How India Can Become a Global Hub for Rare Disease Medicines

India holds the potential to emerge as a major global player in developing and manufacturing affordable drugs for rare diseases, known as orphan drugs. While each rare condition affects very few people, about 8,000 such disorders cumulatively impact hundreds of millions worldwide, with 1,004 rare genetic disorders reported in India so far.

The Challenge of Cost

Historically, pharmaceutical companies showed limited interest in rare diseases because patient numbers were tiny. Many governments solved this by offering incentives like tax breaks, grants, and market exclusivity, such as the U.S. Orphan Drug Act of 1983. Because markets are small, companies often price these medications around $100,000 or roughly Rs 1 crore per year, putting them out of reach without insurance or government support. India's government provides up to Rs 50 lakh for patient treatment, but this often falls short, forcing families to rely on crowdfunding. In mid-2025, a Kolkata family raised Rs 9 crore through crowdfunding for a 16-month-old child's gene therapy, while nearly 4,000 children are listed on a government crowdfunding platform with cumulative annual treatment costs exceeding Rs 9,000 crore.

India's Advantages

India has several unique strengths to tackle these hurdles. With a population exceeding 1.4 billion and high rates of endogamy in certain groups, the prevalence of specific rare conditions is higher here. Beta-thalassemia prevalence reaches up to 10.5% in particular ethnic groups compared to 3.7% in the general population. Patient groups have also organized registries, such as a longitudinal study tracking 70 people with GNE myopathy and Rett syndrome groups tracking over 400 patients. Furthermore, India is already known as the pharmacy of the world, supplying 47% of generic prescriptions in the U.S. and 15% of all biosimilars sold there, alongside producing CAR-T cancer therapies to international standards.

Proposed Solutions

To build a globally competitive rare-disease sector, authors suggest foreign drug makers collaborate with India for clinical trials and manufacturing. Indian patients could participate in trials on the condition that they gain access to the resulting drugs at controlled, affordable prices set for India and the Global South, while companies keep free pricing elsewhere. Additionally, under Rule 101 of the New Drugs and Clinical Trials Rules 2019, the Central Drugs Standard Control Organisation allows approved drugs from six countries like the U.S. and U.K. to bypass new local clinical trials if they meet standards of bioequivalence, safety, and effectiveness.

Context

Orphan drugs are specialized pharmaceutical products created to treat very small patient populations that commercial developers often ignore without regulatory and financial incentives. Benefit-sharing models, similar to discussions under the World Health Organization's Pandemic Agreement, seek to balance fast global access to biological data with fair and equitable distribution of the resulting medical treatments.