April 27, 2015

FDA's "Orphan Drug" Designation Warps Medical Research

Joseph V. Gulfo

Visiting Scholar
Summary

Knowing that the FDA is cautious, investors and drug developers are giving the agency what it wants: a growing pipeline of drugs for rare diseases. Even though science is advancing wonderfully in the big diseases as well, why should investors and companies try to develop novel products for major diseases when the FDA is likely to require extremely time-consuming and expensive trials, and may move the goal posts at the end, regardless?

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With Earth Day and Arbor Day now in our rearview mirror, the countdown to a host of other summertime days of celebration such as Memorial Day, Independence Day and Labor Day can begin. This reminds me of when I was 10 years old, and I complained to my father that there is a Mother’s Day, Father’s Day, Grandparents’ Day, Teacher’s Day and Secretary’s Day, but no Child’s Day. My father exclaimed, “Every day is Child’s Day!”

While we focus a good amount of attention on days like this, it’s worth noting that another special day recently passed us by with little fanfare: Did you know that there is a Rare Diseases Day? It’s Feb. 28. (My friend and health care scholar Bob Graboyes of the Mercatus Center quipped that it should be February 29th. Exactly!)

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