India looks to build rare-disease drug sector using vast patient base and low-cost output

UPDATE - follow-up to Why rare-disease drugs could become India's next pharma frontier: India can transform into a global hub for rare-disease drugs by combining its large patient population, patient groups, and low-cost manufacturing strengths.

Clinical Trials And Manufacturing

While each rare disease affects few people, about 8,000 such disorders cumulatively impact hundreds of millions worldwide, with 1,004 rare genetic conditions reported in India. Due to high endogamy in certain groups, the local prevalence of specific rare diseases can be higher, such as beta-thalassemia reaching 10.5% in particular ethnic groups compared to 3.7% in the general population. India's population of over 1.4 billion and growing patient advocacy groups, including registries for Rett syndrome and longitudinal studies on GNE myopathy, offer ideal cohorts for clinical trials. The government could foster foreign collaboration similar to chip manufacturing and invite multinational firms to set up drug-development centres. Indian patients may assist development if they benefit from the resulting drugs, prompting a proposal that India control prices locally and for the Global South while companies set global prices.

Regulatory Pathways

India already supplies 47% of generic prescriptions and 15% of biosimilars in the U.S., alongside producing personalised CAR-T cancer therapies. Under Rule 101 of the New Drugs and Clinical Trials Rules 2019, the Central Drugs Standard Control Organisation permits drugs approved in six specific countries like the U.S. and U.K. to bypass local clinical trials. This pathway requires bioequivalence, safety studies, and strong pharmacovigilance safeguards.

Context

Orphan drugs historically suffered from low industry interest due to small patient markets until government incentives like the U.S. Orphan Drug Act of 1983 boosted approvals. Developing nations like India are now examining how benefit-sharing models and local manufacturing can address the high costs of these therapies.