REGULATORY REQUIREMENTS OF ORPHAN MEDICINES IN INDIA AS PER CDSCO IN COMPARISON WITH BHUTAN

Dr. Ashok Kumar P.*, Ananya B. S., Anitha A., Harshitha K. A., Sahana M. N. and Kavya T. S.

ABSTRACT

Rare diseases, often referred to as orphan diseases, encompass a diverse group of medical conditions characterized by their low prevalence within a population. In India, a rare disease is defined as a life-threatening or chronically debilitating condition affecting fewer than 5 out of 10,000 individuals. Despite their rarity, collectively, rare diseases affect a significant portion of the global population. These diseases can be genetic, congenital, or acquired and encompass a wide range of disorders, including metabolic diseases, genetic syndromes, autoimmune disorders, and various types of cancers. The rarity of each disease poses unique challenges, including delayed or misdiagnosis, limited treatment options, and a lack of research and development. Orphan medicines, also known as orphan drugs, are pharmaceutical products specifically developed to diagnose, prevent, or treat rare diseases. Given the limited patient populations and often high development costs associated with orphan medicines, regulatory authorities.

Keywords: .