Challenges in orphan drug development and regulatory requirements in India

  • Nisha Chaudhary M.Pharm (Drug Regulatory Affairs)
  • Vikesh Kumar Shukla


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.

Keywords: Orphan drug, orphan drug act, orphan drug regulation, recent initiatives, rare diseases


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How to Cite
Chaudhary, N., and V. K. Shukla. “Challenges in Orphan Drug Development and Regulatory Requirements in India”. International Journal of Drug Regulatory Affairs, Vol. 8, no. 1, Mar. 2020, pp. 31-35, doi:10.22270/ijdra.v8i1.380.

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