Orphan drugs are drugs that treat, prevent or diagnose a rare disease or are not commercially viable for marketing to treat, prevent or diagnose another condition. Australia and the United States have orphan drug statuses, the United Kingdom does not have orphan drugs, per se, but it has something similar, the Early Access to Medicines Scheme (EAMS) which was launched 7 April 2014. Under the EAMS the MHRA will give an opinion based on the available evidence regarding a medicine for which there is a clear unmet medical need for patients. This medical need must involve the treatment of a life-threatening and/or debilitating medical condition. In the European Union (EU) the European Medicines Agency (EMA) also has orphan drugs designations.
- ↑ "Orphan drugs". Therapeutic Goods Administration. Canberra, Australia: Therapeutic Goods Administration. 31 October 2014. Retrieved 31 October 2014.
- ↑ "Developing Products for Rare Diseases & Conditions". FDA. Silver Spring, USA: Food and Drug Administration. 1 October 2014. Retrieved 31 October 2014.
- ↑ 3.0 3.1 "Early Access to Medicines Scheme". MHRA. Medicines and Healthcare Products Regulatory Agency. 10 October 2014. Retrieved 31 October 2014.
- ↑ "Orphan designation". EMA. London, UK: European Medicines Agency. 2014. Retrieved 31 October 2014.