Ranjini D.M, Sadiq Basha G, Prabakaran N.
firstname.lastname@example.org , email@example.com
Ranjini D.M*, Sadiq Basha G, Prabakaran N.
1Regulatory Executive in Generic Formulation Regulatory Science, Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka 5600100.
2Associate Vice President – Regulatory Science Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka 5600100.
3Manager in Regulatory Sciences – Generic Formulations Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka 5600100.
Volume - 14,
Issue - 6,
Year - 2021
Objectives of the present work are as follows:
• To study the current principles of rare diseases & orphan drugs.
• To study the assessment, challenges and regulatory frame work of orphan drugs
• To study the integrated approach for the development and approval of orphan drugs.
• To carry out the study of globalization in orphan drug development strategies in US & EU markets.
Methods: Internet using web page content: The literature was collected using numerous search engines e.g. Science Direct, Google Scholar and many more. Online books also served as a good source of information.
Documents and information’s collected using numerous regulatory websites such as:
a) USFDA: https://www.fda.gov
b) EMA: https://www.ema.europa.eu/en
c) CANADA: https://www.canada.ca/en/health-canada.html
d) TGA: https://www.tga.gov.au/
e) INDIA: http://www.cdsco.com/
Results: US- FDA Approved Orphan Drug ex: Tafenoquine - Treatment of malaria - Krintafel is indicated for the radical cure (prevention of relapse) of Plasmodium vivax malaria. EU – EMA Approved Orphan Drug ex: Eculizumab, Soliris - Treatment myasthenia gravis. Conclusion: The orphan drug guidelines made via distinct countries have established as promoters in development of orphan drugs. The orphan drug regulation in the US and the EU has been a success in offering remedies to the patients with rare diseases.
Cite this article:
Ranjini D.M, Sadiq Basha G, Prabakaran N. Regulatory Strategies for Orphan drug Development in USA–Europe. Research Journal of Pharmacy and Technology. 2021; 14(6):3449-4. doi: 10.52711/0974-360X.2021.00600
Ranjini D.M, Sadiq Basha G, Prabakaran N. Regulatory Strategies for Orphan drug Development in USA–Europe. Research Journal of Pharmacy and Technology. 2021; 14(6):3449-4. doi: 10.52711/0974-360X.2021.00600 Available on: https://rjptonline.org/AbstractView.aspx?PID=2021-14-6-90
1. Singh MK, Sharma PK, Sharma N. Orphan Drug Development: A Brief Review. Journal of Pharmacy Research. 2011; 4(8):2645–7. 2.
2. European Medicine Agency [Internet]. Medicines for Rare Diseases: 3–5; 2016 [cited 2016 Apr 13]. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/ special topics/general/general_content_000034.jsp.
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/Evalua tePharma-Orphan-Drug-Report-2015.
6. Jyothi G, Venkatesh M, Kumar PT, Radhadevi N, Gundavaram R and Sharma KK: Orphan Drug Act: History, Perspective and Challenges for Future. Am J PharmTech Res 2012; 2249-3387.
7. Schieppati A, Henter JI, Daina E and Aperia A: Why rare diseases are an important medical and social issue. The Lancet 2008; 371(9629): 2039-41.
8. Haffner ME: Adopting orphan drugs-two dozen years of treating rare diseases. New England Journal of Medicine 2006; 354(5): 445-7.
9. Stakisaitis D, Spokiene I, Juskevicius J, Valuckas KP and Baiardi P: Access to information supporting the availability of medicines for patients suffering from rare diseases looking for possible treatments: the European Service. Medicine (Kaunas, Lithuania) 2007; 43(6): 441-6.
10. Wästfelt M, Fadeel B and Henter JI: A journey of hope: lessons learned from studies on rare diseases and orphan drugs. Journal of Internal Medicine 2006; 260(1): 1-10.
11. FDA. Available from: http://www.fda.gov/downloads/ For Consumers.