Orphan Drugs anyone?! part 1

Definition: An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

Rare Disease:

In the United States of America, the Rare Disease Act of 2002 defines rare disease strictly according to prevalence, as "any disease or condition that affects less than 200,000 persons in the United States, or about 1 in 1,500 people.

In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan, or about 1 in 2,500 people.

The European Commission on Public Health defines rare diseases as "life-threatening or chronically debilitating diseases which are of such low prevalence (less than 1 in 2,000 people) that special combined efforts are needed to address them.
Conditoins which are statistically rare, but not also life-threatening, chronically debilitating, or adequately treated, are excluded from their definition.

Orphan Drug laws:

The Orphan Drug Act (ODA) of January 1983, was passed in the United States to encourage pharmaceutical companies to develop drugs for a disorder affecting fewer than 200,000 people in the United States and may sell it without competition for seven years and get clinical trial tax incentives.

The European Union (EU) has enacted similar legislation and the orphan drugs are referred to as "orphan medicinal products.". The legislation is administered by the Committee on Orphan Medicinal Products of the European Medicines Agency (EMEA). Orphan drug legislation also exists in Australia and Japan.

Some Orphan Diseases: Glioma, multiple myeloma, cystic fibrosis, phenylketonuria, snake venom and idiopathic thrombocytopenic purpura etc. In the USA, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD).

In my next blog I will deal with the drug products designated as orphan drugs and their development.

References:

1)FDA Consumer. U.S. Food and Drug Administration.http://www.fda.gov/Fdac/features/1999/399_orph.html

2) Pharmacology and Therapeutics by F.S.K Barar, S.Chand publications.