphasizing the ability to live at home as a treatment outcome and changing how a study is explained to them. PCORI has inspired U.S. agencies, drug companies, and health care organizations to incorporate patient-centeredness into their work, says Marc Boutin, CEO of the National Health Council in Washington, D.C., which represents patient groups.
But developing methods for involving patients and setting up review panels was time consuming, and PCORI’s first contracts only went out in late 2012. Many early projects focused on patient education and engagement, such as evaluating “decision tools,” or brief information sheets, to help people and their caregivers choose between treatments. The institute also spent time and money to build PCORnet, a clinical research network that pools electronic health records for millions, which aids in comparisons of treatments and recruitment of patients for clinical trials.
PCORI eventually ramped up its support of comparative effectiveness research, which has consumed 73% of its total award commitments to date, the institute says. Published findings include that oral antibiotics are as helpful as intravenous ones for certain children with serious infections, and that type 2 diabetics who aren’t on insulin don’t need to test their blood sugar every day. Some results have found their way into practice guidelines, and PCORI is now funding efforts to get physicians and health systems to adopt its results.
There has been “substantial disappointment” in the slow pace, however, says bioethicist Ezekiel Emanuel of the University of Pennsylvania, who helped craft the PCORI authorization as an Obama official. By now, he adds, it should be able to “rattle off five or 10 things” that have had a major impact on medical practice. Emanuel contends that its patient engagement efforts went “way overboard.” He also faults the institute for shying away from large-scale drug comparisons for fear of “antagonizing” the drug companies. “They weren’t bold enough,” Emanuel says.
Health insurers, too, feel that PCORI “hasn’t really informed our decision-
making as of yet,” says Kate Berry, senior vice president for strategic partnerships for the America’s Health Insurance Plans, a lobbying group in Washington, D.C.
PCORI’s defenders say its critics have “an unrealistic understanding of how rapidly you could implement large scale trials. Then there’s the lag at the other end—how sluggish the adoption in health care is,” says PCORI interim Executive Director Josephine Briggs, a former National Institutes of Health (NIH) official who replaced its founding director Joe Selby last month. She also defends its decision not to fund more of the long-term drug comparison studies more typically done by NIH. Their cost “could absorb our budget and keep PCORI from doing more of the very practical stuff,” she says.
Although PCORI still has funds left to administer ongoing grants and make a few additional awards, it has no money to initiate new rounds of funding. A search for a permanent director is underway, but Briggs expects any candidate will want to wait to see whether reauthorization goes through.
Two bills approved by House panels would renew the institute for only three to seven more years and leave its operations largely unchanged. A bill introduced in the Senate would give PCORI another 10 years creates an advisory panel to identify high-impact research areas, requires more short-term projects, and allows the institute to consider treatment costs—all changes that Berry’s group and other insurers favor.
Although PCORI’s supporters expect a compromise bill to pass in the coming months, they are watching the process anxiously. After nearly a decade spent building a new patient-centered way to fund health outcomes research, “it would be such a pity if this were to dwindle away,” Fischer says.