• Vikaas Budhwar
  • Ajay K. Singh
  • Manjusha Choudhary


Orphan drugs are the drugs and natural products used in treatment, diagnosis, or prevention of rare disease. The orphan drug regulation varies in different countries. Initially a guideline (Orphan Drug Act) for orphan drugs has been made in the USA. Exemption from application filing fees, tax credits for clinical research, marketing exclusivity for definite period of time and grant for phase I/II clinical trials are among the benefits given to the manufacturer of orphan drugs. As a result of these benefits number of drugs for the treatment of rare disease has been increased significantly in the last few decades. The orphan drug program can help the pharmaceutical companies in earning profit and recovering their investment even with small patient population in the developed countries. In 2001 a conference was held by the Indian Drugs Manufactures Association (IDMA) where a group of pharmacologists requested the Indian Government to establish the Orphan Drug Act in India. Indian rare disease population depends on developed countries approved orphan drugs because regulation for the orphan drugs has not been made by the Indian government. Developing countries are also feeling they should do something to promote the research and manufacturing of orphan drugs. The scarcity of regulation of orphan drugs adversely affects the economic growth of Indian medicinal industries. The present study reviews the orphan drug regulations in US, EU and India and raises some of the recent issues concerned to their regulation.

Keywords: Regulations of orphan drugs, Orphan Drug Act, Orphan drug designation, Orphan drug Status, Orphan drug act amendments, COMP, NORD, OOPD.


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How to Cite
Budhwar, V., A. K. Singh, and M. Choudhary. “REGULATIONS OF ORPHAN DRUGS IN USA, EU AND INDIA-A COMPARATIVE STUDY”. International Journal of Drug Regulatory Affairs, Vol. 4, no. 3, Feb. 2018, pp. 30-37, doi:10.22270/ijdra.v4i3.187.