How do you know what treatment is right for you . . . or your patients?

Much of medical care relies on what doctors have done before. That’s why comparative effectiveness research is vital to patient care — and why PCORI has been critical in changing the way physicians practice medicine. So why is its funding in jeopardy?
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For years, doctors have prescribed a daily dose of aspirin to patients with cardiovascular disease who have had a heart attack. But some doctors prescribe an 81 mg dose, while others prescribe 325 mg. The reason for the discrepancy? There’s no clear evidence about what dosage amount is needed to reduce the risk of a heart attack or stroke while minimizing harmful side effects such as internal bleeding.

Now scientists are getting closer to the answer through the Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-Term Effectiveness (ADAPTABLE) study. Nearly 14,000 patients have enrolled in the three-year clinical trial comparing two common dosages of the anti-inflammatory drug, which could mean more competent care for people with heart disease.  

ADAPTABLE is among the hundreds of research-related projects made possible through the Patient-Centered Outcomes Research Institute, more commonly known as PCORI. As a significant supporter of comparative effectiveness research (CER), PCORI fills a gap in clinical research with its spotlight on research that evaluates two or more health care interventions and strategies while also integrating patient perspectives throughout the process.

“Very often the information that the clinician is using to make these recommendations is not really based on a complete evidence, especially on how different treatment options compare,” says Diane Bild, MD, acting chief science officer at PCORI. 

While organizations such as the National Institutes of Health are renowned for their research capabilities, PCORI is unique in its focus on CER, which can be costly and time-consuming. The organization aims to speed and scale up this research, prioritizing high-cost, high-impact conditions such as asthma, diabetes, and kidney disease. 

“We feel passionate about our mission and we feel that we are supporting a research area that has not had a focal point before,” says Bild.

But that could come to a halt before the year ends, with PCORI’s budget up for reauthorization in Congress. Researchers, clinicians, patients, and other stakeholders who have benefited from PCORI want to keep the momentum going. 

According to an internal AAMC analysis, 70% of the total dollar amount of PCORI-funded research has gone to AAMC-member institutions. 

“Not only are these institutions well-poised both to conduct the research, but as health care providers themselves, also to put the results of that research into practice,” says Tannaz Rasouli, MPH, senior director of government relations at the AAMC.

The story of PCORI

PCORI was established in 2010 under the Affordable Care Act and began funding research in 2012. The institute relies in part on taxpayers to keep it running: Employers providing self-insured health plans must pay a fee toward PCORI, which is currently $2.45 per employee annually. 

That fee has gone toward a wealth of research in the past nine years, targeting five priorities: assessment of prevention, diagnosis, and treatment options; improving health care systems; communication and dissemination research; addressing disparities; and accelerating patient-centered outcomes research (PCOR) and methodological research. 

“We feel passionate about our mission and we feel that we are supporting a research area that has not had a focal point before.”

Diane Bild, MD
PCORI

While studies in PCORI’s research portfolio differ in scope, they all seek to answer questions that can improve either quality of care or life for patients. These results can be found in numerous medical journals: A study published in JAMA Internal Medicine, for example, showed that daily self-monitoring with finger sticks offers little benefit for people with Type 2 diabetes — findings that can save patients unnecessary pain and costs. Research published in the Annals of Internal Medicine can help adults with severe obesity compare the most effective weight loss surgery. And providers can use the results of a study published in BMJ to guide decision-making for prescribing the diabetes drug metformin to patients with prediabetes.  

“PCORI is focused, and uniquely positioned, to really look at what's happening in the health care system now, figuring out what works best and for whom, and then working with our end users to figure out how to best get that information out there so that we can actually inform decisions and change practice for the better,” says Andrew Hu, MPP, PCORI’s director of public policy and government relations.

Power to the patient

Across PCORI-funded research, patients are made to be partners in studies. Patients have long advocated for more representation in the research affecting them, and the value they bring to studies is gaining recognition: A qualitative analysis of engagement in PCORI-funded research found that many studies showed patient engagement improved the quality of research, concluding, “Ultimately, evidence will be more useful and relevant to decision making when patients and stakeholders are fuller participants in the research process.” 

PCORI’s emphasis on patient engagement marks a shift in how research is being carried out, with greater emphasis placed on listening to the patient voice and treating them as experts. It’s also helped researchers think differently about desired outcomes.  

“There are a whole host of outcomes that may be important to patients that the researchers can incorporate into their research based on input from patients to make sure that the research is highly relevant to the patient community,” says Bild. 

The earlier that patients are involved in research, the better, says Rebecca Sutphen, MD, principal investigator at the ABOUT Network. The patient-powered research network focuses on hereditary breast, ovarian, and related cancers, with studies exploring topics such as genetic counseling before BRCA testing and risk-reducing mastectomy. 

By involving patients at the onset of research, “studies could be better designed [and] the collection of outcomes could be improved,” says Sutphen, professor at the University of South Florida (USF) Health Morsani College of Medicine and the Health Informatics Institute at USF Health. “There’s so many things that frankly a researcher would not necessarily think of that a patient would.”

Harnessing a ‘network of networks’

PCORI can produce such a vast amount of research thanks to its PCORnet initiative.

Described as a “network of networks,” PCORnet enables patients, clinicians, researchers, and other stakeholders to harness electronic health data from 13 clinical data research networks, 20 patient-powered research networks, and two health plan research networks. 

“A lot of this work is fundamentally social, it’s about building relationships, and PCORnet is a fantastic forum for not only collaborating on research, but also sharing best practices with each other.”

Christopher Forrest, MD, PhD
Children’s Hospital of Philadelphia

“One focus area for PCORnet was to get the electronic health data into a common data model that can be quality checked and reusable for research across multiple health systems,” says Adrian Hernandez, MD, MHS, co-principal investigator for ADAPTABLE, PCORnet Coordinating Center principal investigator, and vice dean for clinical research at Duke University School of Medicine. “This enables end-to-end research from identifying people who may be eligible for a clinical trial such as ADAPTABLE to engagement, enrollment, and follow-up participants.”

The PEDSnet clinical data research network comprises eight large pediatric medical centers and represents more than 6 million children. But researchers can connect to even more potential study participants when necessary through PCORnet, says Christopher Forrest, MD, PhD, principal investigator of PEDSnet and chair of the PCORnet Research Committee. In addition, having access to a broad network of networks has fostered collaboration in the research space.  

“A lot of this work is fundamentally social, it’s about building relationships, and PCORnet is a fantastic forum for not only collaborating on research, but also sharing best practices with each other,” says Forrest, who is a pediatrician at Children’s Hospital of Philadelphia. “Each of the networks is trying to do very similar things, so by having those relationships we can easily reach out to other networks.” 

Getting the word out

Besides funding research, PCORI works to disseminate and implement findings within communities with initiatives including translating CER into simple language and evidence syntheses to help patients and their families understand previous research on clinical questions and topics. 

Putting research into practice means that results get into the hands of communities. Often, studies will wrap without further dissemination of results to the public, says Sue Friedman, DVM, co-principal investigator at the ABOUT Network. But such information can have a much wider reach with the right collaboration, as is the case with the ABOUT Network. The network represents a partnership between researchers at the University of South Florida and the nonprofit Facing Our Risk of Cancer Empowered (FORCE). 

“Partnering with an advocacy group allows researchers to make sure that the results of their research live on,” says Friedman, executive director and founder of FORCE.  

Up for reauthorization 

With the deadline for reauthorization on the horizon, PCORI is stepping up its efforts to garner bipartisan support for continued funding. The organization has worked to educate and raise awareness of the value of PCORI with members of Congress and other stakeholders to explain the role that such research has in addressing public health concerns facing many Americans, such as opioid addiction and mental health issues, says Hu.   

PCORI’s supporters say that the institute has the potential to shift research for the better and has already made progress toward that end. 

“It has galvanized a community partnership with investigators, researchers, clinicians, and patients to address these problems so that ultimately when there’s a result, the impact is not measured by a citation in a journal, but the impact is measured by how a health outcome trajectory has changed,” says Hernandez. “It’s a different way of thinking.”