By Richard Gliklich, MD, President and CEO, Outcome
We all know, broadly speaking, the mission of comparative effectiveness research (CER), now sometimes called patient-centered outcomes research. Such studies should inform clinical and health policy decisions made by physicians, payers, and regulators to help determine treatment guidelines, coverage policies, and the therapeutic value of new therapies relative to standard-of-care in real-world settings.
But dive deeper, and it’s clear there remains an uncomfortable level of confusion as to what CER will actually be used to do. So complicated is CER that even US federal agencies can’t agree on a unifying definition. Indeed, look across the various Health and Human Services websites and related entities such as the Patient-Centered Outcomes Research Institute (PCORI) and you’ll discover slight differences in emphasis that have big potential impact on CER’s implementation.
For example, the U.S. Federal Coordinating Council defines CER as the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in “real world” settings. But the U.S. Agency for Healthcare Research and Quality defines CER as a type of health care research that compares the results of one approach for managing a disease to the results of other approaches – for instance the utility of drug A relative to drug B or procedure X to procedure Y or drug A to procedure X.
And according to the new Patient Centered Outcomes Research Institute Methodology Committee, CER seeks
“to understand and improve the effects of healthcare and prevention services on outcomes important to all persons with disease or at risk for disease considering individual perspectives, needs, preferences, biological, environmental, behavioral, and cultural determinants of health.”Well, that seems to include just about everything.
Certainly it's a lot of high falutin’ language to digest – and for manufacturers hoping to bring new products to market, it’s critical they get fluent in CER as soon as possible. No matter how its defined, CER IS NOT going away.
Investment by the U.S. government in the concept is soaring.The Patient Protection and Affordable Care Act (PPAC) created the Patient Centered Outcomes Research Institute (PCORI) with hundreds of millions of dollars in funding. It is inevitable that the importance of PCORI will grow and its impact on drug and device discovery as well as post-approval monitoring will become more and more apparent.
So if the different definitions spark confusion, we offer this piece of advice. The best way to think about CER is the audience it serves.While classic research questions arise from intellectual curiosity of scientists, CER informs decisions made by a diverse group of stakeholders across the industry – especially regulators, payers, patients and providers.
Thus, this real-world pragmatism changes the way CER questions are defined and answers are pursued. It also means decision makers will accept complementary forms of evidence to bolster their arguments, not just traditional ‘experimental’ studies. These data span the gamut from prospective observational studies to retrospective analyses of existing clinical or administrative data -- and even include sophisticated models based on such data sets.
Still, it’s one thing to have evidence; it’s another to know which evidence is sufficient to answer key questions. As Sir Michael Rawlins, Chairman of the National Institute of Health and Clinical Excellence (NICE) in the United Kingdom has stated, it is clear the traditional evidence hierarchies are limited when it comes to understanding how effectively drugs and devices work in the real-world. Those limitations have sparked working groups, like the longstanding GRADE (formed in 2000) and the relative newcomer GRACE (started in 2007), which aim to redefine what constitutes “good” research based on the quality of the methods and results and contextual matters.
With no ready answers to what constitutes appropriate CER, drug makers need to spend more time earlier in the drug development cycle considering the kinds of evidence they need to gather. That’s especially important given that going forward non-traditional trials will likely be equally important – if not more important – than the randomized double blind placebo controlled studies preferred by regulators for approval.
What it all boils down to is this: CER involves much more than just the research. As important, it is a process that includes setting priorities, generating evidence, synthesizing said evidence, and disseminating it to the right audiences.We’re starting to move down the path, but it’s still going to be a few years before we really get "there."
In January 2011, PCORI formed a methods committee to help industry chart a map through the CER wilderness. The goal? A translation table to help both decision makers and researchers know what types of studies are appropriate for different types of questions. What is the best way to compare a drug to a procedure in urology in the real-world? Or, given the existing evidence, what more information is needed to make a decision about a health intervention?
Undoubtedly, the guidelines won’t be so succinct they can be etched on two tablets; nor will PCORI have Charlton Heston able to lead us out of this information desert into the promised land.
Let’s just hope it doesn’t take the same 40 years it took for the Israelites to find their way out of the desert. With healthcare costs rising and CER the most frequently cited cure to the impending insolvency of Medicare, we’ll need some more timely answers than that.
Founded in 1998, Outcome, a spin-out of a Harvard affiliated research laboratory, is a leading provider for patient registries, studies and technologies for evaluating real-world outcomes. For more information about Outcome, please contact Renee Hurley: firstname.lastname@example.org.