Orphan Drugs: Advances in the Treatment of Rare Diseases

ABSTRACTRare diseases are a major public health problem and public health challenge. Orphan drugs are medicines intended to diagnose, prevent, or treat life-threatening rare diseases. In a country like India, which is already struggling to provide basic healthcare facilities to its large population, it becomes very difficult for people living with rare diseases to access treatment options. The orphan drug legislation provides the pharmaceutical industry with a set of incentives and conditions to develop drugs for the treatment of rare diseases. The aim of this article is to describe orphan drugs for the treatment of rare diseases, Legislation, Indian Perspectives, Access to Orphan Medicines, Designation, Progress and Sales. Orphan drugs can help pharmaceutical companies reduce the impact of lost sales caused by blockbuster drug patent expirations. In our study we compare essential drugs with orphan drugs and examine approaches and future prospects in the development of orphan drugs.  Keywords: Rare Diseases, Orphan Drugs, Essential Medicines, Orphan Drug Act, NPRD, Advancements in Research.