REGULATIONS OF ORPHAN DRUGS IN USA, EU AND INDIA-A COMPARATIVE STUDY
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.
2. European Medicine Agency [Internet]. Medicines for Rare Diseases: 3–5; 2016 [cited 2016 Apr 13]. Available from:
3. Engel PA, Bagal S, Broback M, Boice N. The Need for Stronger Educational Initiatives for Physicians. Journal of Rare Disorder. 2013; 1(2):1–15.
4. Hall AK and Carlson MR. The Current Status of Orphan Drug Development in Europe and the US. International Journal of Rare Disorder Research. 2014; 3(1): 1–7.
5. Orphan Drug Report [Internet]. Welcome to the Evaluate Pharma Orphan Drug Report. [updated 2015 oct 30; cited 2016 Apr 3] Available from: http://www.evaluategroup.Com/public/reports/EvaluatePharma-Orphan-Drug-Report-2015.
6. Kataria MK, Garg M, Anand V, Bilandi A, Kukkar V, Bhandari A. An Insight on Regulations Governing Orphan Diseases and Drugs. Research Journal of Pharmaceutical, Biological and Chemical Sciences. 2011; 2(3): 373–84.
7. Addison A, Alagille D, Amyotrophic S, Sclerosis L, Hyperplasia A. List of Rare Diseases. Drug News and Perspectives. 1996; 9(8): 4–6.
8. Davis H and Smith J. Orphan Drug Law Matures into Medical Mainstay. International Journal of Rare Disorder Research. 1999; 33(3): 11-16.
9. Kesselheim AS. Innovation and the Orphan Drug Act 1983-2009. Regulatory and Clinical Characteristics of Approved Orphan Drugs. 2015; (DC):1–12.
10. Rare Diseases: Common Issues in Drug Development [Internet]. Guidance for Industry Rare Diseases; 2015 Aug [cited 2016 Apr 10]. Available from:
11. McNeilly EK. Designating an Orphan Product: Drug and Biological Products - Orphan Drug Regulations [Internet]. Regulatory History 283745; 2014 [cited 2016 Apr 16] Available from:
12. Sharma A, Jacob A, Tandon M, Kumar D. Orphan drug: Development Trends and strategies. Journal of Pharmacy and Bio-Allied Sciences. 2010; 2(4):290-9.
13. Seoane VE, Rodriguez MR, Szeinbach SL, Visaria J, Incentives for orphan drug research and development in the United States, Orphanet Journal of Rare Disease, 2008; 4(3):33.
14. Orphanet -The portal for rare diseases and orphan drugs [Internet]. Orphan drugs in the United States of America; 2016 [cited 2016 Apr 26] Available from:
15. European Union. Commission Regulation (EC) No 141/2000 of the European Parliament. Official Journal of European Union [Internet]. 2000 [cited 2016April 30]; L18:1–5. Available from:
16. European Union. Commission Regulation (EC) No 2003/C 178/02 of the European Parliament. Official Journal of the European Communities [Internet]. 2003 [cited2016 May 4]; L178:1-7. Available from:
17. Gajra BB and Limbachiya S. Absence of Regulations for Orphan Drugs in India : Ignorance of the Government [Internet]. 2015 [cited 2016 May 7] Available from:
18. Divya VC and Sathasivasubramanian S. Submandibular Sialolithiasis - A report of two cases. The Journal of Medical Research. 2015; 1(1): 05-07.
19. Hathaway C, Manthei J, Scherer CZ. Food and Drug Law Institute. 2009; (3).
20. Janodia MD, Chauhan A, Hakak SM, Sreedhar D, Ligade VS, Udupa N. Data Exclusivity Provisions in India: Impact on Public Health. Journal of Intellectual Property Rights. 2008;13 (5):442–6.
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
The International Journal of Drug Regulatory affairs require a formal written transfer of copyright from the author(s) for each article published. We therefore ask you to complete and return this form, retaining a copy for your records. Your cooperation is essential and appreciated. Any delay will result in a delay in publication.
I/we have read and agree with the terms and conditions stated Page 2 of this agreement and I/we hereby confirm the transfer of all copyrights in and relating to the above-named manuscript, in all forms and media, now or hereafter known, to the International Journal of Drug Regulatory affairs, effective from the date stated below. I/we acknowledge that the IJDRA is relying on this agreement in publishing the above-named manuscript. However, this agreement will be null and void if the manuscript is not published in the IJDRA.
Download link for COPYRIGHT FORM