Challenges in orphan drug development and regulatory requirements in India
While regulatory policy is well-defined in the United States and Europe for the development of orphan drugs, Indian guidelines on rare diseases is still transforming. In India due to lack of a transparent definition and regulatory approval process for rare diseases therefore until now, has deterrent pharmaceutical industries to pursue drug development for rare diseases. The orphan medication program can help pharmaceutical organizations in gaining benefits and recouping their venture even with little patient population in the developed nation. In 2001 a conference was assemble by the Indian Drugs Manufacturers Association (IDMA) where a gathering of pharmacologists mentioned the Indian government to set up the Orphan Drug Act in India. Since, guidelines for the orphan drug have not been made by the Indian government. Indian rare disease patient relies upon developed nations approved orphan drugs. Developing countries are likewise feeling they ought to plan something for the elevation of orphan drug research and manufacturing. The lack of regulatory guidelines of orphan drug antagonistically affecting the monetary development of Indian medical businesses. One of the significant reasons is that the pharmaceutical company isn't quick to look into the improvement of orphan drugs as these medications don't catch a huge market. However, in this article, we have attempted to concentrate on drug regulation of United States and it has been noted that the two biggest populated nation India and China, both need national enactment for orphan drugs and rare diseases, which carries a significant negative effects on their patient population with rare diseases.
2. Schieppati A, Henter JI, Daina E and Aperia A: Why rare diseases are an important medical and social issues. The Lancet. 2008; 371(9629): 2039-41.
3. Haffner ME: Adopting orphan drugs- two dozen years of treating rare diseases. New England Journal of Medicine. 2006; 354(5):445-7.
4. 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.
5. 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.
6. Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: Challenges and opportunities. J Child Neurol. 2013; 28:1142‑50.
7. Z.Thaker, K.Jethva, D.Bhatt, M.Zaveri and S.Deshpande. Orphan Drugs: Overview and regulatory review process. IJPSR [Internet]. 2019 [Cited 2020 Jan 24]; 10(2):505-518. Available from:
8. Cheng A, and Xie Zhi. Challenges in orphan drug development and regulatory policy in China. Orphanet Journal of rare diseases [Internet]. 2017 [Cited 2020 Jan 27]. Available from:
9. Kumar H, Sarma P, and Medhi B. Orphan Drugs: Indian Perspective. Indian J Pharmacol [Internet]. 2017 [Cited 2020 Jan 25]; 49:267-9. Available from:
10. Budhwar V, Singh AK, Choudhary M. Regulations of Orphan drugs in USA, EU and India-A Comparative Study. Int J Drug Reg Affairs [Internet]. 2018 Feb.13 [cited 2020 Mar.25]; 4(3):30-7. Available from:
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