THE PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE (PCORI) was created by the Patient Protection and Affordable Care Act (PPACA) to examine the “relative health outcomes, clinical effectiveness and appropriateness” of different medical treatments by evaluating existing studies and conducting its own. PCORI is governed by a 21-member Board of Governors, which includes patients, doctors, hospitals, drug makers, device manufacturers, insurers, payers, government officials, health experts and the directors of the Agency for Healthcare Research and Quality (AHRQ) and the National Institutes of Health (NIH).
In 2010, ACA Senior Scientific Advisor Christine Goertz, DC, PhD, and vice chancellor for research and health policy for Palmer College of Chiropractic, became the only DC appointed to the Board of Governors of the then newly created PCORI. Previously, she had served as deputy director of the Samueli Institute and as program officer for NIH’s National Center for Complementary and Alternative Medicine (now called the National Center for Complementary and Integrative Health, or NCCIH).
PCORI explains on its website that traditional medical research, for all the remarkable advances it produces, hasn’t been able to answer many of the questions that patients and their clinicians face daily. And even when reliable information does exist, it’s not always available to patients, or those who care for them, in ways they can understand or use most effectively. In order to rectify that, PCORI began funding research in December 2012 and now funds a portfolio of projects designed to improve patient care and outcomes through patient-centered comparative clinical effectiveness research, or CER.
The PCORI Difference
“At PCORI we like to say that the patient is our North Star and that we focus on research designed to answer questions about things that really matter to patients,” says Dr. Goertz. “That questions about chiropractic care really matter to patients is evidenced by its fairly common use, especially in people with low-back pain.”
Michael Schneider, DC, PhD, an associate professor in the Department of Physical Therapy, School of Health and Rehabilitation Sciences, and associate professor of the Clinical and Translational Science Institute at the University of Pittsburgh, is the only chiropractic physician so far to receive a PCORI grant (see sidebar).
Dr. Schneider notes that PCORI grants are very different from traditional NIH grants. Dr. Schneider says, “PCORI was created as clinical scientists recognized that NIH was traditionally focused on research studies about mechanisms of action and basic science. This focus led to NIH funding of clinical trials that were designed to be extremely fastidious, which takes away from the practical nature of clinical research.” All the PCORI grants have patient-centered outcomes as their focus. “That is a very big deal,” says Dr. Schneider, as “it sounds like it would just make common sense, but a lot of research is not patient-centered.”
Dr. Goertz believes that the study of chiropractic and PCORI are a natural fit. “The concepts of pragmatic trials and patient-centered care are issues we talk about as if they were new frontiers of science. However, I’ve been doing pragmatic trials on patient-centered care since I began my research career, because that is about your only option when you study chiropractic in a clinical setting,” she says.
Dr. Schneider says historically a lot of the chiropractic profession’s interest has been focused on biomechanics, neurophysiology and animal research — not clinical trials. “Clinical trials require a lot of money, and big grants to chiropractic researchers up to this point have been basically NIH grants, but now there is another place to go with research grant applications and that is PCORI,” he says. “Unfortunately, I am the only chiropractor that has a PCORI grant so far, and I’m not at a chiropractic college.” [For more on funding of chiropractic research, see Chiropractic College Research: Evidence as Validation, May 2015 ACA News, Page 32 at http://mydigimag.rrd.com/publication?i=255471
Dr. Goertz is disappointed that PCORI hasn’t received more chiropractic research applications. “Research dollars are very hard to come by these days, and I think PCORI and the mission and interests of PCORI are very well-aligned with chiropractic research,” she says.
PCORI and Chiropractic
The NIH/PCORI dichotomy is easily illustrated. Before Dr. Schneider submitted the stenosis grant application to PCORI, he explored the possibility of submitting it to NIH. However, after explaining his research design, he was told that NIH would not likely be interested in this type of research project because it was too pragmatic and not fastidiously controlled. He explains that in a controlled trial, all treatments must be very specific with protocols that allow for little or no deviation by the treating clinicians.
Dr. Schneider calls his stenosis trial much more pragmatic. “Believe it or not, there never has been a large clinical trial providing a head-to-head comparison of non-invasive methods, such as spinal manipulation and exercise for the treatment of lumbar spinal stenosis,” he says. “Every trial has involved a surgical component or an epidural injection component — there never has been a direct comparison of different nonsurgical approaches.” The end result is that many DCs and physical therapists in the United States are treating patients with spinal stenosis, and there is no evidence to guide them.
For severe stenosis, research shows surgery does give people at least short-term relief for a couple of years. But two years after surgery they are about the same as if they didn’t have surgery. “Worse yet is if you have mild to moderate stenosis, which is clearly not surgical. When you look for the evidence as to what to do from a nonsurgical perspective, there is nothing,” Dr. Schneider explains. ”Should patients get traction or manipulation or exercise – there is really no literature at all,” he says.
To understand PCORI, you have to examine the grant process. The PCORI grant mechanism requires the principal investigator to involve community stakeholders from the outset. “For a traditional NIH grant, I sit down, write a grant, submit it and I either get funded or I don’t,” explains Dr. Schneider. “At PCORI, you can’t submit a grant unless you have stakeholders that help you design that grant, so PCORI wants you to ideally have patients with the disorder on your stakeholder panel and help to design the research question.”
Dr. Schneider had to interview people with stenosis in focus groups and ask them what they thought mattered to them clinically. “It would seem to be common sense, but you would be surprised how many research studies have been done where nobody has actually spoken to the patients who have the condition being researched,” he says. So he asked patients, for example, whether he should measure pain scales as a primary outcome. The patients explained there isn’t much pain when sitting, but their main concern is inability to walk; so they suggested that it would be more important to have some type of measure of how far they can walk before and after treatment.
Dr. Schneider went to other stakeholders, including community senior centers. Many patients with stenosis were in exercise classes and would ask staff if exercise might be helpful, but staff didn’t have an answer. That led Dr. Schneider to include group exercise classes at senior community centers as another of the arms in his study. His study has three groups to which people can be randomized. But he explains that including a group exercise arm in a randomized trial would probably not be allowed with an NIH grant, because the seniors can self-select what exercise class to go to, introducing too many uncontrolled variables in the study.
PCORI allows for pragmatic trials so not all patients in any one arm receive the same treatment; instead the treating clinician is allowed to individualize it for the patient, as in real life. “In an NIH trial, you would probably not be able to do that — the trial would be expected to be more regimented. For example, in an NIH-funded trial, I would be expected to delineate exactly what treatments the chiropractor would do to each and every patient, such as 'do Cox Technique for five minutes and stretch both hamstrings for 30 seconds,' but I couldn’t vary that because it wouldn’t be fastidious,” he says. “In our PCORIfunded trial, we have a treatment protocol that includes a ‘package’ of manual therapy techniques and exercise interventions that the chiropractors can do, but they are given the leeway to use their clinical judgement to make modifications from within that ‘package’ in order to individualize the treatments for each patient.”
Dr. Schneider laments that PCORI is currently scheduled to sunset in 2019. It would have to be reauthorized and refunded, so it’s impossible to tell if it will exist five years from now. “Chiropractic researchers have an advantage here because we are more patient-centered to begin with,” says Dr. Schneider, noting that "the window of opportunity may close."
Dr. Schneider’s PCORI grant was approved in the first round of applications, when only 25 grants were awarded nationally. “The profession should take advantage of the publicity around one of the first 25 PCORI grants being awarded to a chiropractor,” he says. Not only is it good publicity for the profession; even researchers from some Ivy League institutions have been calling him for advice about how to write a successful PCORI grant. “But I realize many of these researchers won’t get a PCORI grant, mainly because they can’t make the shift from efficacy studies to pragmatic studies,” he adds.
Dr. Goertz wants to ensure that DCs know PCORI exists and understand PCORI’s mission. “PCORI is committed to collecting the data that patients and clinicians of all kinds, including DCs, need in order to make well-informed healthcare decisions,” she says. “But PCORI also is interested in dissemination, in figuring out how to make sure that clinicians and patients are aware of that data once it actually exists.”
It takes about 17 years for new research findings to trickle down into clinical practice, according to Dr. Goertz. "I hope that this time frame is accelerated by the rapid mechanisms we have now for information dissemination, and I believe that PCORI is, and will continue to be, a very important vehicle not only for collecting data but for disseminating findings to patients and clinicians."
Spinal Stenosis Research Funding
ON JAN. 22, 2013, PCORI APPROVED $1.67 MILLION FOR A THREE-YEAR RESEARCH PROJECT LED BY ACA MEMBER MICHAEL J. SCHNEIDER, DC, PHD, that studies nonsurgical treatment methods for patients with lumbar spinal stenosis.
The funding came after a highly competitive process; only 5 percent of the applications submitted were awarded funding. In all, 25 applicants were approved for contracts totaling $40.7 million over the next three years as part of PCORI’s patient-centered comparative clinical effectiveness research projects — the first of four areas of its National Priorities for Research and Research Agenda.
Lumbar spinal stenosis is found in about 30 percent of older adults, and it is the most common reason people older than 65 have back surgery. However, such operations are expensive and risky, with a high number of complications that cause many patients to be readmitted to the hospital. Moreover, a large number of patients with stenosis can be treated with other methods, such as chiropractic services, exercise, physical therapy and medication. Health care providers just don’t have enough research indicating which treatment works best for which patient and under which circumstances. Dr. Schneider aims to provide more information about the effectiveness of the various nonsurgical choices for managing stenosis.