European Journal of Biomedical and Pharmaceutical Sciences

ORPHAN DRUGS-CREATING RARE DISEASES, A PUBLIC-HEALTH AND STUDY PRIORITY

Shinde Supriya*, Shinde Vivek, Pandya Sudhir and Pawar Pooja

ABSTRACT

Research in rare diseases has promoted considerably toward the existing understanding in the pathophysiology of the common diseases. However, medical requirements of patients with rare diseases have always been mistreated or ignored by the society and pharmaceutical industries based on their small figures and unprofitableness. The Orphan Drug Act (1983) was the initial serious effort to address the unmet medical requirements for patients with rare diseases and to deliver incentive for the pharmaceutical industry to encourage orphan drug development. The procedure of drug expansion for rare diseases is not unlike from common diseases but includes momentous cost and infrastructure. The drug-approving authority must work-out their scientific decision and ensure due flexibility while evaluating data at various stages of orphan drug development. The emergence of patent cliff combined with the administration incentives led the pharmaceutical industry to understand the good commercial scenarios in developing an orphan drug despite the small market size. Really, many drugs that were given orphan designation completed up being blockbusters, the present review scientifically examined the tasks associated with living with a rare disorder in grown-ups. Results were categorized according to three areas: consequences of living with a rare disorder, social aspects of living with a rare disorder, and Indian scenario of orphan drugs.

Keywords: Orphan, Orphan drug act, Clinical study, Repurposed drugs, Psychological & social impact.