‘Comparative effectiveness research’ tackles medicine’s unanswered questions

Nobody familiar with American medical care in the 21st century should be surprised that a 73-year-old woman can be minutes away from getting a painful collapsed vertebra filled with liquid plastic and it’s impossible to say whether the procedure works, or how.

It may be that Marcia Henry could get as much relief from injections of local anesthetic, from physical therapy or just from more time to heal as she will from the $3,137 “vertebroplasty” she’s about to undergo at the University of Virginia Medical Center in Charlottesville.

  • ( PETER CADE / GETTY IMAGES ) -
  • ( DAVID BROWN / THE WASHINGTON POST ) - As interventional radiologist Avery J. Evans, left, looks on, Derek Kreitel, a radiology fellow at the University of Virginia Medical Center checks the placement of a needle in a patient’s vertebra with a fluoroscope, which provides reral-time X-ray video.

( PETER CADE / GETTY IMAGES ) -

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“The studies have been contradictory. Which one trumps which one? We don’t know,” says interventional radiologist Mary E. Jensen as she sits in a dimly lit X-ray viewing room and watches a colleague lay out a tray of instruments in a procedure suite next door. “It leaves the treating physician in a dilemma.”

American medical care is rife with such treatments, whose usefulness is uncertain not just to the doctors who deliver them but also to the patients who receive them.

These days, however, many people are pinning their hopes on “comparative effectiveness research” as way to solve the dilemma of how best to treat this and hundreds of other common problems in day-to-day medicine.

“What’s remarkable is how much we do with so little evidence to support what we do, especially when it comes to the patient right in front of us,” said Harlan Krumholz, a 53-year-old cardiologist and researcher at Yale University.

Comparative effectiveness research goes beyond the basic question — “Is this safe and effective?” — that must be answered before new a new drug or device goes on the market. Instead, this emerging field tries to determine where a drug, a procedure, a test or a therapeutic strategy fits into the world of what’s already available and being used.

Are pregnant substance abusers more likely to get sober if they’re treated as inpatients or outpatients? Which is better for staving off dementia in the elderly, regular exercise or brain-teaser games? What are the best strategies for treating high blood pressure in African Americans? Which is the better way of diagnosing kidney stones in the emergency room, ultrasound or CT scan?

These are among hundreds of questions being addressed by comparative effectiveness research studies now underway and funded by $1.1 billion in the Obama administration’s 2009 economic stimulus package. The purpose isn’t to declare hands-down winners (although that occasionally happens). It’s to provide practical guidance when there’s more than one reasonable option.

“For us, ‘true north’ is really what clinicians and patients need to know to make the best possible decision,” said Carolyn M. Clancy, director of the federal Agency for Healthcare Research and Quality, which this year is spending about $21 million on comparative effectiveness studies.

This has never been a high priority for the country or its scientists.

Only 1.5 percent of money spent on medical research goes to “outcomes research,” of which comparative effectiveness is a sub-category. About 13,000 new clinical studies start up each year; about 112,000 are running now. A meticulous search in 2008 revealed only 689 studies that fit the general description of “comparative effectiveness.” Many experts believe that’s not enough.

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