By STEVE FINDLAY

Earlier this month the Center for Public Integrity (CPI) published a sharp-edged piece on PCORI—the Patient-Centered Outcomes Research Institute. 

The piece raised some salient issues and it’s timely to take stock of PCORI at the half way point of its authorized funding.  (Unless renewed, PCORI sunsets in 2019.) 

The Affordable Care Act created PCORI as an independent nonprofit (non-government) entity.  But PCORI’s funding and structure makes it more or less quasi-government.  It gets its money from the Medicare trust fund, treasury general funds, and a tax on private insurers and self-funded insurance plans ($2.08 per covered life).  PCORI launched in late 2010 and began funding research in earnest until 2013.  The main focus of that research, mandated by Congress, is to compare treatments in a way that results in meaningful results for doctors and patients as they make clinical decisions.  No small task. 

The CPI piece probes the emerging debate about how PCORI is being operated and spending its money—roughly $450 million a year in 2014 and 2015.  The core lead-in graph of the piece: “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.”   

The report airs legitimate concerns but it skews overly critical and doesn’t fully appreciate the challenge PCORI faces.  As someone who labored in the fields of comparative effectiveness for several years, I think PCORI deserves time to get fully underway and prove itself.  It certainly doesn’t need ideologically driven attacks and budget threats just because it was launched by the ACA.   (The House Appropriations Committee in late June voted to cut PCORI’s funding by $100 million, dubbing it wasteful spending.) 

Nor does PCORI need idiotic climate denier-like missiles such as Kansas Republican Pat Roberts’ bill earlier this year to explicitly prevent Medicare from using PCORI results to “deny or delay coverage.”

If anything, you’d think Republicans would see that PCORI has (in theory) the long-term potential to reduce health spending even as it improves people’s chances of getting the right care and better treatment outcomes.    

The legitimate concerns

From the CPI piece and my own observations:    

(1) PCORI has been overly bureaucratic and cautious to date, lumbering along too slowly as federal agencies sometimes do, with little apparent sense of urgency even as suboptimal care and excessive costs pose huge long-term problems for the federal budget, businesses, and families.   

(2) PCORI’s infrastructure was quickly dominated by scientists and researchers who put too much (perhaps self-serving) emphasis on solving somewhat arcane methodological and scientific issues, instead of producing research results as quickly as possible.  According to CPI’s analysis, PCORI has spent some $70 million on projects to improve methods for conducting research or build links to the medical research community. 

(3) With a mandate to involve stakeholders from throughout the healthcare system, and especially consumers/patients, PCORI spent too long (a couple years) setting up procedures and processes to be (perhaps overly) inclusive.   The $70 million figure cited above included nearly $10 million in “engagement” awards and “meeting and conference” subsidies for medical societies and other groups.  The CPI report says one physician organization got $250,000 just to survey its members on what they think about PCORI and its work. 

(4) PCORI has spent millions on outreach and meetings to determine how it can best disseminate and communicate the findings of its’ research to both healthcare professionals and consumers.  While its web site has been getting better, some of the explanations and summaries of research projects are, in the words of the CPI report, “freighted with academic and scientific language” that would be hard to understand even by front-line docs. And, as yet, there are no meaningful summaries for consumers on research results; that’s promised for later this year. 

(5) PCORI is badly hamstrung by its authorizing legislation in that it’s prohibited from having its’ research findings “construed as mandates for practice guidelines, coverage recommendations, payment, or policy recommendations.”  

That provision was a sop in the ACA to conservatives who didn’t want PCORI producing anything resembling federal treatment guidelines or dictates, and especially those that were based on an assessment of cost effectiveness.   

The problem now is that PCORI’s leadership has too eagerly embraced this limitation.  It bends over backwards to avoid any appearance of crossing the line into cost effectiveness.  In the words of PCORI’s executive director, Joe Selby, as quoted in the CPI report: “We are not in any way a cost effectiveness shop.  That is not our job and there is a certain wisdom in saying that we shouldn’t get involved.”

That may indeed seem like a wise policy with the House and Senate now in Republican hands.  But avoiding the issue of cost altogether is illogical, short-sighted and makes no policy sense.  It never did. 

A very simple example:  A large-scale PCORI study finds treatment A and treatment B completely equal in effectiveness and safety.  Treatment A cost $1,000.  Treatment B cost $100. Under the current policy, PCORI would literally be unable to say anything about that cost differential in its communications to doctors and the public. Yes, journalists and others can point the differential out, but it’s not the same. 

Moreover, as the CPI piece notes, PCORI is not restricted from funding research that aims to improve the “efficiency” of healthcare.  But PCORI critics concur it is shying away from research that cuts to close to the cost effectiveness bone on that front, too.  With the Agency for Healthcare Research and Quality (AHRQ) also under fire (from Republican threats to defund it), it’s likely that in coming years privately funded efforts will have to fill the gap on ferreting out waste in the system.  Sad.   

What PCORI is getting right

PCORI is on the leading edge of the health data revolution.  It has committed substantial funding to create broad information networks wherein the sharing of data can drive improvements in care. 

For example, PCORI has spent or budgeted $237 million to support the development of PCORnet, also known as the National Patient-Centered Clinical Research Network.  PCORnet is made up of 11 clinical data research networks and—here’s the really novel part in my view—18 so-called Patient-Powered Research Networks (PPRNs). 

The PPRNs are operated by patient groups and oriented around getting patients and their families directly involved in feeding data and their experiences into a distributed database to help improve care.  Across all 18 patient networks, an estimated 500,000 to 1 million people are expected to enroll and participate in the next few years.    

Up that same alley, PCORI has taken seriously its’ mandate to involve consumers/patients in research.  Patient advocates are involved from the beginning in the screening of research topics, and even in the choice of which grant applications get funded.  This is a novel approach and even docs and research experts agree it’s a positive development.   

Despite the slow ramp up and perhaps excessive expense, PCORI does have the potential to accelerate the diffusion of research results and innovations.  Uniquely, it’s asking tough questions about why it takes so long for doctors to change their behavior and practices when research clearly indicates a change is needed.  And it’s exploring novel ways of communicating to consumers.    

Finally, PCORI is ramping up the kind of research it was created to fund.  A few quick examples:

— A PCORI-funded study of blood thinner use among 12,500 stroke survivors found that those taking the meds had better quality of life, including more time at home and out of the hospital—a favorable “patient-centered” outcome.   

— A study of whether breast cancer screening tailored to women’s individual risks and preferences will detect cancer and minimize screening-associated harms more effectively than standard annual screening.

— A study to determine the best daily dose of aspirin to prevent heart attacks and strokes in people with heart disease.  The study will be conducted mostly through PCORnet. 

— A study to find out whether antibiotics or surgery is more effective and safer for treating patients with uncomplicated appendicitis.  (Recent research indicates that surgery is often unnecessary.) 

— A study to find practical and the best ways to prevent falls among people aged 65 and over, a major cause of broken hips and disability.

— A study of whether standard primary care or prompt referral to physical therapy plus cognitive behavioral therapy is more effective in preventing acute low back pain from progressing to chronic pain.

— A study comparing the treatment options to prevent potentially deadly blood clots in patients getting hip and knee replacements.

— A study comparing the various kinds of weight loss surgery

PCORI grew out of the very practical for “applied” research comparing treatments.  That need exists because companies making drugs and devices and the other tools in the healthcare shed have absolutely no incentive to compare their products to others; quite the opposite.  Also, research over the past 25 years has documented that (a) many treatments and a shocking proportion of what doctors do are not all that effective and (b) doctors and consumers are often confused or uninformed about the full range of treatment options—the pros and cons, the risks, etc.

The impetus to create PCORI was, then, fully justified, and its’ mission and mandate meshes well with the rest of the ACA.  That said, PCORI has limited time to prove that it can make a difference.   

Steven Findlay is an independent journalist who covers medicine and healthcare policy and technology.