10 years after inception, industry backs the federal research institute

After a contentious start a decade ago, the Patient-Centered Outcomes Research Institute was able to demonstrate how much perceptions have changed when Congress recently reauthorized its operations for another 10 years with bipartisan backing.

The formation of the institute, often called PCORI, was one of the most controversial elements of the Affordable Care Act because of concerns flourishing among Republicans that it would lead to rationing of care.

The institute funds comparative effectiveness research, which examines the benefits and risks of medical interventions to help patients and clinicians make better healthcare decisions. There were strong concerns among some that the research would cause insurers to deny coverage when factoring in cost differences between interventions. At one point, it wasn’t certain if PCORI, or something like it, would even make it into the ACA.

Comparative effectiveness research fell into the commentary about “death panels” among conservatives during the ACA debates. Former Alaska Gov. Sarah Palin coined the term to describe governmental entities and others that might have the power to decide if someone can get medical care.

More recently, though, descriptions of PCORI include words like “crucial” and “innovative,” sentiments that helped the institute to get reauthorized by Congress in December. 

During the reauthorization debate, the most heated discussions were over how long PCORI should be reauthorized for. Should it be 10 years or three? It ended up being reauthorized for the full 10 with funding direct federal appropriations similar to what it received under the ACA, which was $150 million annually. It also receives additional funding—about $255 million in fiscal 2018—from a mandated tax on insurers. The recent reauthorization removed funding from the Medicare Trust Fund and it will now be funneled through congressional appropriations but it totaled $115 million in 2018. Overall, total revenue of PCORI was about $507 million in 2018.

And concerns about care rationing also seemed to have dissipated considering the reauthorization changed the language of PCORI’s statute with explicit mentions about its responsibility for research to consider the economic burden of interventions on patients, including out-of-pocket costs. The language in the ACA was less explicit about how PCORI should factor in costs.

All of this signals to policy experts and researchers that PCORI and the research it supports is better understood than when the ACA passed a decade ago. By evaluating the value of medical interventions including certain drugs and devices, comparative effectiveness research is overwhelmingly viewed by researchers and patient advocacy groups as a necessary investment to improve patient care and lower rising healthcare costs.

“Comparative effectiveness research has entered the mainstream,” said Dr. Albert Wu, director of the Center for Health Services and Outcomes Research at Johns Hopkins Medicine who has conducted research funded by PCORI. “Comparative effectiveness research and PCORI ended up being perhaps the least controversial part of the ACA, which is gratifying but also maybe not so surprising because people are interested in what works better when making healthcare decisions. That is a bipartisan issue.”

PCORI’s recent reauthorization is being viewed by policy experts as an opportunity for the institute to show the impact comparative effectiveness research can have, which some argue hasn’t been so obvious in its first 10 years.

“I’ve been a little concerned by what I thought was the excessively cautious strategy that PCORI used in its early years, which is not hard to understand because of its almost near-death experience and the political pushback they were facing,” said Gail Wilensky, a former adviser to President George H.W. Bush who has expertise in comparative effectiveness research. “I think it’s time to be over it, especially now that they have gotten reauthorized.”

Experts say that PCORI’s research should play a key role in discussions about rising healthcare costs and how to change the trajectory. “The healthcare debate is all about costs and we aren’t thinking about what we get for those costs,” but PCORI’s research does, said Jennifer Graff, vice president of comparative effectiveness research at the National Pharmaceutical Council who has participated in engagement efforts by PCORI.

And with the reauthorization bill, which was led by a bipartisan group of senators, including much clearer requirements for PCORI’s research to consider how patients are economically affected as a result of healthcare interventions, there is hope that the institute can have a stronger influence on bending the cost curve.

Given all the controversy around comparative effectiveness research being used to ration care, PCORI “may have gingerly approached all discussions about costs,” said Dr. Anand Parekh, chief medical adviser at the Bipartisan Policy Center.

Medicare has always been able to use research funded by PCORI for coverage, but it just can’t be the only piece of information it uses. “The prohibitions are still there—what the research can and can’t be used for (regarding costs)—but this language will hopefully help (encourage) its use a little bit more,” Parekh said.

But Dr. Sharon Levine, founding board member of PCORI and board member of the Kaiser Permanente Bernard J. Tyson School of Medicine, disagrees with the criticism that PCORI’s influence hasn’t been strongly felt yet across the industry.

Since its inception, PCORI has funded about 750 research studies and other projects totaling $2.6 billion. More than a third of PCORI-funded studies are focused on common conditions such as stroke, heart failure, cancer and diabetes.

Levine highlighted the study PCORI funded on Type 2 diabetes as an example of the impact the institute can have. The study, conducted by researchers at the University of North Carolina at Chapel Hill and published in 2017, found that patients with non-insulin treated Type 2 diabetes who routinely self-monitored their glucose levels by using finger sticks didn’t have better hemoglobin a1c levels compared with patients who didn’t use the device.

“When you think about the out-of-pocket costs for those finger sticks for the large population of patients with Type 2 diabetes, that is huge,” Levine said.

Wilensky said similarly, PCORI is doing the hard part, which is funding complex, long and costly studies. It’s not particularly difficult for insurers to then take those results and make economic conclusions.

Indeed, a spokeswoman at America’s Health Insurance Plans said the lobbying group was pleased to see the reauthorization had more language about how PCORI should incorporate costs into research. Health plans are taxed to help fund PCORI, so it’s to their benefit to leverage the institute’s research.

The multimillion-dollar yearly investment both Medicare and private payers make is appropriate when you consider how much the U.S. spends on healthcare, Graff said. Spending $150 million is “not even a drop in the bucket” compared with the $3.6 trillion the U.S. spends on healthcare annually, she said.

PCORI did have a slow start as it used the first year to hire staff and establish research priorities and methods before it funded any studies, which opened up the leadership to criticism, Wu said.

But the tactic PCORI took in the beginning has paid off, he argues. Its methods are now replicated by others, including the National Institutes of Health, he said.

“If they had rushed more quickly or they hadn’t been thoughtful about their methods” it would have teed their study results up to criticism, Graff added. “It makes good political sense that PCORI took a more thoughtful and cautious approach.”

In terms of how PCORI spends its money, some have faulted the institute for how much goes toward efforts other than research. While about 80% of its budget is spent on research, the rest is used on administrative costs and other projects such as engaging stakeholders. Since PCORI was formed, spending on engagement has increased from $1.8 million in 2011 to $31.4 million in 2018.

“Keeping a constituency informed and involved is important but it’s not really what I would see as their primary mission,” Wilensky said. “You want them (stakeholders) to be with you, not fighting with you and that involves some outreach, but also PCORI should recognize that doing so distracts from what your real mission is, which is research.”

Levine said engagement efforts represent only 4% of PCORI’s total budget and it’s a critical aspect of its work. PCORI funds work groups made up of patients, employers, providers, health plans and others focused on how researchers should conduct studies for certain interventions or conditions. The feedback from the groups helps PCORI decide what research to fund. These opportunities are called Engagement Awards and in fiscal 2020 PCORI plans to give out about $21 million to interested groups.

“The Engagement Award program is intended to bring more patients, caregivers, clinicians, and other healthcare stakeholders into the research process,” said Jean Slutsky, chief engagement and dissemination officer at PCORI.

The work around engagement has been criticized, however, for its inclusion of participants with vested interests, Graff said. “Some stakeholders have worried that the questions that mattered most to physicians and patients would be discounted in favor of those questions that matter to groups with a financial stake in the game,” she said.

To address that, PCORI has created conflict-of-interest policies in which individuals with direct conflicts will recuse themselves from panels. Additionally, researchers must reach out and get advice from patients and providers on the methods of their study as part of the application process to get research funded by PCORI.

For researchers, the process is unique but fulfilling. “It has been one of the most worthwhile experiences I have worked in because we are engaging our stakeholders,” said Dr. Laura Young, associate professor of medicine at UNC who lead the PCORI-funded Type 2 diabetes study.

This tactic initially caused some concerns among researchers worried about how patients who don’t have expertise in research may impact their studies, Wu said. But because PCORI spent a lot of time figuring out how to involve patients appropriately and do it in a reliable way, worries have since dissipated, he said.

The influence of PCORI’s engagement efforts has spread across the industry, Wu added. It’s now much more common practice at health systems than a decade ago to involve patients in decisionmaking.

The focus on engagement helps with PCORI’s dissemination efforts because stakeholders have been involved from the beginning on the research projects and are therefore invested in knowing the results and spreading them, Levine said.

The ACA mandated PCORI to disseminate findings from its studies to encourage changes to practice patterns. PCORI is required to publish its research but the board knew that wouldn’t be enough to have impact, Levine said.

“We have understood from the beginning that investing in both dissemination efforts as well as investments in the science of implementation were going to be critical to fulfilling our commitment to having impact and demonstrating impact,” she said.

PCORI only began investing in dissemination in late 2016, which is when results from its research started rolling in. They plan to increase their investment in dissemination in the years ahead as more study findings are released, Slutsky said.

In 2016, PCORI spent roughly $145,000 on dissemination. In 2018, that figure was $7.2 million.

For PCORI, the returns on investment will be seen when findings from its research are common practice. For instance, Slutsky pointed to a large PCORI-funded study that found narrow-spectrum antibiotics are just as effective as broad spectrum antibiotics and using the former on children for relevant conditions could save $131 million annually.

Dissemination involves providing funding to researchers so they can spread their findings to pertinent stakeholders. Slutsky said studies are selected for dissemination based on the strength of the findings and the size of the study.

Others agree that dissemination should be a main focus area for PCORI going forward.

“The next 10 years needs to be about dissemination,” Parekh said. “That is sort of the return on investment—it’s both improvements in health outcomes but there is also the potential that some of these findings can reduce waste. That has to be critical.”

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