A study of Regulatory aspects on Registration of Orphan Drugs in US and EU

  • S. Vasantha Mounika
  • P. Ramya
  • M.S.L. Divya
  • S. Naga Swaroopa
  • K. Venkateswara Raju PROFESSOR
  • N.L. Prasanthi


The medicinal products used in the treatment, prevention, and diagnosis of rare disease like Myoclonus disease, Huntington's disease, Tourette syndrome are called as Orphan drugs, when it comes to defining rare disease and approving them as “Orphan designations.” The Criteria varies from country to country based on severity, prevalence of the disease and alternative therapies available.

In 1983, the US government brought the Orphan Drug Act to facilitate and encourage pharmaceutical company to develop orphan drugs without which they show reduced interest in the research and development of these drugs. The reason being their low market value and sales of orphan a drug is so miniscule that it will not enable them to even recover a fraction of the investment made in them.

After the inception of “Orphan Drugs” act 4500 orphan designations have been approved, 730 biologic drug products have been approved to treat 250 Orphan designations.

In the past decades, orphan designations have doubled in number when compared to its preceding decade but only 1/5th of the developed medications to treat them and received regulatory approval.

Keywords: Orphan drugs, rare disease, orphan designations, world health organization, food and drug administration, European medicinal agency


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How to Cite
S. Vasantha Mounika, P. Ramya, M.S.L. Divya, S. Naga Swaroopa, K. V. Raju, and N.L. Prasanthi. “A Study of Regulatory Aspects on Registration of Orphan Drugs in US and EU”. International Journal of Drug Regulatory Affairs, Vol. 10, no. 4, Dec. 2022, pp. 45-52, doi:10.22270/ijdra.v10i4.563.