Objective-

An orphan disease is defined as a condition that affects fewer than 200,000 people nationwide and an orphan Drug is used to treat such a condition. However, these cases receive very little attention in developing nations such as India as compared to developed countries such as the United States, Europe, Japan and Australia. The purpose of this paper is to identify the hurdles in the field of orphan drugs in India and suggest solutions to address the same.

Method-

The scenario of orphan drugs in India was analysed and compared to developed nations using search engines such as PubMed, government websites like Ministry of Health and Welfare (MOHFW), US Foods and Drugs Administration, European Medicines Agency (EMA) and other publicly available resources. Data was extracted from various journals published between 2002-2018 pertinent to the challenges faced by the orphan drug development and the present scenario of orphan drug legislation in India.

Results-

Upon reviewing the Indian Orphan drug regulations and the orphan drug policies of other developed countries, challenges identified to be were- 1.Difficulty in obtaining public and private funding for research and development 2.Finding an appropriate clinical research design for a small population, 3.Inadequate information on orphan diseases 4.Lack of knowledge and training in health professionals and 5.Affordability of Orphan Drugs.

Conclusion

The results of the study have highlighted the requirement of an appropriate Orphan Drugs Act in India to combat the challenges faced by the domestic pharmaceutical industry. As the role of the government is pivotal for the advancement of the field, it should prioritise extending financial support and providing regulatory concessions such as shorter clinical trials and assistance to form collaborations with government organizations to encourage research for orphan drugs.