Something about Orphan Drugs

Something about Orphan Drugs

The drugs having the Pharmaceutical agent that is capable of treating a rare medical condition or rare disease are referred as Orphan drugs and the rare medical conditions itself being referred as rare diseases.

Orphan disease only reflects a small percentage of population. Mostly orphan diseases are genetic and their symptoms do not appear immediately. 30% of children having the rare diseases will die very early (even before reaching 5th birthday). 1

A disease may be considered as rare in one part of the world, but still common in another country. Every country has slightly different definitions for the Orphan Drugs. Cystic fibrosis, Wilson’s Disease and homozygous familial hypercholesterolemia are some of the Orphan Disease for which the drugs are discovered.

Regulations for Orphan Drugs:-

In Japan revised Orphan drugs regulations were established in 1993 (amendment of the pharmaceutical Affair Act and Drug Fund for Adverse Reactions) and the Intractable Disease Information Centre ( was established in 1997 which provides vast information on rare and Intractable Disease.2

In South Korea, the orphan drug guideline was established in 2003 and the Korean Rare Disease Information Database and Korean Organization for Rare Disease provides vast information on rare disease.
In Taiwan the Rare Disease Control and Orphan Drug Act was established in 2000 which also provides exclusive marketing rights for 10 years.
In US, the Orphan Drug Act (ODA) was passed in Jan. 1983 which encourage pharmaceutical companies to develop Orphan Drugs (21CFR 316.20 and 316.21).1
Rare Diseases Act was signed into law in 2002 to establish the office of rare disease.
The European Union has legislation, regulations (EC) No 141/2000 for Orphan Drugs and European Commission gives marketing exclusively for 10 years in the EU.

Incentives for Orphan Drugs:-

  • Exclusive Marketing Rights
  • Financial Subsidies
  • Fast Track Approval
  • Tax Exemption
  • Protocol assistance and fee waivers



Rare diseases need more attention due to lack of proper diagnosis and treatment. Rare disease are an important public health issue and a challenge to medical care. Currently there are more than 400 Orphan designated Drugs in Clinical trial process.
Majority of the drugs are being developed in the US followed by Europe. Although Orphan disease population are the smallest, the cost per patient is very large which creates more pressure on Pharmaceutical companies to provide budget drug. Other countries like Asian countries should adopt the similar legislations or provide more incentives to encourage pharmaceutical companies to invest money in R & D of Orphan Drugs.3