Under the Radar is an occasional series in which ľֱ looks at organizations and agencies in the nation's capital that readers may know little about. In this first installment, we look at the Patient-Centered Outcomes Research Institute (PCORI).
David Penson, MD, MPH, sat in a congressional hearing room in Washington a few weeks ago, discussing the results of a study his team did on the side effects of various prostate cancer treatments. In his study, researchers surveyed about 3,600 men in five different parts of the country who had been diagnosed with prostate cancer to find out about the side effects of treatments they had undergone.
"What we're able to show was, if you have surgery, people think it is the best chance for control and cure -- but there is a very high risk of erectile dysfunction and leaking urine," said Penson, who is director of the Center for Surgical Quality and Outcomes Research at Vanderbilt University, in Nashville, Tenn. "[We also] looked at external beam radiotherapy ... you can get sexual dysfunction after [that] therapy but it will be less -- but [also you find] bowel dysfunction including loose stools and blood in the stool." Even active surveillance or "watchful waiting" also showed some incidence of sexual dysfunction, he added.
"So now if you're a man faced with this [choice], you have this information ... so you can make an informed decision," said Penson. He noted that when he presented his study results at a Canadian urology meeting, attendees described the findings as "shocking" and "practice-changing" -- not because they weren't aware of these possible side effects, but because this study showed them to occur in much higher numbers than previous studies, which were usually single-center analyses using carefully selected patients.
Penson was one of several panelists at a briefing hosted by the (PCORI), and his study, which was partly funded by the institute, epitomized the organization's stated mission: to give physicians and patients the information needed to make better decisions about patient care.
Origins in the ACA
PCORI began in 2010 as an outgrowth of the Affordable Care Act (ACA). The reads, "There is authorized to be established a nonprofit corporation, to be known as the 'Patient-Centered Outcomes Research Institute' ... which is neither an agency nor establishment of the United States Government ... The purpose of the Institute is to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments [and] services."
But the agency has been criticized for not fully living up to its mission when it comes to funding comparative effectiveness research (CER) like Penson's. A issued by the Center for American Progress (CAP), a left-leaning Washington think tank, found that "the institute allocated 58% of its grant funding to CER between December 2013 and January 2016. Cumulatively through January 2016, PCORI has now dedicated 51% of funding to CER. However, this remains far short of CAP's longstanding recommendation to devote at least 80% of funding to CER."
The report's authors - Ezekiel Emanuel, MD, PhD; Topher Spiro; and Thomas Huelskoetter -- were quick to note that these numbers did represent progress. "CAP's [earlier] analysis found that in its first 4 years, PCORI allocated only 37% of its grant funding to comparative effectiveness research. Most of the institute's funding had gone to studies of communication tools, patient decision aids, methodological approaches, and the establishment of data infrastructure -- rather than to actual CER studies of medical interventions."
But Joe Selby, MD, MPH, PCORI's executive director, said his organization dissented somewhat from CAP's definition of CER. "For example, they argued if you were comparing a drug or treatment to usual care, then they didn't feel that was CER; we just disagree," said Selby, who spoke during an interview at which a public relations person was present. Sometimes physicians need studies that compare new treatments to usual care because they need to figure out whether they should stop doing what they're doing and switch to the new treatment. "So [in that case], usual care is a legitimate comparator," he added, noting that PCORI is careful to tell grant applicants that they must describe exactly what usual care consists of.
PCORI's show that of the $1.68 billion it has awarded for grants so far, $1.24 billion -- or 73.7% -- has been given to CER studies, $324 million (19.2%) has gone to studies of healthcare infrastructure (such as use of healthcare navigators and discharge planning), and $199 million (7.1%) has gone to studies on improving research methodology.
Lack of Prescription Drug Studies?
In their report for CAP, the authors expressed particular concern over PCORI's lack of studies that compare prescription drugs to one another. "CAP's previous analysis found that only about 2% of grant funding went toward studies that involved drugs," the authors wrote. "Since December 2013, 11.3% of funding has gone toward CER studies involving at least one drug -- but only half of this funding went toward studies that actually included head-to-head comparisons of two or more drugs. Cumulatively, only 4.4% of PCORI's funding has gone toward CER comparing two or more drugs ... This funding is far too low."
Actually, PCORI has "really tried to keep eye on the ball when it comes to new, high-priced agents," Selby said. For example, the organization is funding a large comparative effectiveness study of various hepatitis C agents and several studies of high-priced biologics.
Unfortunately, however, CER might not be the best way to figure out what the price of a drug should be, he added. "Sometimes this is a problem settled better by policy than by research. One nagging issue is that it takes a long time to do research -- recruiting patients and following them can take 4-5 years, so CER is not the quickest [way] ... to help decide what price of a drug should be."
Organizing Research Networks
The organization also has its staunch defenders. "They are doing a tremendous job," said Tom Concannon, senior policy researcher at the RAND Corp. in Boston and a member of one of PCORI's 33 clinical research networks -- groups of clinicians, patients, and other stakeholders focused on sharing research about particular diseases or patient populations.
PCORI has been instrumental in developing an effective way to structure a large clinical trial involving many hospitals, Concannon said in a phone interview. "There are all sorts of governance issues regrading hospitals using data for research purposes, both competitive issues and patient protection issues," he said. For example, "If you're a health system with a high number of patients receiving a high-revenue service, it's easy to identify which hospital it is if that data goes to research," even when the data have been scrubbed of individual patient identifiers.
Patient privacy issues are paramount when it comes to research, Concannon continued. "Every health system and provider is required by HIPAA [the Health Insurance Portability and Accountability Act] to protect the privacy of patients receiving care in their institution. So they not only need to be sure data are de-identified, but also that they can't be re-identified by overlaying other data on them. That can only be done by a large public/private startup like PCORI ... What PCORI has done is something that has not been done by other parties."
Not reauthorizing PCORI -- whose funding runs out in 2019 -- would be a "disaster for patients," he said. In Chicago, "there are 10 health systems involved in the PCORI's clinical data research network. That's just something you haven't seen elsewhere -- an entire regional mission collaborating to produce research-ready data sets."
PCORI is reaching out to Congress about its work, Selby said. "Now we can talk increasingly about our results and how it's a different kind of research." And it is making a plea to physicians as well.
"We know you have a tough job ... so get in touch with us, work with us, help us identify the questions bothering front-line physicians," he said. "There is also a chance for physicians to get on our advisory panels, and working with physician organizations is a good way to get to us."