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What Works in Health Care? What Doesn't?

With dozens of treatment options for even the most routine issue, who’s to say which one’s best? Turns out, it’s science.

Physicians have a dizzying array of choices for treating any given illness or condition, and sometimes their training and professional judgment aren’t much help in selecting among the options. Would a compression bandage or a below-the-knee cast be the right choice for that severe ankle sprain? Should a patient with back pain be recommended for surgery, or would it be better to wait and see? Which of the dozen or so available statins is right for a patient with diabetes? Does anyone know?

The answer—to a large degree—has been: not really. Employers with geographically diverse operations can see evidence of this in their claims data—rates for various clinical therapies differ by factors of three, four, or even five from one part of the country to another. The same goes for surgeries, prescriptions, tests, talk therapy, and other interventions.1 So where are employees getting the “right” care and where is the waste?

IMPORTED: __media_BE2C2A1451094DA6A2C7ACD28F141BED_w_133_h_200_as_1.jpg Enter comparative effectiveness research. Comparative effectiveness research is just what it sounds like: the study of what works best. For example, a comparative effectiveness study might compare competing antibiotics, the merits of the 111 different treatments available for autism, or even whether surgery and drug therapy or diet and exercise work best for a particular cardiac condition. Such analyses typically focus on the relative medical benefits, but costs may be considered as well. Is a pricey new statin sufficiently better to justify the cost premium, or could a cheaper, older drug do just as well? More sensitive studies also can help target particular interventions to particular populations. For instance, surgery might be a poor option for back pain for one population, but an ideal choice for another.

Perhaps the best known comparative effectiveness study to date was a 2003 analysis by the National Institutes of Health that found that, for pennies a day, thiazide diuretics were more effective than much more expensive drugs at reducing cardiac risk in older patients.2

Some comparative effectiveness research already takes place in the private sector, though not much compared with the apparent need. The Blue Cross Blue Shield Association’s Technology Evaluation Center produces 20 to 25 new assessments of drugs, devices, and other medical technologies each year. Kaiser Permanente has a similar effort and academic medical centers such as Tufts–New England Medical Center’s Cost-Effectiveness Analysis Registry also do work in this area, but according to recent congressional testimony from Office of Management and Budget Director Peter Orszag, “the private sector will probably not produce as much research on comparative effectiveness as society would value.”

Fortunately, in a boost to such studies, the American Reinvestment and Recovery Act of 2009 appropriated about $1.1 billion to fund comparative effectiveness research from the budgets of three different federal agencies. The Act also established the 15-member Federal Coordinating Council for Comparative Effectiveness Research, a body that will help set priorities and coordinate research efforts.

No one expects that comparative effectiveness research will prove to be a cost control silver bullet, but rather a tool that will help employers, health plans, and the government spend health care dollars more wisely. And while using comparative effectiveness research to guide health care decisions may still be a novel concept in the U.S., it’s old news elsewhere; Canada, Australia, Germany, France, and notably the U.K. all have well-established comparative effectiveness programs tied to their national health care systems. According to many sources, these countries all rank higher than the U.S. in terms of overall health care quality.

It’s no coincidence that the surge in interest in comparative effectiveness research comes just as health care reform is being so hotly debated. Many on Capitol Hill see comparative effectiveness research playing a pivotal role in future payment decisions. For instance, should Medicare continue paying for interventions deemed less effective than available alternatives? What about those deemed not effective at all? Critics worry that research-driven payment rules might lead to impersonal, one-size-fits-all medicine, with only the least expensive therapies approved for reimbursement. But those critics largely equate comparative effectiveness research with cost benefit analysis, which it isn’t. In fact, most comparative effectiveness studies don’t even look at cost issues.

“Comparative effectiveness studies tell you what works and for whom,” said Elliot Fisher, M.D., of Dartmouth Medical School. “You need to know that before you can even start to think about costs, but much more importantly it allows you to think about improving care. That’s what comparative effectiveness research is all about.”

1 Tracking the Care of Patients with Severe Chronic Illness: The Dartmouth Atlas of Health Care 2008
2 Furburg et al., "Major Outcomes in High-Risk Hypertensive Patients Trial," Journal of the American Medical Association, 2002 (288):2981–97.

For more on comparative effectiveness research check out:

  • OMB director Peter Orszag’s 2007 testimony to Congress on the issue.
  • This excellent overview piece from Newsweek.
  • An article in Health Affairs by Gail Wilensky about establishing a center for comparative effectiveness research.
  • A webcast of a Chamber of Commerce presentation, “Comparative Effectiveness: New Solutions to Address Complex Health Issues,” featuring former HHS director Mark McClellan. Also, view the related PowerPoint presentation.
  • This seminal NIH study showing that low-cost diuretics are just as effective (if not more so) at treating high-risk hypertensive patients than much more expensive drugs, "Major Outcomes in High-Risk Hypertensive Patients Randomized to Angiotensin-Converting Enzyme Inhibitor or Calcium Channel Blocker vs. Diuretic: The Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT)," in Journal of the American Medical Association, 2002 (288):2981–97
  • Or this article on autism treatments from, which makes a very clear case for comparative effectiveness research, without even bringing up the topic.

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