How does one intervention or diagnostic test stack up against another in terms of patient outcomes, including perceived quality of life? Answering such questions is the goal of the comparative effectiveness research funded by the Patient-Centered Outcomes Research Institute (PCORI).
A nongovernmental nonprofit established through the Affordable Care Act, PCORI finances and promotes patient-oriented, clinically useful, evidence-based studies.
“What we fund are studies that evaluate the choices patients are facing,” says Joe Selby, MD, MPH, PCORI’s executive director.
Dr. Selby encourages more osteopathic physicians to apply for funding. PCORI has awarded more than $460 million in research funding since it became operational in 2011, but so far only one of the funded studies is headed by a DO principal investigator: Jeffrey Swigris, DO, of National Jewish Health in Denver is studying the effects of supplemental oxygen on patients with pulmonary fibrosis.
Besides Dr. Swigris, 42 MDs and more than 30 nonphysicians with PhD or other doctoral degrees are serving as principal investigators of studies funded in PCORI’s first two funding cycles.
Osteopathic physicians may be underrepresented among principal investigators because they are less likely to be affiliated with large academic medical centers, which have an established research infrastructure, Dr. Selby says. But DOs backed by osteopathic or allopathic medical schools, community hospitals or health systems, and national, state or specialty medical societies are urged to pursue funding if they have a research question and study design that meet PCORI’s standards.
“You have to have an institution behind you,” Dr. Selby says. “We aren’t permitted to make awards to individuals or to medical practices.”
PCORI is interested in funding studies on manual medicine and alternative medicine because such interventions are among the choices patients face every day, according to Dr. Selby. “The more we can shed comparative effectiveness light on these procedures and therapies and identify their optimal use, the better off patients will be,” he says.
Part of PCORI’s first funding cycle, a study led by a chiropractor compares three nonsurgical methods for treating patients with lumbar spine stenosis. Similar studies comparing osteopathic manipulative treatment to other interventions could be strong candidates for funding, Dr. Selby says.
However, PCORI’s standards are rigorous, he says, noting that “only about 10% or 11% of the applications submitted get funded.”
PCORI is committed to the following broad research priorities:
- Assessing prevention, diagnosis and treatment options.
- Improving health care systems.
- Communicating and disseminating research.
- Addressing disparities.
- Accelerating patient-centered outcomes research.
Guided by these priorities, PCORI in late 2012 issued a list of targeted research topics: treatment options for back pain, uterine fibroids, obesity in diverse populations, and severe asthma in African-Americans and Hispanics, as well as prevention of fall-related injuries among older adults.
But the organization is open to funding research on other topics, says Dr. Swigris, whose own research area, pulmonary fibrosis, is not on that list.
“What PCORI is looking for is research that is going to affect a large number of people, including patients who don’t have access to classic research centers,” Dr. Swigris says.
All research funded by PCORI must involve patients as participants in the research team, Dr. Swigris says. “Patients aren’t just used as guinea pigs to test our hypotheses,” he notes. “They should be involved in formulating the question. And they should weigh in on how the study is designed and on what outcomes are meaningful.”
For his study, Dr. Swigris selected his patient advisers, as PCORI calls them, from a pulmonary fibrosis support group at National Jewish Health. To come up with a study question, physicians can also use focus groups of patients representing a cross-section of demographics and conditions.
Not engaging patients in the research design is a key reason many applications are rejected, Dr. Selby stresses. “In the application, we want to see evidence that the investigators have opened up a relationship with patients as early as possible in the development of the research question and that they are going to be working with them throughout the study,” he says.
The application should describe the role of patients in detail. “We need to be convinced that investigators understand engagement and the importance of it,” Dr. Selby says.
Research funded by PCORI must also be patient-centered. “Applicants must convince us that the research question matters to patients, caregivers and clinicians,” Dr. Selby says. “Are you studying outcomes that matter to patients?”
For example, he notes, a study examining the effects of interventions on patients’ blood-pressure levels would be less likely to receive funding than a study that looks at specific symptoms arising from different blood-pressure treatments. And research on cancer and heart disease should not just look at survival but at quality of life.
“In other words, we want a considered discussion of the outcomes that were selected and why these are important outcomes to patients,” Dr. Selby says.
In addition, applicants must detail how their research will adhere to nearly 50 methodology standards, which range from requirements for formulating research questions to requirements for data integrity and rigorous analysis.
“These are not difficult standards to follow—they are really baseline or floor requirements,” Dr. Selby says. “But certainly, if you transgress those requirements, you aren’t likely to receive funding.”
Another important consideration is that PCORI does not fund studies of cost-effectiveness, Dr. Selby points out.
Dr. Swigris, who previously received funding from the National Institutes of Health, notes that PCORI monitors the progress of studies more closely than the NIH does. “Every six months, you need to send PCORI a progress report,” he says. “So you can’t slack off. I actually like it because I feel the pressure.”
Unlike grants from the NIH, almost all PCORI funding is through contracts, Dr. Selby explains. “With contracts, by their very nature, you set out milestones and deliverables,” he says. “So we do monitor research to make sure that projects are meeting their milestones.
“We also need to make certain that the research we fund is done well and is as timely as possible.”
An additional reason for closely monitoring a study’s progress is so that PCORI can disseminate any important interim findings that emerge, Dr. Selby says.
Most of the awards funded by PCORI provide up to $1.5 million for direct costs. So far, approximately 45% of these 270 or so studies have been randomized controlled trials, while the rest have been observational studies of various designs.
PCORI recently announced a new series of awards for “pragmatic clinical studies” that will provide up to $10 million for direct costs. Most of these large studies will likely be randomized controlled trials involving multiple institutions, Dr. Selby says.
To increase community involvement in research, PCORI has also created smaller “pipeline to proposal” awards, which are designed to foster partnerships among patients, physicians and other health care stakeholders who lack the resources to prepare sophisticated research proposals. Bestowed in three phases—in grants of $15,000, $25,000 and $50,000—these awards provide seed money that PCORI hopes will lead to significant studies.
In addition, PCORI periodically announces awards that are targeted to specific topics, such as asthma or blood-pressure control in minority patients.
Ways to get involved
Osteopathic physicians who don’t have the wherewithal or the inclination to apply for funding can still have an impact on PCORI-funded comparative effectiveness research, Dr. Selby notes. “There are many ways for the osteopathic community to contribute to our efforts,” he says. “For example, we need nonresearch clinicians to serve on our application review panels.
“You can also submit research questions that you think should be studied, and we’ll take them into consideration. And from time to time, physicians are needed to serve on our advisory panels.
“I encourage DOs to visit our website and look for opportunities to get involved with us.”