In recognition of National Rare disease Day I biked through the part of Arlington where my aunt Marion and Uncle Si used to live. A little bit on Aunt Marion’s connection to rare diseases:
“In the 1970s, the FDA began to investigate what it saw as a gap in the development of therapies for certain patient populations. In June 1979, an interagency taskforce issued a report to the Secretary of Health, Education, and Welfare (predecessor to Health and Human Services) addressing the lack of development of “significant drugs of limited commercial value.” The taskforce found that many drugs essential for diagnosis or treatment of “rare diseases” were not available mainly because “research, development and production are deemed too expensive relative to expected economic return.”
The taskforce on significant drugs of limited commercial value was chaired by FDA’s Associate Director for New Drug Evaluation, Marion J. Finkel, M.D. After publication of the report, Dr. Finkel was named as FDA’s first Director of Orphan Products Development in 1982, and, in this position, she played an instrumental role in FDA’s implementation of the ODA. Additionally, it was Dr. Finkel who championed use of the term “orphan” exclusively for therapies for rare diseases. Accordingly, even though she did not coin the term, we have Dr. Finkel to thank for our use today of the term “orphan.” Additionally, we can also be grateful that her doing so moved us away from the less euphonious term: “significant drugs of limited commercial value” (imagine a law entitled the “Significant Drugs of Limited Commercial Value Act of 1983”!).”
Surce: http://www.fdalawblog.net/2015/02/how-orphan-drugs-came-to-be-called-orphan/
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Dr. Marion Finkel
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At East West Coffee (photo courtesy of Nadine)
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