How might the Orphan Drug Act be involved?
Because of reduced profit potential, the normal drug sponsors are reluctant to invest the time and money necessary to develop treatments in a limited market such as patients with a rare disease. Other potential sponsors, such as research hospitals and universities, often lack the capital and business expertise to develop treatments for limited patient groups. To address this disparity, Congress enacted the Orphan Drug Act, which grants special privileges and marketing incentives to drug sponsors that help develop pharmaceutical treatments for rare diseases. The Orphan Drug Act defines orphan products as ones used to treat diseases or conditions affecting fewer than 200,000 patients in the United States. Of course, there are far fewer than 200,00 FRDA patients in the country. Typically, FDA approves orphan products much more quickly than products aimed at potentially huge markets. Nonetheless, some in the orphan diseases community say FDA approval still takes too long. The agency may ai
