JOE SELBY, M.D., is a family physician and researcher who was tapped as the first executive director of the Patient-Centered Outcomes Research Institute. The organization was established in the Affordable Care Act as a way to help patients make more informed decisions while improving health care delivery. The institute has been accepting applications for some $96 million in funding for research this year, including its Challenge Initiative, which is seeking ways to match patients and researchers for collaborative projects. The deadline is April 15. | Interviewed by Marty Stempniak

Talk a little about PCORI and its mission.

SELBY: PCORI, the Patient-Centered Outcomes Research Institute, is an independent, nonprofit organization authorized by Congress and the Affordable Care Act, and its mission is to fund and conduct research that will provide information to help patients and those who care for them make more informed health care decisions.

Why has traditional research in health care failed to answer patients' questions?

SELBY: Well, traditional research has other motivations than answering patients' questions. It often tends to ask the wrong questions [and makes] the wrong comparisons. It doesn't necessarily compare the options patients face or are considering, and even when it does, it often fails to include all of the outcomes or consequences of those decisions. Two quick examples: Studies that compare a treatment with a placebo don't answer a patient's questions, because the patient isn't thinking about taking a placebo; also, many studies fail to measure the rate at which patients are able to return to work after a procedure. If the outcome is not known, then patients lack some of the information they often need.

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Joe Selby, M.D., says that getting patients more involved in clinical and outcomes research will make studies more relevant. Running time: 7:30.

Why should patients be more involved in research and in their care?

SELBY: It makes the research better and certainly makes it more relevant. It makes sure we get the questions right. It makes sure we get the outcomes right. If patients actually are engaged in doing the research, it helps to make sure that the research is valid; and at the end of the line, if the study uncovers important information, it gives us a jumpstart in disseminating that information.

So, if we involve not only patients, but also other key stakeholders or users of the information, it helps us get that information out at the end of the study.

What sorts of things do patients want to know more about?

SELBY: Often key outcomes or consequences of treatments aren't measured — things like the quality of one's life or your ability to function. And sometimes there are just too many choices, such as with back pain. People with back pain that has lasted more than three weeks or so, depending on where you go and whom you see, will get recommendations that range from massage to surgery, with a lot of intermediate stops like chiropractic, drugs, etc. So, those comparisons are often incomplete. And even when the comparisons have been done, they often have not looked to see how specific patients and patient groups have done. Patients really want to know how they will do. Very little research has looked at how treatments work for patients like them, patients with particular characteristics.

What specific methods will be used to get patient input?

SELBY: One example is that we've set up on our website a simple survey into which patients can enter questions. We hold workshops or work groups where patients or other stakeholders come together to discuss particular clinical areas, and ask questions. When we engage with patient organizations or the organizations that represent other stakeholders, like clinicians or caregivers, we get questions from those sources. We are creating standing advisory panels that represent all the major stakeholder groups. So, yes, patients; yes, caregivers; but also clinicians, hospitals and health care systems, health plans and the life sciences industry. All of these are represented. The advisory panels, we think, will help us to both identify questions and help us prioritize them.

Some have expressed concerns that PCORI is pushing for "one-size-fits-all" medicine.

SELBY: First of all, comparative-effectiveness research really is, I think, a big step away from the notion of one-size-fits-all. It really asks the question, "What works for whom?" That very question makes it clear that we're not saying, "One thing works best for everybody." We're dedicated to and bent on pushing the research boundaries to figure out what works best for whom? We tell all of our awardees that they have to attend to the question: "Does this work the same for everybody, or are there specific patient groups for whom this works well and for whom it doesn't?" In no way do we make policy. Mainly, our research is driven by what patients and clinicians tell us they need to know, and our job is to communicate the research findings with the community.

What challenges lie ahead as you look to roll out your vision in 2013?

SELBY: We spent a lot of 2012 setting up and establishing connections or engagement with our various stakeholder groups, and in 2013, without a doubt, the biggest challenge is to get the funding out the door, the funding of comparative, clinical-effectiveness research. We aim to commit at least $350 million in this year alone, so that's challenge No. 1. Continuing and expanding that engagement to other stakeholder groups, including clinician groups and hospitals and health plans, is the other big challenge. And then, bringing these various stakeholder groups together in different kinds of settings so that we can network and achieve consensus as to what's needed in research is a third big activity for this year.

How will you prioritize whether to address diseases that impact many patients vs. diseases that impact only a few?

SELBY: One way to measure impact is by the number of patients affected. A second way is to ask, 'How bad is the suffering and how might the answer to this question relieve the suffering?' And the third way is to say, 'There may not be a great number of patients impacted by this, but this is an area where there's really a burning question and we don't see another organization that's going to fund it.' It's a comparative question, it's a critically important question for patients who are affected, be they few or numerous, and nobody else is going to take this on.


CV: Family physician who conducted health services and outcomes research and led a research center for Kaiser Permanente. "I was very taken with answering practical questions that could help improve health care delivery and meet the needs of decision-makers there. And over that time, as comparative-effectiveness research began to be talked about, I became very fascinated with it."

Hobbies: "My hobby is playing the piano; I love to do that and I play a little bit every night just to sooth my nerves. the other thing is exercise — I like running and fitness training."

Reading List: "I just finished a book on Winston Churchill about the years between World War I. and World War II.