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The new Patient-Centered Outcomes Research Institute (PCORI) has been asking different stakeholders about the most important issues to address with the hundreds of millions of dollars the quasi-governmental group will shortly be doling out in grants. Not surprisingly, the stakeholders have been more than happy to respond.

PCORI’s most recent day of dialogue, which I attended as a representative of the Society for Participatory Medicine (SPM), was characterized by genteel civility and a big question mark: “Is PCORI serious about transforming health care?” When I asked directly, I didn’t get much of an answer. The reason, I suspect, goes to PCORI’s origins. It is the offspring of a shotgun marriage between goo-goos and pinky-ringers, and no one is quite sure yet what this child will be once it grows up.

Let me pause here a moment to parse the political shorthand. “Goo-goos” (Wikipedia) are “good government” types, the kind of folks who trumpet the need for transparency in government or better public transit. Goo-goos, seeing the half trillion dollars or so of waste in U.S. health care system, called for a new national organization to carry out comparative effectiveness research in order to help Americans get the most value for our money.

The goo-goos pointed out that our current regulatory structure is designed to ensure that treatments are safe and effective, not compare them. Nor does the private sector have much incentive to pay for comparative studies that may undermine products currently selling quite nicely, thank you.

In contrast to the goo-goos are the pinky-ring set of political realists, whose motto was succinctly described by the late columnist Mike Royko: “Where’s mine?” Though some political pragmatists gush with goo-goo intentions – pacem Rahm Emanuel ­­­–they did not attain power by pulling all-nighters with a civics textbook.

Pinky-ringers know that one man’s waste is another man’s comfortable income, and that what looks like a rational buying decision to one person can appear to be rationing by bureaucratic fiat to another. Or, as I heard a drug company official put it a couple of years ago, comparative effectiveness research opens the door for telling kids with cancer it’s not worth funding an effort to find drugs to treat it.

To head off this perceived threat, the pinky-ringers formed the Partnership to Improve Patient Care, which – according to the Wall Street Journal – included “the lobbying arms of the drug, device and biotechnology industries, as well as patient-advocacy groups and medical-professional societies.” The partnership says it favors “well-designed” comparative effectiveness research, but is concerned about research that might be used “in an inappropriate manner that may limit treatment options for patients.”

As the Affordable Care Act inched towards law in 2010, the goo-goos and pinky-ringers reached a compromise. PCORI was set up as an independent non-profit, with a budget coming from ACA monies without need for a yearly Congressional appropriation. However, the agency’s mission was carefully defined, any reference to “comparative effectiveness” was taken out of the title and “cost effectiveness” became a no-no.

The organization’s vaguely upbeat name, however, has drawbacks. “Patient-centered outcomes research” is really a euphemism for “comparative clinical effectiveness research,” which might be confused with “cost-effectiveness” which might remind someone of rationing. Since the whole point of a euphemism is not to acknowledge it, PCORI and academics with dollar signs in their eyes have to pretend they’re inventing something new. Sometimes they are, sometimes they aren’t.

At the workshop I attended PCORI gave the example of a 77-year-old black male with diabetes and hypertension who’d had a heart attack and needed to choose between a stent and bypass surgery. What patient-oriented research was needed?

Sadly, I’m not sure PCORI’s statement of principles ­– “elevating and repositioning patients, caregivers and others as valuable but previously untapped partners in the patient-centered research enterprise” – has much to add here.  The conventional outcome measure of mortality seems to me as patient-centric as you can get, whether or not patients were involved with the study design. “Don’t kill or injure me” will always rank high on any patient’s list of “preferences and values.”

A couple of attendees suggested a more patient-centered target would be a middle-aged woman. That’s confusing two separate issues. Research to save a woman’s life is intrinsically no more patient-centric than research to save a man’s life; we, too, worry about “our bodies, ourselves.” Expanding the diversity of clinical research is a separate issue, but PCORI is hardly the first to address it.

Similarly, traditional clinical research on chronic disease is moving by necessity to include “patient-centered outcomes” such as pain and functional status. PCORI will accelerate that trend. But whether PCORI will push researchers towards breakthroughs in patient-centeredness or more creative relabeling remains to be seen.

It’s certainly encouraging that PCORI board and staff are walking the walk when it comes to modeling partnering. Towards meeting’s end, I felt the urge to rush the stage and present various PCORI luminaries with SPM membership cards to recognize their participatory props. Except we don’t have membership cards.

In any event, by then my SPM goo-goo side was too busy arguing with the pinky-ring part of me that’s spent way too much time at Washington meetings. Here’s how it went:

Goo-goo: PCORI is spending more than $100 million this year, $300 million in 2013 and $400 million in 2014 on patient-centered outcomes research. What a tremendous victory for patients!

Pinky-ring: Sure, they deserve kudos for prodding a closed research community to make sure the patient’s voice becomes an integral part of the process. But let’s wait and see whether that includes patients saying something other than, “My disease needs a lot more funding.” Not that there’s anything wrong with that.

Goo-goo: PCORI’s statement of principles declares: “Patients and those who care for them should be involved in every stage of the research process.” That’s very close to the SPM goal of “active involvement by patients, professionals, caregivers and others on all issues related to an individual’s health and health care.” I see the beginning of a beautiful friendship.

Pinky-ringer: Yes, welcoming patients as research partners is an important step SPM fully supports. But it’s only part of the picture. When you’re at your doctor’s office, or searching the web for information about your illness, you’re not involved in formal research. What’s PCORI doing that affects health care today?

Goo-goo: They want patients to have better tools to “make informed health care decisions.”

Pinky-ringer: So does SPM. And the first crop of PCORI awardees could publish promising ideas on how to do that somewhere around 2016.

Goo-goo: You seem to forget that one PCORI priority is “communication and dissemination.” It says right here: “empowering people to ask for and use information and supporting shared decision-making.”

Pinky-ringer: “Communication and dissemination research.” You want to support three PhDs and a statistician studying patient-centered communication, or dissemination of innovations, PCORI is your go-to place. You want to persuade people to change their behavior today, based on the evidence we know today about patient engagement, you’re on your own. Funding research that someone can use sometime to transform American medicine someday is a worthy endeavor, but irrelevant to activists pushing for change now.

The pinky-ring part of my personality has been reinforced by critical articles from respected PCORI observers such as Harold Sox and Douglas Kamerow. Still, PCORI could evolve. Maybe PCORI plans over time to pair activism with its knowledge gathering. Maybe PCORI’s outreach will build bridges of trust that allow it to have a more sweeping impact on patients’ lives in a shorter period than now appears likely. Or maybe my expectations of a research organization looking towards Congressional renewal of its mandate in 2019 are unrealistic.

At the workshop, the president-elect of the American Medical Association, Dr. Ardis Dee Hoven, repeatedly urged PCORI to quickly produce decision tools for doctors that are “relevant at the point-of-care.” She also was careful to endorse shared decision-making. I took that as a hopeful sign.

Because I can’t think of anything more patient-centered working with my participatory doctor to make sure that the very best outcomes happen to me.


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