Only a small portion of the population was impacted, which limited study opportunities. Additionally, manufacturers were deterred from addressing unmet needs in this area because of the enormous divergence and variety across the group of disorders, which contributed to the lack of understanding of diseases. However, the development of some expensive orphan treatments, such as the US$ 125,000 per vial spinal muscular atrophy therapy Spinraza, and huge unmet needs have caused manufacturers to focus on Rare Disease Drugs for uncommon diseases.
A small percentage of the population is ever affected by rare diseases, sometimes known as Rare Disease Drugs. Medicines prescribed for the treatment of rare disorders are designated as orphan drugs. An orphan disease, as defined by the U.S. Food & Drug Administration (FDA), is one that affects fewer than 200,000 persons nationwide. Under the Orphan Drugs Act, a producer may ask the FDA to designate a drug as an orphan drug if it is designed to treat a rare disease or condition. The FDA reports that over the past few years, the number of requests for orphan medication designation has consistently climbed.
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