The Patient-Centered Outcomes Research Institute (PCORI) is launching new programs that are expected to encourage more analysis of the effectiveness of specific drugs and medical products, while also building infrastructure that could support clinical research on biopharmaceutical products. Since it was established by the 2010 health reform legislation, PCORI has devoted much of its resources and energy to building institutional infrastructure and capacity for evaluating research proposals, for establishing standards for comparative effectiveness research (CER), and for communicating the results of funded research. Now, with its annual budget expanding to $650 million this year, PCORI executive director Joe Selby says that the Institute is poised to invest more in assessing and comparing important medical products and procedures.
One initiative is PCORI’s “pragmatic clinical trials” program, which is slated to fund a handful of large simple studies designed to determine the risks, benefits, and costs of an intervention as used in clinical practice under real-life conditions. PCORI plans to award up to $15 million to support six-to-nine projects that will run for three-to-five years, and compare at least two approaches considered effective in treating a clinical conditions, including drugs, devices and medical procedures. The plan is to answer important research questions that don’t lend themselves to analysis in randomized clinical trials or larger observational studies. More broadly, these longer-term studies will produce meaningful outcomes on mortality, functional status, quality of life and general symptoms. Letters of intent are due in March, and full applications in August.
New research network
Some of these large, multi-year studies will be conducted by research organizations participating in the National Patient-Centered Clinical Research Network (PCORnet). This new organization consists of 11 Clinical Data Research Networks (CDRNs) based in hospitals and health care systems, plus 18 Patient-Powered Research Networks (PPRNs) formed by patient groups to help develop treatments for particular conditions. PCORI is providing some $90 million to support the network, which will establish research sites able to ramp up quickly to launch clinical trials. The process of forming this broad network will necessarily formulate standards needed for compiling and collecting clinical data across the various networks and address a range of operational, scientific, and ethical issues important for academic, public, and commercial clinical research. This includes how researchers based in health systems should deal with patients and providers, determining if a research project involves “minimal risk,” managing IRB oversight, and clarifying options for randomization.
PCORnet will test its capabilities in coming months by addressing questions related to obesity treatment, such as which antipsychotics are most associated with weight gain; which types of bariatric surgery produce the best outcomes with least cost and risk; and what determines variation in uptake of new weight-loss drugs. Eventually the network could be used to study experimental therapies, observes Robert Califf, Vice Chancellor for Clinical Research at the Duke University Medical Center, who regards PCORnet as an opportunity for the clinical research enterprise to overcome some of the problems that make clinical trials very slow and costly. National Institutes of Health Director Francis Collins similarly envisions that NIH-funded researchers will be able to access patient databases and clinical trial infrastructure through PCORnet to test treatments and new cures and help build “a solid foundation for personalized medicine.”