Assessment of the availability of repurposed orphan drugs in India

India has a massive burden of rare diseases (RDs), with an estimated 96 million people living with RDs but limited options for treatment. Repurposing drugs used for other common conditions are essential alternative for RDs due to their cost-effectiveness and reduced timeline. India’s patent regime prevents the evergreening of drugs, and a large generic manufacturing industry provides ample opportunity to explore the potential of repurposed drugs for treating RDs, known as repurposed orphan drugs (ROD). However, there is no portal or other source for information on orphan drugs in India. This study assesses the availability of RODs in India through quantitative analysis. In the absence of a separate orphan drug designation in India, we consider USFDA-approved orphan-designated products as the reference. We searched USFDA-approved RODs in recognized sources in India, such as CDSCO, AYUSH gazette, FSSAI, and Indian Pharmacopeia, which provides a list of drugs approved for marketing in India. We classified the drugs into separate groups based on their record from different sources and explored the regulatory implications of the differential representations. We found that almost 76% of the USFDA-approved RODs are listed in one of the Indian regulatory bodies’ records either in the same form (61%) or in a different fixed-dose combination. For 67 drugs no information was found, these drugs have to be imported for use. Only 17 of the 43 RDs mentioned in the National Policy for Rare Diseases, matched the indications listed for approval of one of the 279 RODs identified in the study. This underscores the scarcity of RD treatment and the pressing need for accessibility in India. This information on RODs will help relevant stakeholders to efficiently manage RDs in India. The study also highlights existing gaps in Indian regulatory databases that limits access to accurate information about the availability of drugs.