Abstract

A REVIEW ON ORPHAN DRUGS- INDIAN PERSPECTIVE

Carolina Nissy Sharon V.*, Bhavya Sri Macharla, M. V. Nagabhushanam, Brahmaiah Bonthagarala, D. Nagarjuna Reddy and G. Ramakrishna

ABSTRACT

WHO defines rare disease as a disease or condition with a prevalence of ≤1/1000 population. Other definitions are diseases affecting <1/2000 population in European union, whereas USFDA defines it as any disorder affecting <200,000 population at a single time point. Ultra rare disease is a disease affecting <2 patients/100,000population. However, rare diseases are indeed not so rare. As most of definitions are based on the prevalence of disease, the orphan disease burden is high in countries with high population. A disease can be rare in a region but may be very common in another region, for example, IgA nephropathy is common in Asia and Africa, but rare in European Union. Lots of issues complicate the drug development process of rare diseases, for example, less understood pathophysiology, lack of validated preclinical models, less research, and lack of standard comparator drug. Clinical issues such as lack of information about natural history of the disease, poorly defined endpoints, poor trial design and inadequate sample size, recruitment problems, lack of well-defined diagnostic criteria, and other issues such as nonexistent comparator drug and funding problems. Although multicentric trials can short out this issue, it has own drawback such as lack of consistency in diagnostic facility in small centers and regional sociocultural variation. Like other drug development, orphan drug developmental is also a costly process. Industries show negligible interest in the development of treatment for rare diseases as there is less return on investment. Mostly, orphan drug research is dependent on government incentives. USFDA, EMEA, Japan, and many other countries offer benefits such as protocol assistance, fast-tract approval, waiver of fees, and marketing exclusivity. In this context, here, we are going to deliberate the different initiatives to accelerate the development of orphan disease research in India.

Keywords: Orphan Drugs, Orphan Diseases.