Amidst the health care reform media frenzy, I am surprised by how little press is devoted to comparative effectiveness (CE) research. CE is, quite simply, the comparison of different management options for a given medical condition, such as surgery and drug therapy for the same condition. More than $1 billion of stimulus funding was recently allocated to CE, which is sure to usher a sea-change in the way that physicians on the front lines practice medicine as well as the way that medical devices and other health care products are developed.
One billion dollars is a huge sum of money, but trivial in comparison to the estimated $700 billion each year that currently goes to health-care spending that can’t be shown to lead to better health outcomes, according to the Congressional Budget Office. So last year, Congress introduced legislation to establish a comparative effectiveness program that will attempt to provide evidence to inform not only how clinicians manage their patients, but how they get reimbursed for their work.
CE represents a sweeping attempt at standardization of care. Today, standards differ across regions, hospitals, even individual practitioners. I know this from personal experience, having trained or practiced in three states and several different hospitals, where approaches to even well-defined conditions like fever or head injuries vary greatly.
The seminal Dartmouth Atlas Study found tremendous variability in both treatment choices and health care costs across the country. In one example, the study revealed a fourfold variation in the number of patients receiving a coronary artery bypass graft, correlated with regional per capita concentration of cardiac catheterization labs but not with the number of patients in the region suffering from coronary artery disease. Most importantly, they found that regions with the highest per capita expenditures on health care do not have the best health outcomes. Atul Gawande provides a very readable and informative overview of this phenomenon in his New Yorker article,"The Cost Conundrum."
The implications on medical device, drug, and health care product development are vast. Our industry looks to the FDA to define safety and effectiveness, and it is generally the exception, rather than the rule, to compare products against alternatives other than placebo as part of the regulatory approval process. The costs of conducting additional trials to demonstrate safety and efficacy for a broader set of patients, or against another treatment generally outweighs potential gains. In fact, comparative research carries risk, should the comparison product be found more effective.
But as we all know, the FDA does not determine coverage or payment. As payers and providers embrace CE, there will be greater motivation for drug and device manufacturers generate products that yield substantial health gains, or that substantially lower health costs. It raises the bar for meaningful validation of a product: that it yields greater results for a given condition under given circumstances for a given population.
We cannot lose sight of common sense through a radically empirical approach to medicine and medical product development. Sometimes you don’t need a randomized controlled clinical trial to demonstrate benefit. However, I am hopeful that CE will provide a real sanity check and a return to basics for our medical device industry. It is a call to develop products that truly optimize health outcomes and serve the greater good through demonstrable effect.
