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  1. FDA's Rare Disease Day

Using Economic Analysis to Promote Innovation in the Treatment of Rare Diseases

Kathleen L. Miller, Ph.D.
Economist, Office of Orphan Products Development

Photo of Kathleen Miller, Ph.D.As an economist in the Office of Orphan Products Development, in the Office of the Commissioner, my job is to provide the FDA leadership with economics analyses on matters that affect rare diseases. To do this, I study topics ranging from trends in orphan drug development, to modeling the effects of new and current policies.

My colleagues and I are especially interested in analyzing innovation in pharmaceutical treatments for rare diseases. In recent years, we have seen increases in both the quantity and innovative qualities of new drugs, and we have found that rare disease treatments are leading the way in both areas.

In 2000, only 10% of new drugs approved by the FDA were indicated to treat a rare disease. However, over the past five years, more than 40% of these drugs were for the treatment of a rare disease.

And, even more notable than the quantity of these approvals, is their quality. An important layer of innovation in new drugs is whether they are "first-in-class," which means they use a new pathway for treating a disease.

Prior to 2000, the FDA approved roughly 3 first-in-class new drugs per year for rare diseases. However, since 2011, this number has more than doubled, to an average of over 7 first-in-class approvals per year. Not only do these approvals reflect promising new approaches to the treatment of patients with rare diseases, but many of these drugs were the first ever treatment for the disease.

Working in rare diseases is very meaningful to me. The first time I presented at a rare disease conference, while I was still a student, many patients come up to me and thanked me for the work I was doing. This had never happened to me before at a conference, and it really made a huge impact on me. To have those patients in front of me, thanking me and thinking that my work was valuable, that made me feel like there was a larger, tangible, purpose to my research.

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