Some 25 years ago, when New York State was considering epidemiological studies of the contaminated Love Canal neighborhood in Niagara Falls, state scientists frequently noted that the population of that small area was too statistically insignificant to link cancer rates among former residents to the hazardous waste in their backyards.
Indeed, the scientific world has long preached what the managed care world is practicing via outcomes studies: You need enough population, i.e., people or patients, to make an informed decision.
That was borne out in a recent 450-patient study in the New England Journal of Medicine, which reported on the results of a clinical trial that implanted wire mesh stents into the brain arteries of patients who were at high risk for a repeat stroke. The compassionate use of the stents was approved by the federal Food and Drug Administration after a 45-patient trial in 2005.
The NEJM study found patients who received drugs and underwent lifestyle modification did much better than those implanted with the stents. To wit: the 30-day rate of stroke or death was 14.7 percent in the stent group, as compared to 5.8 percent in the drug management group. The study concluded that aggressive medical management was superior to the stents for two primary reasons: The risk of early stroke after stent implantation was high, and the risk of stroke with aggressive medical therapy alone was lower than expected.
Comparative effectiveness research has been on the minds of healthcare stakeholders in a big way since President Obama set aside $1 billion for it in his 2009 stimulus package; CER was then included in the healthcare reform bill. At a 2010 managed care contracting conference, a statement was made that the pharmaceutical industry was scared to death of CER. Since health information technology is designed to act as a measurement the link between evidence-based medicine, HIT and CER especially when using large population data sets was said to have the potential of rendering clinical trials unnecessary.
Other naysayers think it has the potential of tripping up the booming personalized medicine movement, which to undoubtedly oversimplify it a bit seeks to customize treatments to a person's genetics. They say the conclusions drawn by CER, by virtue of using data and groups of people, are generalizations. And the public itself worries, according to a 2010 brief in Health Affairs, that CER will limit healthcare choices and lead to restrictive reimbursement decisions.
Yet the NEJM study would seem to indicate the value and logic of numbers. Take ten times as many people as the original 45, and you get entirely different results. Does that mean that everyone in the new study had a negative result from an implanted brain stent. Probably not. But it does give patients about to undergo a risky procedure some better information from which to make a life-or-death decision. And it may just lead to reduced costs and better medicine down the road.