The not-for-profit organization tasked by the healthcare reform law with promoting comparative effectiveness research has spent the 16 months since its inception in the planning stages, building infrastructure and seeking feedback from clinicians and patients.
But the time has come to get the funds awarded and the research started, said Dr. Joseph Selby, executive director of the Patient-centered Outcomes Research Institute.
“The legislation does not specify a date when funding has to start,” said Selby, referring to the Patient Protection and Affordable Care Act, “but the board and staff of PCORI feel strongly that we need to get funding and research under way. Time is of the essence.”
On Jan. 23, PCORI released a draft copy of its five-item priority list and research agenda, which the organization says it will use to guide its funding decisions.
PCORI received a flood of more than 850 applications for its Pilot Projects Grants Program by the Dec. 1 deadline. The awardees—PCORI says there will be approximately 40—will be announced in May and research will begin right away, Selby said.
The large number of interested applicants is to be expected, said Dr. Michael Steinman, associate professor in the division of geriatrics at the UCSF School of Medicine, and director of comparative effectiveness research at the university's Clinical and Translational Science Institute.
“It's not surprising because this is a major, emerging area of research in an era of shrinking budgets from the National Institutes of Health and other traditional sources of funding,” said Steinman, adding that several UCSF researchers had applied for pilot grants from PCORI.
PCORI's development phase has taken a while, but that makes sense, given the organization's mission, Steinman said. “It's a highly valuable organization that is basically starting from scratch with an entirely new way of doing research,” he said. “The good news is that they're not just accepting by default the traditional ways of approaching research. The bad news is that does take time. I think everyone in the field wants clarity, but we also want to get it right.”
The newly released draft priority list covers five wide-ranging areas: comparisons of different treatments, diagnoses and prevention strategies; improving healthcare systems; communication and dissemination of research; addressing healthcare disparities; and accelerating outcomes research and methodological research.
PCORI arrived at the list by analyzing priorities from government agencies, such as HHS' Agency for Healthcare Research and Quality, as well as those from independent, not-for-profit quality improvement organizations, such as the National Quality Forum.
Those lists contained many of the same target areas, including chronic-disease management, prevention and appropriateness of care, Selby said. PCORI staff took those topics and framed them in a way that would emphasize patient-centeredness and allow for a wide variety of projects, he explained.
For instance, in describing the goal related to healthcare systems improvement, PCORI highlights the need for research that promotes patient self-care, increased access, use of health information technology, care coordination, and new roles for allied health professionals.
“The list is intentionally broad,” Selby said. “We're just getting started and we wanted to leave open the possibility that any compelling research question could be considered and funded. If we narrowed the agenda and said that we only wanted to study these conditions or these interventions, we'd cut ourselves off from large sections of the provider and patient communities.”
The organization's priorities will inevitably become more specific as stakeholders provide feedback and new goals emerge, Selby said.
The research agenda that accompanies the priority list addresses each of the five areas in more detail and provides examples of sample projects that might fit the bill. For instance, under the goal of disparities, PCORI provides a list of subtopics, including research that addresses barriers to care, such as homelessness and unemployment, or research that identifies the best ways to share comparative effectiveness data with different patient populations.
C. Daniel Mullins, a professor of pharmaceutical health services at the University of Maryland School of Pharmacy, Baltimore, is working with PCORI to find ways to best engage patients in comparative effectiveness research. Mullins was awarded a $125,000 contract to study hard-to-reach populations, including minority patients, patients with impairments and those with low socioeconomic status.
Mullins' team will be conducting focus groups with people who meet the “hard-to-reach” definition, and asking their opinions on how research can be made more meaningful.
The results of that project, along with 14 others conducted by researchers across the country, will be used to draft PCORI's methodology report, scheduled to be released in mid-May, Selby said.
“I think what they are doing is terrific,” Mullins said of PCORI. “They're committed to seeking input and making sure that whatever they do is applicable broadly to many patients. What they've just put out is a good starting point, and I'm interested to see how they refine it.”
