#Orphan 2 plus

They are pathologies whose incidence at birth is less than 1 in 2000. Progress has increased, but the 300 orphan drugs and devices approved in the last 25 years are still only a drop in the bucket compared with the many thousands of orphan diseases.Дiseases that manifest in patient populations representing at the maximum 6вЂ“8% of the world population are defined as rare diseases or orphan diseases. Positive developments include formation of the National Organization for Rare Diseases, the Orphan Drug Act, the development of a grant program to fund orphan drug development, the formation of the National Institutes of Health Office of Rare Diseases, and the passage of orphan drug legislation by other countries. But many disincentives are involved, which include career disincentives, lack of funding, and the multiple areas of expertise that are required. Academic physician-scientists have tried to fill this therapy vacuum by working on developing orphan drugs. The orphan diseases are often so rare that a physician may observe only one case a year or less. Actually, the indications of a drug may also be considered as вЂњorphanвЂќ since a substance may be used in the treatment of a frequent disease but may not have been developed for another, more rare indication. Rare diseases in small patient populations thus вЂњorphanedвЂќ by the pharmaceutical industry, having but a few approved drug treatment options available are called вЂњorphan diseases.вЂќДЄ medicinal product designated as an orphan drug is one that has been developed specifically to treat a rare medical condition, the condition itself being referred to as вЂњorphan disease.вЂќ It may be defined as drugs that are not developed by the pharmaceutical industry for economic reasons but which respond to public health need. The spiraling cost of drug development in tune with stringent regulations, coupled with the low return on investment, often tends to discourage pharmaceutical innovators from developing products for extremely small patient populations. Recent developments are seen in orphan drug approval, the various drugs in orphan drug pipeline, and the future prospectives for orphan drugs and diseases.

In our study, we have compared the policies and orphan drug incentives worldwide alongwith the challenges faced by the pharmaceutical companies. 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. Incentives for drug development provided by governments, as well as support from the FDA and EU Commission in special protocols, are a further boost for the companies developing orphan drugs. The new business model of orphan drugs could offer an integrated healthcare solution that enables pharma companies to develop newer areas of therapeutics, diagnosis, treatment, monitoring, and patient support. Orphan drugs may help pharma companies to reduce the impact of revenue loss caused by patent expiries of blockbuster drugs.

#Orphan 2 plus

Therefore, the current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies from the essential medicines to the new business model - niche busters, also called orphan drugs.

Many blockbuster drugs will loose their exclusivity in next 5 years. The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines.