Orphan Diseases and Drugs

Abstract

An orphan disease refers to a disease with a low prevalence of less than 6.5–10 cases in 10,000 people. The National Organization for Rare Disorders (NORD) and Orphanet are the organizations working for the welfare of people with orphan diseases. Orphan drugs are defined as the drugs used for the diagnosis, prevention, or treatment of orphan diseases. The US-FDA provides a list of orphan drug designations and approvals. Partial orphan drugs are those drugs having both orphan and non-orphan indications. Orphan drug development faces many challenges since pharmaceutical companies are generally reluctant in undertaking drug development for orphan diseases due to their low market value. Thus, in order to promote orphan drug development, various strategies have been tried like the enactment of specific legislations and providing incentives for the development of orphan drugs.