Assessing the availability of Repurposed Orphan Drugs in India

Abstract

AbstractIndia has a massive burden of rare diseases (RDs), estimated to be around 96 million living patients but limited options for treatment. Drugs used for RDs are known as Orphan Drugs. Globally, 95% of RDs do not have an approved drug for treatment. Novel orphan drugs are characteristically expensive and out of reach for most Indian patients. Repurposing drugs used for other common conditions have been considered an essential alternative for RDs due to their cost-effectiveness and reduced timeline resulting in higher success rates than novel drugs. 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. However, information on the availability of repurposed orphan drugs (RODs) in India is limited. Also, there is no portal for information on orphan drugs in India. This study assesses the availability of RODs in India through quantitative empirical analysis. In the absence of a separate orphan drug designation in India, we consider USFDA-approved orphan-designated products as the reference. We searched for the availability of FDA-approved RODs in recognized sources in India, such as CDSCO, AYUSH gazette, and Indian Pharmacopeia, which provides a list of drugs approved for marketing in India. We classified the drugs into separate groups based on their availability in different sources and explored the regulatory implications of the differential representations. We found that 76% of RODs approved by the USFDA are entirely or partially available in India. Information on RODs will help relevant stakeholders in the better management of RDs in India.