Orphan Drugs Overview and Regulatory review procedure

  • Varshith Kumar Gubyal .
  • Prakash S Goudanavar
  • Suhana Elangoli
  • Akshay Saj


To treat, prevent, or diagnose a rare condition (such as Huntington's disease, myoclonus disease, Tourette syndrome, etc.), a treatment or vaccination is known as an orphan drug. Although the definition of uncommon diseases differs between countries, most take disease incidence, severity, and the availability of alternative therapy choices into account. The laws and policies that each area or nation adopts determine the laws and policies that apply to a rare disease. ODA (Orphan Drug Act, 1983) has been effectively promoting R and D investments to create new pharmaceutical products for the treatment of rare illnesses for the past 40 years. It has been implemented in a number of nations throughout the world (including the USA, Australia, the European Union, Japan, and others). Incidences of certain diseases have been rising faster than the rate at which new medications are discovered and developed. Most notably, it has been emphasised that China and India, the two most populous nations, lack national laws for orphan drugs and rare illnesses, which might have serious detrimental effects for their patient populations with uncommon diseases.

Keywords: Orphan disease, Rare disease, Orphan drugs, Incentives, Marketing exclusivity


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How to Cite
Gubyal, V. K., P. S. Goudanavar, S. Elangoli, and A. Saj. “Orphan Drugs Overview and Regulatory Review Procedure”. International Journal of Drug Regulatory Affairs, Vol. 11, no. 2, June 2023, pp. 86-93, doi:10.22270/ijdra.v11i2.609.