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FDA: Invisible Arbiter of What Constitutes Disease

According to Wikipedia, “disease” refers to any condition that causes pain, dysfunction, distress and social problems. (http://en.wikipedia.org/wiki/Disease). What constitutes disease is more varied and changeable than this definition might suggest.

The nature of disease and its constant changes are pertinent to FDA, which often makes decisions on behalf of society that reshape our understanding of disease.

Some diseases are culturally-defined. Homosexuality has long been treated as a disease or medical disorder. Scientific knowledge and societal attitudes have shifted over the last 25 years. Homosexuality is increasingly viewed as a variation on human behavior, rather than a disease or condition. FDA would never consider an “anti-homosexuality pill.”

In the opposite direction, obesity has long been a symbol of wealth and well-being. Today, it is being redefined as a disease, complete with claims that it is an epidemic. FDA decided obesity was a “disease” years ago and has considered a number of “anti-obesity pills.”

Other diseases reflect new thinking about what constitutes illness. Examples would include post-traumatic stress disorder, attention-deficit hyperactivity disorder and restless leg syndrome. FDA has approved drugs that treat each of these.

The Food Drug and Cosmetics Act provide the agency some leeway in how it looks at disease. The term “drug” means, among other things:

  • “articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; and
  • articles (other than food) intended to affect the structure or any function of the body of man or other animals.” (FD&C Act, Section 201(g)(1)).

This dual definition has allowed the agency to finesse the issue of pregnancy. I assume that birth control and fertility drugs are viewed as affecting the structure or function of the body. The agency would not want to claim that pregnancy is a disease or that preventing (or causing) it constitutes disease treatment.

FDA seems to make these judgments without any fanfare. My own benchmark is the formalization of erectile dysfunction (ED) as a disease. I expected FDA to go through some extra, visible steps, to show that it had considered whether ED was a disease for which therapies were appropriate. I did not see this occur during the FDA review or at the time of approval.

The question “is this a disease” doesn’t come up every day at the agency. When FDA does make judgments, it appears embedded in the work rather than a notable event.

One of the great future challenges will be for FDA to decide if “anti-aging” drugs are possible. The answer may be “yes,” pointing to the function and structural changes that are clearly made by aging and the opportunity to stabilize and reverse them. This would allow anti-aging to be a drug’s formal indication for prescribing and would be allowed as a label claim.

If “no,” then sponsors of anti-aging drugs will need to show a direct link between their drug and an aging-related disease, such as diabetes or dementia. My understanding is that most anti-aging pipeline drugs are being developed on the assumption that FDA is not ready to decide and that “anit-aging” may never be an approved indication.

When the aging issue becomes timely, FDA will be there to decide. It will bring more experience to the process than most FDA watchers realize.


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