2/15/2010

Last night I saw a TV ad for a device that claimed to be “clinically proven” to help back pain. Because of my own back problems it caught my attention. Know what? I couldn’t find any information about their clinical trial. Not only that, but the treatment isn’t FDA approved. So, to me their claim is meaningless.

How do we know if a medical device (or drug) is effective? By testing it in a well-designed clinical trial. Okay, so what’s a clinical trial? It’s a scientific experiment designed to show that the new treatment is more effective than what is available.

Let’s say hypothetical company, XYZ Inc, wants FDA approval for a whiz bang therapy for back pain. They must first document all previous science supporting that their drug has a reasonable chance to be effective. This usually includes evidence from laboratory animals. The FDA may then allow the company to conduct a small (feasibility) study on limited number of people to show that the treatment is safe. Because feasibility studies are typically small, usually they are not sufficient to prove efficacy. If the treatment appears safe, the next step is a larger, pivotal study to prove efficacy.

Why do patients agree to be experimented upon? Is it altruism? No. It’s because they hope the new treatment offers more benefit than what is already available.

Implicit in this agreement is the assumption the volunteers are completely informed of the risks. But this hasn’t always been the case. Between 1932 and 1972 an experiment in Tuskegee, Alabama purposely withheld treatment from African-Americans infected with syphilis in order to document the disease’s natural progression. Even worse, the subjects were never informed of the risks. Since then, sweeping changes in medical research have occurred.

On July 12, 1974 the National Research Act was signed into law. In 1979, after the Belmont Report summarized guidelines for the ethics of human experimentation, the Office for Human Research Protections (OHRP) was established. Now, human experimentation is carefully monitored by special review boards.

Occasionally I hear complaints about the glacial speed of the FDA. However, the agency has provisions to fast-track new therapies that address problems which have no other effective treatment. Balancing clinical need against due diligence is sometimes difficult. I can remember the FDA being criticized for not releasing the drug thalidomide to treat morning sickness in pregnant women when it had already been approved in Europe and Canada. It was then determined to cause severe birth defects.