Above All, Do No Harm
The standard paradigm of doctors, "doing no harm," means that the proof of safety is the first and requisite step in determining whether a treatment should be used. Over and again, new therapies are approved by the US FDA with the assumption by doctors that this means that safety is proven. Unfortunately, the FDA's proof of safety differs from disease to disease and drug to drug, as well it should. A person with a life threatening disease may need to accept some additional risk if the treatment is the only chance for survival, whereas I would rather suffer with a stuffy nose than take a cold medicine that carries a risk for dropping dead suddenly, even if that risk is very unlikely to happen.
As a practical example of this issue, consider the medicine Vytorin, approved by the FDA this month for the treatment of elevated cholesterol levels. Vytorin is a combination of a statin (Zocor) and another cholesterol medicine already on the market called Zetia. It reduces cholesterol levels very well. It reduces cholesterol levels better than either of the components alone. It is not associated with any major risks, at least in preliminary studies. However, the studies have been performed over a short period of time in a relatively small number of people. This means that we don't know with certainty that Vytorin cuts the risk of heart attacks or dying young, the way that a higher dose statin would do (such as Zocor, Lipitor or Pravachol).
More importantly, there is no study that looks at the safety of taking Vytorin or Zetia alone long-term. We just don't know if Vytorin or Zetia is safe for people to take for years. Do I think they are? Yes, I do. But I do not prescribe these medicines to my patients because of two reasons. First, I could be wrong and the medicines may have some idiosyncratic effect on the body that causes liver dysfunction or other complications. Second, we don't know that reducing cholesterol by using Zetia means that the risk of heart attacks, strokes or deaths will be lowered. Merely because we think so does not mean it will be so.
The medical journals document many treatments that seemed to be such sure things in early studies that had no effect in long-term studies, or worse yet, led to a higher risk of death or disability.
Many leading doctors support the usefulness of this drug in the absense of proof of safety. Such an attitude, approach and recommendation is diametrically opposed to a primary focus of the practice of medicine. Several proven tools exist; there is no reason to use one unproven. Such a conservative approach will minimize risk and keep the focus on optimizing quality of life.
