The US' Patient-Centered Outcomes Research Institute (PCORI), a budding comparative effectiveness research (CER) organization formed under the Patient Protection and Affordable Care Act (PPACA) of 2010, has announced its plans to spend as much as $68 million to develop a new clinical research network that it says will bolster the US' capacity to compare treatments.
PCORI's 2010 founding under the PPACA-better known as "Obamacare" by some-was rife with controversy.
The legislation called for an entity that could, somewhat like the US Preventative Services Task Force (USPSTF), issue treatment recommendations based on comparative effectiveness research. But because some legislators feared that PCORI could eventually become like the UK's National Institute of Health and Care Excellence (NICE), which acts as a CER body and issues rejections for medicines it feels are too costly, PCORI is notably banned from issuing formal recommendations or assessing quality-adjusted life years (QALYs).
While those restrictions are significantly limiting for the agency-the agency can gather evidence, but can't make conclusions from it-PCORI s advocates hope that others, and particularly doctors, will be able to leverage its research to make informed choices about which treatments to use.
The source of the organization's money is also relatively novel. As of 2012, all insured individuals are being assessed a $1 fee to fund the agency's research under the theory that health plans will be the ones to benefit the most from the use of intelligence healthcare practices.
The agency recently put out a call for experts to staff its advisory committees, which are set to deal with patient engagement, assessment of prevention, diagnosis and treatment options, improving healthcare systems, and addressing healthcare disparities. With just 84 spots open for members, PCORI said it had already received 1,295 applications-a possible reflection of the considerable prestige and influence the agency might one day have.
Uncovering Industry Biases?
Before now, PCORI's actions have been relatively measured. In January 2012, for example, the agency announced it would fund 40 grants to the tune of $26 million in the hopes of laying the groundwork for future, larger CER efforts. PCORI's COO, Anne Beal, told Kaiser Health News that she hoped the agency could become the "go-to place for patients and, frankly, even for their providers" over the next decade.
For the pharmaceutical and medical device industries, that means their products, which have historically only been subject to head-to-head comparisons when the companies decide it is in their best interest to make those comparisons, will likely be subject to research to determine which products get the best results.
And PCORI has made it clear that it thinks industry-run trials might be rife with biases that will only be uncovered with the help of robust, independent research.
"A pharmaceutical company that conducts studies of its own products will normally choose whom to study, what to compare, and what outcomes to measure so as to support an application for regulatory approval and ultimately success in the marketplace," explained PCORI in a July 2012 draft report. "As a result, the apparent efficacy or safety of a medical treatment may depend on who is studying it and what they have to gain or lose by the results."
This misalignment of incentives can have negative effects for not only patients, but for regulatory agencies and industry as well, argued PCORI. "Sometimes, rather than performing a comparison to the best alternative treatment to see which is better, investigators may choose, and sometimes regulatory agencies encourage, a comparison to a placebo, or an inferior alternative."
"Sometimes investigators are less inclined to publish results when the study shows no difference," continued PCORI. "Failure to fully publish the results of research also undermines trust and can create a false impression of the effectiveness and safety of treatments." This, in turn, can hurt regulatory authorities-as well as industry-by creating a perception that research is rigged from the start, and that regulatory assessments of healthcare products are not to be trusted.
A large part of its research, it said, will be working on "identifying the key problems in existing research from the perspectives of the patient and of … other stakeholders."
A New Trials Network
But if that July 2012 report dealt with hypothetical approaches, a new announcement made on 23 April 2013 shows that PCORI is serious about undertaking major research to accomplish its goals. Under two separate funding announcements, PCORI will provide up to $68 million to support a "National Patient-Centered Clinical Research Network (NPCCRN)."
Such a network would be used as the basic infrastructure to conduct PCORI's CER research initiatives, said Joe Selby, PCORI's executive director. "A national data-rich infrastructure that advances high-quality, efficient CER will benefit all Americans," he said in a statement.
Those two funding announcements will go toward two separate elements of the NPCCRN: Clinical Data Research Networks (CDRNs) and Patient-Powered Research Networks (PPRNs).
"Together, PCORI believes, these initiatives can unite patients, researchers and healthcare systems to support efficient, effective observational and interventional studies with active participation from broad patient populations," PCORI said in its funding announcement.
At least eight CDRNs will receive funding totaling $56 million, while PPRNs will get $12 million in support of up to 18 "new or existing" entities that can build a "reusable, scalable and sustainable research network."
PCORI explained that both systems are expected to bring together clinicians and patients to play "key roles in governing the direction and uses of the networks … and that the interests of patients will be central to decision-making about the network's structure, function and uses."
It is envisioned by PCORI that eventually the two systems-CDRNs and PPRNs-will converge and become more of a single entity.
PCORI: Patient-Centered Outcomes Research Institute to Invest Up to $68 Million to Develop a National Patient-Centered Clinical Research Network