Often, at scientific conferences, the most important learning happens in the question and answer period.
I spoke at the American Diabetes Association conference earlier this year, presenting results of an observational study we did on medication adherence and diabetes.
We found that if people starting using the online patient portal (sometimes called the personal health record), to order their medication refills, they were more likely to take their medication regularly. Dr. Katherine Newton of Group Health Research Institute spoke before me, describing a randomized study showing that a clinical pharmacist-led blood pressure management program did not lower blood pressure any more than usual care by an outpatient provider.
The first audience comment came from a program officer from the National Heart, Lung, Blood Institute, part of the National Institutes of Health. Program officers are incredibly important because they help set the research priorities for the major funding mechanism for medical research. I will never forget her comment, because it was so strongly worded.
But a New York Times story this week was eye-catching for a different reason: author Gina Kolata mostly assailed Medicare’s researchers for how they’re choosing to do that research.
“Experts say the center is now squandering a crucial opportunity,” Kolata wrote in a front-page article. “Many researchers and economists are disturbed that [CMMI] is not using randomized clinical trials, the rigorous method that is widely considered the gold standard in medical and social science research.”
But many researchers and economists that I talked to at this week’s Academy Health conference say that’s not the case at all. (And some were disturbed to learn that they were supposed to be disturbed.)
“RCTs are helpful in answering narrowly tailored questions,” Harvard’sAshish Jha told me. “Something like—does aspirin reduce 30-day mortality rates for heart attack patients.”
“However, for many interventions, RCTs may be either not feasible or practical.”
“While RCTs may be the gold standard for testing some hypotheses, it is not necessarily the most effective or desirable model for testing all hypotheses,” agrees Piper Su, the Advisory Board’s vice president of health policy.
CMMI’s ambitious goals
On its surface, Kolata’s article is built around a reasonable conclusion: RCTs offer plenty of value in health care, and we’d benefit from more of them.
As Jha alludes to, think of a double-blinded pharmaceutical study where half the participants randomly get a new drug and the other half get a placebo; that’s an RCT.
The famous RAND study that found having health insurance changes patients’ behavior: An RCT.