Abstract

A REVIEW ON RARE DISEASES AND ORPHAN DRUG DEVELOPMENT

Anagha Upasani*, Ashwini Thongire, Shifa Pathan, Harshada Mahajan and Shweta Jadhav

ABSTRACT

Orphan drugs are specialized drug therapy for rare diseases. Rare diseases follow Mendelian pattern of inheritance. The defining criteria of rare disease changes as per the jurisdiction and other different parameters. There are about 5000-8000 diseases identified as rare diseases. Currently more than 3.5 million people suffering from rare type of diseases. Many countries across the globe are providing incentives and other facilities for fast approval from regulatory authorities. New approaches are developing in the field of orphan drugs and rare disease drug development. The Orphan Drug Act has objective to encourage for development of orphan drugs. Universities and research institutes, academic hospitals, pharmaceutical companies play important role in overall development of orphan drugs and its access to patients. But there are several challenges for the process of drug development due to less no. of patients and difficulties in collection of clinical data. In developing and some of the developed countries the treatment of rare disease and access to orphan drugs should be put on agenda because all the patients should have equal rights towards access of medicines.

Keywords: Orphan drugs; Rare disease; Orphan Drug Act; Drug Development.