Regulators Will Likely Continue to Make Development Difficult, Even After Aging is Recognized as a Medical Condition

It is the nature of regulators at the FDA to aim for zero risk at any cost, and this is particularly apparent in the case of preventative therapies intended to be deployed widely in comparatively healthy people. Absent unusual political pressure, any number of ongoing deaths while therapies are assessed is treated as an acceptable cost to avoid even small numbers of deaths that may occur due to use of a new therapy. Thus even after aging is recognized as a medical condition by regulators, it is likely that they will make it too expensive to assess potential therapies. Instead, companies will gain clinical approval for treatment of specific age-related conditions, and widespread off-label use will become a political battle, clinics and physicians versus regulators. The result of this will be the usual consequence of heavy regulation: a dramatic slowing of progress, and increased cost to patients.

There is a major challenge for discovering and developing anti-aging drugs. How does one design a clinical trial to convince patients, physicians, payers and, especially, the FDA that a drug actually works? To do this, a company would have to prove that its drug extends lives. You can't test such a drug in young or even middle-aged people, as these groups still have considerable life left - assuming a life expectancy of 80 years. Thus, you would probably need to study the drug in healthy 70-year-olds (with a placebo control group as comparator) and then follow these subjects for a decade or more to see if those on the drug live meaningfully longer than those in the placebo group. In order to see a statistically meaningful longevity effect, however, the study would need a minimum of 20,000 subjects. This "outcomes" trial would be similar to what is now done for new drugs to treat heart disease, in which a drug's efficacy is determined by whether it reduced heart attacks and strokes. Such studies are not cheap. The costs can be on the order of $2 billion.

The FDA would likely set a very high bar for safety and efficacy for such a study. Unlike studying patients with heart disease, here you would be testing your drug on 70-year-olds who are relatively healthy. Yet, these patients are entering a decade when they become more susceptible to various cancers, neurological disorders, cardiovascular diseases, etc. You would have to be certain that your drug was no different from placebo when studying these safety parameters. The FDA's caution would be well justified. Such a drug would be in tremendous demand should it actually work. Should such a drug be approved and then later shown to increase major side effects, the fallout would be unprecedented.

Given these enormous challenges, why would anyone actually engage in research and development to produce anti-aging drugs? After all, the people investing in these field are accomplished scientists and investors. They are aware of these challenges. Despite the hype around extending the human life span by 10 to 20 years, these companies will not look to conduct life extension studies right out of the gate. Rather, the first drugs will be tested against age-related diseases.