Comparing medical treatments to find the best and the cheapest may be a pillar of U.S. healthcare reform efforts, but very little such research is being done, according to a report published on Tuesday.

Most of the so-called comparative effectiveness research is done at academic institutions or by other noncommercial enterprises, and less than 20 percent examine the safety of treatments, researchers reported in the Journal of the American Medical Association.

Most of the comparative effectiveness studies we reviewed simply tested whether medication 'x' is better than medication 'y,' rather than addressing fundamental questions such as: How can we use this medication more effectively? When is this medication better than surgery? Which among two effective approaches is the safest? said Dr. Danny McCormick of Harvard Medical School in Boston, who led the study.

McCormick and Dr. Michael Hochman of the University of Southern California in Los Angeles chose 328 studies in major medical journals that evaluated drugs.

About a third, 104 of them, compared a drug to something else. Forty-three percent compared one drug to another, 11 percent compared a drug to a non-drug therapy, 15 percent focused on different dosing schedules, 19 percent looked at safety and just 2 percent included an analysis of cost effectiveness.

While most drug trials are sponsored by companies, noncommercial entities paid for 87 percent of the comparative effectiveness studies, Hochman and McCormick found.

Many of our nation's research priorities are driven by the pharmaceutical industry, Hochman said in a statement.

These companies, not surprisingly, focus most of their attention on new therapies.


Healthcare reform is U.S. President Barack Obama's signature political strategy and he has proposed comparative effectiveness research as one big component. Last year Congress approved $1.1 billion for comparative effectiveness research as part of an economic stimulus plan.

Additional information about existing comparative effectiveness research could help guide policy makers as they determine the amount and types of comparative effectiveness research that are most needed, Hochman and McCormick wrote.

In particular, our findings suggest government and noncommercial support should be increased for studies involving nonpharmacologic therapies, for studies comparing different therapeutic strategies, and for studies focusing on the comparative safety and cost of different therapies.

The Food and Drug Administration should require such studies for drug approvals when possible, they added.

In a separate commentary, Dr. Alec O'Connor of the University of Rochester Medical Center in New York agreed.

Given the continued progress of science, approval of a new drug or device implies to physicians and the general public that the product represents an advance over older treatments, O'Connor wrote.

But this is not always true. Recent studies have shown, for instance, that older, cheap drugs such as generic diuretics to treat high blood pressure, or metformin to treat diabetes, work better than newer drugs in many cases.

For example, if a new medication to treat depression is approved based only on placebo comparisons, it's very difficult to know how the new drug compares with the dozens of medications already approved for depression, he added in a statement.

(Editing by Mohammad Zargham)