Vol. 2, Issue 8 (2013)

Author(s):
S Ramachandran*, D N Anusha, S P Sahoo

Abstract:
Pharmaceutical (drug) and biotech companies are constantly researching and developing new medications to treat medical conditions, and new drugs come on the market frequently. People who have rare diseases or disorders, however, have not had as much research attention in past decades. This is because their numbers are small, often having such low prevalence and therefore the potential market for new drugs to treat them is also small. Orphan drugs are medicinal products intended for the diagnosis, prevention or treatment of orphan diseases. Orphan diseases are a spectrum of medical conditions with very different etiologies, the common denominator being the infrequency of their occurrence in the population. The new business model of orphan drugs could offer an integrated healthcare solution that enables pharmaceutical companies to develop newer areas of therapeutics, diagnosis, treatment, monitoring, and patient support. Incentives for drug development provided by governments, as well as support from the FDA and national organizations in special protocols are a further boost for the companies developing orphan drugs. Although there may still be challenges ahead for the pharmaceutical industry, orphan drugs seem to offer the key to recovery and stability within the market. The aim of this article is to review on the concept of orphan diseases in various countries with their status and some orphan diseases, affecting commonly among the rarity.