Obamacare research institute plans to spend $3.5 billion, but critics question its worth

Introduction

On the 9^th floor of a glassy high rise in downtown Washington, partitions are coming down to make more room for workers handing out billions of dollars in Obamacare-funded research awards.

Business has been brisk at the Patient-Centered Outcomes Research Institute or, PCORI, as it is known. The institute was created by Congress under the Affordable Care Act to figure out what medical treatments work best — measures largely AWOL from the nation’s health care delivery system.

Since 2012, PCORI has committed just over $1 billion to 591 “comparative effectiveness” contracts to find some answers, with much more spending to come. Money has thus far gone to researchers and medical schools, advocacy groups and even the insurance industry’s lobbying group, which snagged $500,000.

Institute officials say they are reshaping medical research by stressing “patient centered” projects that offer practical guidance to people living with chronic diseases. They cite a $14 million study to settle the debate over how much aspirin people should take daily to help ward off heart disease, or the $30 million project to reduce serious, even deadly, injuries from falls in the elderly.

But like all matters rooted in Obamacare, there are sharp disagreements — both political and scientific — over the core mission of the independent institute. PCORI expects to spend $3.5 billion by the end of the decade. Then it expires.

On both the right and the left, there’s simmering doubt about whether the unusual nonprofit can live up to expectations, or even what those expectations should reasonably be. Others argue these sorts of decisions should have been made prior to committing up to $3.5 billion.

“PCORI seems to have become almost invisible. Maybe they think that’s the best way to stay under the political radar screen,” said Gail Wilensky, a former Medicare chief under President George H.W. Bush. The institute has yet to “offer much value,” she said.

Some Republicans are viscerally hostile. They want to kill off, or at the very least, hamstring the institute, fearing it will lead to rationing of medical care by interfering with medical decision-making. The House Appropriations Committee in late June voted to cut PCORI’s funding by $100 million — dubbing it wasteful spending. Earlier this year in the Senate, Kansas Republican Pat Roberts filed a bill to prevent Medicare from using PCORI results to “deny or delay coverage of an item or service.”

“Americans do not want the federal government limiting their treatment options and deciding what is best for them,” Roberts said at the time.

Liberals aren’t singularly thrilled, either. They fault the institute for not evaluating enough drugs and medical devices head-to-head to see which offer the best results — and thus the biggest bang for the health-care buck.

“If it doesn’t prove its worth soon there will be increasing calls for getting rid of it, or reducing its funding,” said Topher Spiro, vice president for health policy at the Center for American Progress, a liberal think tank. “That concerns us.”

PCORI executive director Joe V. Selby, a family physician and researcher, accepts some of the flak. But he argues that Congress directed the institute to explore research topics patients want and need — not to issue edicts on which treatments offer the best bargain.

“We are not in any way a cost effectiveness shop,” Selby said in an interview. “That is not our job and there is a certain wisdom in saying that we shouldn’t get involved.”

Clearly, PCORI’s stated mission means different things to different people. Five years after setting up shop, PCORI is sponsoring many projects that offer great hope to doctors, patients and their advocates. But the institute also has spent hundreds of millions of dollars on activities only tangentially related to discovering which medical treatments are the most successful, a review by the Center for Public Integrity has found.

Among the findings:

• PCORI has spent only about 28 percent of its contracting budget on projects that assess how best to prevent, diagnose or treat diseases. Selby says more such projects are coming, including head-to-head evaluations of drugs and medical and surgical treatments. He cited a hepatitis C study coming in the fall that will pit the highly costly drug Sovaldi against other options. Much of PCORI’s other spending, though, concerns how to accelerate its research or improve health care systems.
• More than $70 million in PCORI awards cover projects intended to improve methods for conducting research, or to pay for contracts that are essentially public relations gestures to build support and good will in the medical community.

Those figures include nearly $10 million in “engagement” awards and “meeting and conference” subsidies for medical societies and other groups. America’s Health Insurance Plans, the industry trade group, got two engagement awards this year that total $500,000. One physicians’ organization got $250,000 to find out what its members think about PCORI and its work. Institute officials said these types of awards are necessary to get their work noticed and amount to a mere “rounding error” in terms of total money spent.

• PCORI has allocated an additional $61 million to help spread the reach and impact of its activities. But some projects, at least in summaries posted on the institute’s website, are so freighted with academic and scientific language that it’s hard to imagine how they could attract a wide audience. One project, for instance, looks at how doctors can create a “Zone of Openness” with patients.
• PCORI faces steep challenges in making its mark on the everyday practice of medicine. The Affordable Care Act states that PCORI’s findings are “not to be construed as mandates for practice guidelines, coverage recommendations, payment, or policy recommendations.”

Some critics say that language handcuffs the institute by limiting how its findings can be put to practical use. Others argue that PCORI has plenty of authority to push for more efficient health care spending, but has been too timid in wielding that power.

The health reform law gives PCORI “more flexibility than it is willing to use,” said Nicholas Bagley, who teaches at the University of Michigan Law School.

Sense of Purpose

In the world of federally-funded medical research, the mammoth National Institutes of Health in Bethesda, Md., is sometimes viewed as the “discovery” agency, where scientists study the origins of disease and search for breakthroughs and cures. There’s also the much smaller federal Agency for Healthcare Research and Quality, with a budget of about $440 million and a mission to “make health care safer, higher quality, more accessible, equitable and affordable.”

PCORI is a third entrant with a different mission, though it can and does also collaborate with other federal agencies. Congress created PCORI in 2010 as an independent institute that specializes in comparative effectiveness research. Under the ACA, the institute receives a mix of Medicare money, general revenue and funding from a tax on health plans.

PCORI is run by a board whose 21 members are picked by the Government Accountability Office, the watchdog arm of Congress, and it has 191 full-time staff. That’s up from 153 in September 2014.

Oversight is minimal. A GAO audit in March found little to fault, though auditors noted they had heard concerns that PCORI’s research priorities were “too broad and lack specificity.” GAO also noted that the institute won’t undergo an outside critique of its performance until 2020, after it has run through that $3.5 billion.

Common Sense

At least in theory, comparative effectiveness is pretty hard to fault. Common sense dictates that doctors need to know which drugs, medical devices and other treatments work best. And it makes little sense for anyone to pay for health care services that are shown conclusively to be ineffective.

But that’s not how things work. New drugs, for instance, come to market based on whether they are “safe and effective,” not if they are clearly superior to the competition. While many people might assume that a new medicine or device that costs much more than what’s already available must be better, there’s no such guarantee. In fact, more than half of medical treatments lack clear evidence of their effectiveness, according to the Institute of Medicine.

As such, doctors often can’t find persuasive evidence to advise them how best to get sick patients well. But while many medical groups strongly back research to find these answers, getting their members to embrace recommended changes isn’t always easy or quick to occur. Some doctors may be slow to pick up on the most current medical information, while others may resent suddenly being told how they should alter their practice.

Dave deBronkart, a kidney cancer survivor, recalled talking to a doctor who derided “cookbook” medicine. “One doctor told me, ‘I want autonomy to practice as I see fit,’” deBronkart said. Like many other advocates, he favors a much greater role for patients in their own care.

Manufacturers of drugs, medical devices and other equipment also have a big stake in comparative research. Some companies have noted a worldwide move to restrict payments for health care services, and certain types of drugs, that can’t clearly demonstrate they are worth the price.

Drafters of the ACA tried to take note of all these competing interests and needs. Despite keen interest in using comparative effectiveness research to cut costs, they yielded to fears that patients could be denied some treatments. As a result, the law appears to restrict use of research findings for cutting costs at the same time that it allows PCORI to consider “the effect on national expenditures associated with a health care treatment” in setting its research priorities.

Patients First

Given its mandate, it’s perhaps no surprise that PCORI chose many projects that were seemingly worthwhile, but also unlikely to threaten any powerful health care factions.

Since 2012, PCORI has let $389 million in contracts for accelerating its research agenda or “improving healthcare systems.”

For instance, the $30 million study anchored by medical schools at Harvard, Yale and UCLA, hopes to reduce falls in the elderly.

Falls “represent grievous events for older persons and a major public health problem,” according to the study.

Albert Wu, a health policy professor at Johns Hopkins University, who also is involved in the study, said researchers are looking at whether a “specially trained nurse” can work with the elderly and their relatives to find ways to cut down on these injuries.

“This is a topic that concerns every American who is over 65,” said Wu. “We all live in deathly fear an elderly relative will fall and break a hip. It causes terrible worry and distress and a substantial portion of those people die.”

Though PCORI director Selby wants little to do with health care financing controversies, the falls study appears to be an exception.

“Putting a nurse in every (doctor’s) office doesn’t come cheaply,” he said. “Who pays for that?” Selby said, confirming unassailable health benefits of hiring the nurses “would be a big step toward getting coverage for that.”

The $14 million aspirin study also has widespread health ramifications because if people take too high a dose they can suffer internal bleeding that may outweigh any heart benefits.

Researchers at Duke University said that every year, 720,000 Americans have a heart attack, and nearly 380,000 die of coronary artery disease. They said that “increasing the use of an inexpensive yet effective therapy, such as aspirin… will save thousands of lives globally.”

“We know that aspirin can be beneficial in preventing heart attacks but surprisingly we haven’t known the appropriate dose,” said Ann Bonham, chief scientific officer for the Association of American Medical Colleges.

“That’s an important piece of information…a gap that may not be recognized by a lot of people.”

PCORI has also let 18 contracts worth $44 million for research about rare diseases. Among them is $2.6 million awarded to the Cincinnati Children’s Hospital Medical Center to study which type of diet to give to children with a condition called “eosinophilic esophagitis.”

Overall, PCORI can point to $279 million in contracts for the “assessment of prevention, diagnosis and treatment options.” Still, that’s less than a third of total spending and less than one-fifth of the total number of contracts issued thus far.

Engaged

The Center for Public Integrity review found that tens of millions of dollars in other contracts are for studying research methods, or are looking into how to get findings noticed, or are difficult to decipher.

PCORI has directed $64.5 million toward 66 contracts whose purpose is to improve methods for conducting research. In June 2012, PCORI funded more than $30 million in 50 “pilot projects” mostly at universities and medical schools. One was a boot camp at the University of Colorado Denver for $675,000 over 30 months.

The authors said they would “activate personal relationships to bring together community members, clinical practices, patients, providers, and researchers to identify the important health issues they each face.” After several meetings, the group would “focus these topics into a priority list for further work.” The goal was to translate medical terms to make them easier for patients to grasp.

Some projects toss around esoteric phrases such as “how to capture stakeholder inputs” or “quality metrics to inform integrated care” that aren’t likely to be clear to the average reader.

Consider the $674,452 project funded at the Palo Alto Medical Foundation Research Institute with the catchy title: “Creating a Zone of Openness to Increase Patient-Centered Care.” The 2012 project explored how medical professionals can foster a climate where patients don’t fear being labeled as difficult “for asserting themselves in clinical decision making.” PCORI officials said the research produced important insights, though they concede they need to make their work sound more compelling.

Jean R. Slutsky, PCORI’s chief engagement and dissemination officer, agreed that heavy use of jargon can be a turn off, especially for patients the institute is trying to reach.

“We are in the process of putting in lay language to communicate with people who are not scientists,” she said. “We wish we could change this.”

The 36 “engagement awards” to health care organizations, universities and groups cost more than $8 million. That includes $500,000 to AHIP, the insurance industry trade group, to “build and maintain support from health plan leaders” and to “identify important gaps in availability of health insurance administrative data,” according to summaries of the contracts. AHIP spokeswoman Clare Krusing said sharing health plan data is “complex” and “requires a significant amount of review and expertise from the industry.”

PCORI also has provided “meeting and conference support” to medical organizations that totaled more than $1.8 million in 17 grants.

The Society for Academic Emergency Medicine, for instance, received $50,000 in 2015 to develop and publish “a consensus research agenda.” The year before, the Society of General Internal Medicine, whose 3,000 physician members teach at medical schools, received a $249,960 grant for a two-year program “to help us develop a better understanding of the attitudes and knowledge of our membership… and how they may best be engaged to participate.”

PCORI director Selby said these awards are necessary to get through to busy professionals who “may not answer our phone calls.” Selby said: “Our job is to build relationships and to get on their radar, adding “This helps us get their attention.”

Patient priorities

Though it may be largely unknown to the public, PCORI has won support from a wide range of parties.

Tony Coelho, a former Democratic congressman from California who chairs the Partnership to Improve Patient Care, said the institute takes pains to “get first-hand views on what questions really matter to patients.”

Coelho’s group, a coalition of patient advocates, drug manufacturers and medical groups, applauds PCORI for conducting research “in a manner that is patient-centered and ultimately useful at the point of health care decision making.”

Other experts said that putting patients first is itself a worthy research legacy.

PCORI “is filling a really important void,” said Justin W. Timbie, a Rand Corporation policy researcher. “This is really practical research to help people make decisions (about their health). I think that’s a lot of bang for the buck.”

This story was co-published with NPR.