The Patient-Centered Outcomes Research Institute (PCORI) — an organization established by the US government to enable research on the comparative effectiveness of clinical therapies — has announced its first batch of primary-research grant programmes at a meeting of its governing board in Denver, Colorado.
“Today marks a major milestone in our work as we build a portfolio of patient-centered research that will provide patients and those who care for them better information about health care decisions they face,” said Joe Selby, PCORI executive director, in a statement. “Our funding announcements reflect PCORI’s commitment to a patient-centered research agenda, emphasizing the inclusion of patients and caregivers at all stages of the research.”
In practical terms, the announcement means that PCORI, which was established in 2010 as part of US President Barack Obama’s sweeping health-care reform law, will distribute some US$96 million for research projects across four categories: decisions patients must make on prevention, diagnosis, and treatment options; improvement of health-care systems; communication and dissemination; and efforts to address health disparities. An additional $24-million grant programme that will focus on accelerating patient-centred and methodological research is slated for release this summer.
“We believe there’s a pent up demand for this kind of research, and we’re very excited to see what kind of response we get,” Selby told Nature.
The goal of comparing prevention and treatment options for patients (or groups of patients) is to help them to make more informed health-care decisions, resulting in better outcomes and greater efficiencies. “The question for the patient is often not whether the twelfth drug down the line is better than the placebo,” says Michael Steinman, director of comparative effectiveness research at the Clinical and Translational Science Institute at the University of California, San Francisco. Instead, says Steinman, it is about figuring out which treatment option is best and which is best for the patient. “Traditional research often doesn’t address those questions,” he adds.
The grants also come with the unique stipulation that patients be part of the project team, along with researchers and other stakeholders. This category could include family caregivers, parents, or health-care system administrators, depending on the scope. “We think that will help ensure that we’re getting the questions right and that we’re picking the right outcomes to study — the ones that patients care about,” says Selby. These outcomes could range from survival to quality of life and the ability to function or continue to work amid illness. As Selby puts it, the institute wants to look beyond the simple result of a blood test.
“Rather than specify very discrete questions, we created a framework around the kind of questions that we wanted answered to see what teams around the country will come up with in terms of projects that will have the best impact for patients,” says Harlan Krumholz, a member of PCORI board of governors and a cardiologist at the Yale University School of Medicine in New Haven, Connecticut.
“It’s a great idea. No one knows exactly how to do this yet,” says Steinman. “A lot of the work that PCORI will do is in figuring out how to do it — how best do you engage stakeholders in the research process, in addition to the narrow scientific questions.” Steinman notes that there will be hiccups along the way, but PCORI is “carving out its niche”.
Because PCORI is funded by the 2010 Patient Protection and Affordable Care Act, its fate is now hanging in the balance as the US Supreme Court ponders challenges to the larger law. The court is expected to issue its decision some time next month. But until then, both Krumholz and Selby say that all PCORI can do is press on. “It’s our responsibility to keep going until someone tells us otherwise,” says Krumholz.