Orphan Drug Essay

Orphan drugs under normal market circumstances would not be produced by the pharmaceutical companies due to the expense incurred and potentiality of financial loss due to low levels of sales. Orphan drugs are created to treat, prevent, and diagnose rare diseases with a prevalence of less than 5 in 10,000 per community. An Orphan medicinal product must show an enhanced therapeutic benefit over existing treatments against rare life threatening or debilitating diseases. An orphan drug is not a rare drug but a rare disease and the drug can be an old drug if you like but can a new drug also. At the moment there are a plethora of old drugs without the advent of a patent to shield it from competitors. The terminology in the US to describe drugs which treat rare diseases is known as Orphan drugs whereas in the EU these drugs are coined rare disease …show more content…
Another special incentive is access to a centralised procedure bestowing marketing authorisation upon the specific sponsor within each member state immediately under Regulation (EC) No 726/2004,5.Since it’s inception the EU Regulation on orphan medicinal products 126 Orphan Drugs have been accepted and 1,329 designations for Orphan Status. In order to take advantage of the aforementioned incentives a designation must be approved which means a drug must be deemed safe and effective with supported scientific data in the form of clinical trials or in vitro data to back up this statement. Applications are ratified by the committee for orphan medicinal products and were accepted by article 3(a) and on grounds of insufficient financial returns. The