LAUSR

In 1983 through advocacy from the National Organization for Rare Disorders and others, including the actor Jack Klugman and his medical television series, Quincy, M.E., the Orphan Drug Act was passed into law in the United States to facilitate drug development for rare disorders. Prior to this act the limited prevalence of a particular disease served as a barrier to research and development. Using the definition in the Orphan Drug Act, any condition affecting fewer than 200,000 people in the United States is considered rare. Pediatric urology treats a collection of rare diagnoses (spina bifida, bladder exstrophy, prune belly etc) that continue to have research barriers because of their limited prevalence and treatment effects decades into the future. Two articles in The Journal this month highlight these limitations for rare disorders.