1Regulatory Executive in Generic Formulation Regulatory Science, Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka5600100
2Associate Vice President - Regulatory Science Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka5600100
3Manager in Regulatory Sciences - Generic Formulations Biocon Pharma Limited, 20th KM, Hosur Road, Electronic City, Bengaluru, Karnataka5600100
*Corresponding Author E-mail: ranjinidm56@gmail.com
Online published on 25 August, 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.
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:
USFDA: https://www.fda.gov
EMA: https://www.ema.europa.eu/en
CANADA: https://www.canada.ca/en/health-canada.html
TGA: https://www.tga.gov.au/
INDIA: http://www.cdsco.com/
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.
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.
Regulations of orphan drugs, Regulatory challenges, Regulatory Orphan drug life cycle
