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Rare diseases offer great potential value to medical research
By Thomas Maeder
Disruptive Women in Health Care
The only people interested in rare diseases are those who have them, and that’s not a lot,” an editor once told me when rejecting my proposal for a book on orphan diseases.
In reality, nearly thirty million Americans – one in ten – suffer from some 7,000 rare diseases, making them collectively very common indeed. Though they differ wildly in their causes and manifestations, they share many characteristics in terms of the uncertainty, isolation, and costs imposed upon sufferers and their families, the challenges they pose for our health care system and sense of social responsibility, and their potential value to medical research.
In the United States, orphan diseases were defined by the Orphan Drug Act of 1983 as those that affect fewer than 200,000 people domestically.
