ABSTRACT
Drugs to treat “rare” diseases or the limited drugs available to treat mostly genetic disorders, including Wilson disease, Norrie disease, cystic fibrosis, and arthrogryposis, are known as orphan drugs. In the USA, the National Organization for Rare Disorders (NORD) was formed in 1982 to facilitate the treatment of orphan drugs, whereas, in India, the National Policy for Treatment of Rare Diseases (NPTRD) was formed in 2017 under the directive of the High Court of Delhi. The Orphan Drug Act (ODA) is enforced to achieve drug availability at low cost to people suffering from the orphan diseases, and the company or developer manufacturing the drug gets market control exclusivity for six to seven years. The Committee on Orphan Medicinal Products (COMP) in Europe controls the orphan drugs through Directive 141/2000. In this chapter, we describe the process of orphan drug development and regulatory procedures.
