"Comparative Effectiveness . . . or What Works and What Doesn’t" By Allen Weiss, MD, MBA, President and CEO

Comparative Effectiveness . . . or What Works and What Doesn't

July 1, 2009 - This past February, President Obama signed the American Reinvestment and Recovery Act which included $1.1 billion to support research on the comparative effectiveness of drugs, medical devices, surgical procedures and treatments. The purpose of this comparative-effectiveness research (CER) is to fulfill three important objectives, according to a recent New England Journal of Medicine set of editorials. The three objectives are:

  • Acceleration of research into everyday care.
  • Enhance the opportunities for doctors and patients to define value (balancing benefits with costs).
  • Allow care givers and patients to communicate with researchers and policy makers about clinically important issues.

Along with the above objectives, evidenced based medicine (EBM) has been placed in the forefront of medical care. EBM is the use of proven diagnostic techniques and therapies based on rigorous scientific studies. Most practices in medicine today are supported by some research and are based on what appears to be effective, rather than on double-blind controlled studies. These double-blind controlled studies typically take two groups with comparable characteristics (age, sex, severity of illness, etc.) and give one group the therapy believed to be the best available and the other group the new therapy. Sometimes the new therapy is tested against a placebo to see which is more effective or which has more side effects. This is done because receiving the placebo helps some people but causes side effects in others probably due to psychological reasons. Years ago, in a study to measure the efficacy of aspirin versus placebo in the prevention of heart attacks; strangely, the placebo group had more side effects.

One might also be surprised by the length of time needed to implement healthcare changes that have been proven to save lives; and, conversely, the number of therapies which are commonly used although they have been proven to be ineffective. A Harvard Business Review study showed that it takes 17 years on average to implement changes suggested by evidence based medicine (EBM).

Here are some examples of therapies which still are not universally implemented but have been proven to save lives. The acute treatment of a heart attack has been shown to be best when the heart attack can be stopped within ninety minutes by opening the blood vessel to the heart muscle using direct physical intervention in a Cath Lab (a place where cardiac angiography is performed). This technique which has been in place at NCH since 2000 is still not used in the majority of hospitals in the United States. Other hospitals use a clot buster type of medicine which does not work as well as physically placing a stent in the blocked blood vessel.

An example of a therapy which was never rigorously tested but was used for years has recently been shown not to be effective. One of the recommended therapies for osteoarthritis (the wear and tear type of arthritis) of the knee was washing out debris (lavage) or debridement of the knee joint using arthroscopy. In a 2002 article in the New England Journal of Medicine entitled, “The placebo effect: reduction of pain after knee surgery” by Dr. J. B. Moseley and colleagues, claimed that this technique was subsequently shown to be no better than sham surgery or no treatment at all. Please note: this study was not directed to the effectiveness of total knee surgery or the use of arthroscopy for mechanical derangements in the knee in which replacement or repair with arthroscopy, respectively, does work.

Another example where the jury is still out is low back pain—second only to the common cold as a cause for seeking medical care. Conservative therapy for low back pain, including rest, physical therapy, and medications, was just as effective after one year as surgery in most patients. This observation is according to a 2006 Journal of the American Medical Association article which reported the following conclusion:

“Patients with persistent sciatica from lumbar disk herniation improved in both operated and usual care groups. Those who chose operative intervention reported greater improvements than patients who elected non-operative care. However, non-randomized comparisons of self-reported outcomes are subject to potential confounding—and must be interpreted cautiously.”

The same result was reported in the Journal of the American Academy of Orthopaedic Surgeons in February where the following quote appears: “In most patients with low back pain, symptoms resolve without surgical intervention; physical therapy and nonsteroidal anti-inflammatory drugs are the cornerstones of nonsurgical treatment.”

So we can all appreciate the current thrust to implement comparative-effective research which will both improve quality and decrease cost. As our President has stated, “Rising healthcare costs are the single most-pressing fiscal challenge we face by far” and “If we don't tackle healthcare, then we're going to break the bank.”


Dr. Allen Weiss is CEO & President of the NCH Healthcare System. He is board certified in Internal Medicine, Rheumatology and Geriatrics, and was in private practice in Naples, Florida from 1977 - 2000. Dr. Weiss is active in a variety of professional organizations and boards, and has been published in numerous medical journals, including the American Journal of Medicine and the Journal of Clinical Investigation.