Orphan regulations for orphan drug development in India

D. Saikiran Reddy, T. M. Pramodkumar, Yugendar Reddy, K. Sirisha


Through this review article an attempt has been made to put forward the challenges faced by rare disease drug development
and the current scenario of orphan drug legislations in India. An orphan drug is a pharmaceutical agent that is used to
treat a rare medical condition (viz., glioblastoma multiforme, nocardiosis, Tourette syndrome, etc). Developed countries
such as United States (US), Europe, Japan, and Australia have laid down legal framework for combating rare diseases. A path
breaking legislation was formulated by the US government way back in 1983, known as “Orphan Drugs Act (ODA).” The
key purpose of ODA was to incentivize R and D initiatives for such drugs to treat millions of population suffering from
“orphan diseases.” Though the percentage of patients suffering from “rare diseases” in India is reportedly higher than the
world average, unfortunately even today such cases get little help from our government. Indian government should also
encourage its domestic pharmaceutical industry to get engaged in research for orphan drugs by putting an “ODA” in place
and extending financial support, and regulatory concessions like smaller and shorter clinical trials, without further delay.
Thus, India could well‑demonstrate that the concept of orphan drugs for orphan diseases is really not orphan in India.
Key words: Orphan diseases, orphan drugs act, orphan drug

Full Text:



FDA. Available from: http://www.fda.gov/downloads/ForConsumers/

ConsumerUpdates/ucm107301.pdf. [Last accessed on 2013 Jul 20].

Tapray blog. Available from: http://www.tapanray. i n /


[Last accessed on 2013 May 10].

Pharma Express. The eternal orphan. Available from: http://www.

expresspharmaonline.com/20080831/research01.shtml. [Last accessed

on 2013 Apr 13]. (Accessed on April 2013)

Sharma A, Jacob A, Tandon M, Kumar D. Orphan drug: Development

trends and strategies. J Pharm Bioallied Sci 2010;2:290‑9.

Orphanet. Available from: http://www.orpha.net/consor/cgi‑bin/

Education_AboutOrphanDrugs.php?lng=EN and stapage=ST_


[Last accessed on 2013 May 13].

DOI: http://dx.doi.org/10.22377/ajp.v8i2.350


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