India's Emerging Opportunity in Rare-Disease Drug Manufacturing
How India could become a global hub for orphan drug production and access
1The orphan drug paradox
While each rare disease affects few people, 8,000 such conditions collectively impact hundreds of millions worldwide. India has documented 1,004 rare genetic disorders, creating a substantial patient base. Historically, pharmaceutical companies avoided developing treatments due to small markets, labeling them 'orphan drugs'. The U.S. Orphan Drug Act of 1983 changed this by offering tax incentives, 7-year market exclusivity, and grants, leading to approvals jumping from 38 to 1,122 drugs by 2022. Similar policies in Japan, Australia, and the EU followed.
2Prohibitive pricing challenge
Orphan drugs often cost over $100,000 annually, making them inaccessible without insurance or government support. India provides up to Rs 50 lakh per patient, but this covers less than a year's treatment for many conditions. Crowdfunding has become common, with Rs 9 crore raised for one child's gene therapy in 2025. Nearly 4,000 children on a government crowdfunding platform require over Rs 9,000 crore annually. Despite development efforts, current drugs only address 5% of rare diseases, leaving most patients without options regardless of cost.
3Clinical trial advantages
India's 1.4 billion population and high endogamy rates create concentrated patient pools for rare diseases. Beta-thalassemia prevalence reaches 10.5% in some groups versus 3.7% nationally. Organized patient advocacy groups maintain registries like 70 GNE myopathy patients and 400 Rett syndrome cases. The Indian diaspora's unique genetic mutations add research value. However, Indian patients rarely participate in international orphan drug trials, representing untapped potential for global pharma collaborations.
4Manufacturing infrastructure
India produces 47% of generic drugs used in the U.S. and hosts the most FDA-approved manufacturing sites outside America. It supplies 15% of U.S. biosimilars and recently began producing CAR-T cancer therapies. Indian manufacturers excel at small-batch production where Western firms decline, offering quality at low cost. Rule 101 of India's 2019 drug rules allows approval of drugs cleared in six reference countries without local trials, though clearer guidance documents could streamline this process further.
5Proposed benefit-sharing model
The authors suggest India negotiate drug pricing for domestic and Global South markets while allowing free pricing elsewhere. This mirrors WHO's Pandemic Agreement concept of equitable benefit-sharing. With rare disease patients willing to participate in trials primarily for treatment access (not payment), such arrangements could make India an attractive partner for U.S. and Chinese firms racing to develop these drugs. China offers patient access and low-cost manufacturing, but India combines both with existing pharma infrastructure.
6Policy recommendations
Key proposals include production-linked incentives for orphan drugs, tax benefits for manufacturing investments, and technology transfer support. Advanced market commitments could guarantee government purchases at set prices for distribution through rare disease centers and public health schemes. The goal isn't perpetual subsidies but de-risking initial investments to establish commercial viability. Combined with streamlined regulations and pharmacovigilance systems, this could position India as a global orphan drug manufacturing hub.
7Regulatory pathway optimization
India's current system allows approval based on foreign approvals plus bioequivalence, safety, and post-market effectiveness studies. However, the process needs transparent guidance documents like those used by foreign regulators. A robust framework must maintain quality and safety standards while reducing duplicate paperwork. The Central Drugs Standard Control Organisation could accelerate approvals by clarifying requirements for orphan drug applications, particularly for generics of already-approved treatments.
8Manufacturing incentives blueprint
The proposal suggests incentives tied to production volume, investment size, export performance, and patient access metrics. Support could include collaborations with national labs for process development and export incentives targeting low-middle income countries. Government procurement through existing healthcare networks would provide demand certainty during early production phases. This multi-pronged approach aims to replicate India's generic drug success in the orphan drug sector without creating long-term subsidy dependencies.
9Global accessibility vision
India has demonstrated how to make generic drugs affordable worldwide. The next frontier is applying this model to rare-disease treatments. By combining its manufacturing capabilities, patient populations for trials, and innovative pricing models, India could transform orphan drug access globally. Success would require coordinated policy across regulatory simplification, financial incentives, and public-private partnerships - potentially making India the pharmacy not just for common ailments but for the rarest conditions too.