A Thought About Testing

Source: "Medication Under a Microscope: Studies Raise Questions About Drugs' Efficacy Against Disease" by Rob Stein, The Washington Post, February 19, 2008.

Many studies of pharmaceutical efficacy focus on an intermediate outcome rather than a health outcome. Does a drug for diabetics control blood sugar, rather than does it reduce the probability of complications of diabetes? Does a drug reduce cholesterol, rather than does it reduce the probability of cardiovascular disease?

This approach makes sense if and only if the intermediate indicators is really implicated as a causal factor of the negative health outcome. It is much stronger if there exists a tracking system that allows public health officials to track whether, when approved and used in the population, the drug is associated with better health, or whether there is too high a rate of side effects.

One aspect of the decision as to whether this process is acceptable is cost-effectiveness. If one were using a comparable technique for quality control of a pot manufacturing line (e.g. testing the quality of clay being used to make the pots rather than the strength of the pots coming off the line) that would be enough. Who cares if a few pots break?

In the case of pharmaceuticals, one wants to balance the health risks to the subjects of the research with the health risks to patients who will take the drug if it is approved.

I have hope that we will do much better in the future in figuring out which patients are helped and which are not helped by drugs. There seems to be a lot of interaction of the pharmaceutical and the genetics and epigenetics of the patients. So too, there is a lot of placebo effect. Perhaps as we know more, we can generate information more effectively and more safely.