The acceleration of clinical trials studying rare diseases over the past 3 decades has occurred largely because of the Orphan Drug Act, which was enacted in 1983 as a result of patient advocacy groups working with congressional sponsors.1 This act facilitated the development and commercialization of products (drugs, vaccines, and diagnostic agents) to treat populations with rare diseases, defined as those affecting fewer than 200 000 US residents, or those without reasonable expectation that drug development costs will be recoverable by US sales. The act has resulted in more than 3900 orphan product designation requests to the US Food and Drug Administration (FDA) with the approval of more than 450 products to treat approximately 250 rare disorders, of which approximately one-quarter in the past decade have been for pediatric diseases.2,3
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