Rare-disease drugs can be India’s next pharma frontier

Why in the News

Researchers at the Tata Institute for Genetics and Society propose that India build an industry for orphan drugs, medicines for rare diseases. India has reported 1,004 rare genetic disorders, yet patients rely on crowdfunding because such drugs are scarce and costly.

What are orphan drugs, and why are they out of reach?

1. What they are: Each rare disease affects very few people, so firms saw little profit in treating it. Its medicines became orphan drugs, like products no company wants to adopt.
2. Collective scale: Around 8,000 rare diseases together affect hundreds of millions, yet drugs exist for only about 5% of them.
3. High prices: A small market leads firms to charge around $100,000 (Rs 1 crore) a year, unaffordable without insurance or state cover.
4. Support falls short: Government support of up to Rs 50 lakh per patient rarely covers a year, so almost 4,000 children are on a government crowdfunding platform.
5. The takeaway: Rare diseases are rare one by one but common together, so India needs its own route to affordable orphan drugs.

How did the US Orphan Drug Act change incentives?

1. Orphan Drug Act, 1983: The United States gave the first approved product seven years of market exclusivity, meaning no rival could sell it. Firms also got tax incentives, grants and fee waivers.
2. Approvals surge: Lower costs and assured profit raised US orphan drug approvals about thirtyfold.
3. Laws elsewhere: Japan, Australia and the European Union passed similar laws.

Why is India suited to run orphan drug trials, and on what terms?

1. Large patient pool: India has over 1.4 billion people, and endogamy (marriage within a closed community) makes some rare diseases commoner. Its diaspora gene pool also carries unique mutations. Eg. Beta-thalassemia affects 10.5% of some groups.
2. Organised patients: One study tracks 70 people with GNE myopathy, and Rett syndrome groups keep a registry of over 400 patients.
3. Missing from global trials: Indian patients rarely join international trials. The government could broker foreign tie-ups, as in chip manufacturing, and invite drug development centres.
4. Access in return: Patients will help only if they benefit, so India should set prices for India and the Global South, leaving developers free elsewhere. This is the benefit-sharing idea debated for the World Health Organization (WHO) Pandemic Agreement.

Can India make orphan drugs, and how should the state help?

1. Pharmacy of the world: Indian generics fill 47% of US generic prescriptions, and India has the most US-approved plants outside the US.
2. Advanced and small-batch capacity: Indian firms supply 15% of US biosimilars (near copies of biological drugs), make chimeric antigen receptor T-cell (CAR-T) therapy, a cancer cell therapy, and will make small volumes cheaply.
3. Rule 101 pathway: Under Rule 101, New Drugs and Clinical Trials Rules, 2019, a drug approved in one of six countries named by the Central Drugs Standard Control Organisation (CDSCO) needs no fresh Indian trial. It lacks guidance for applicants.
4. Incentives and purchase: The state should offer Production Linked Incentive (PLI) style rewards for output, investment, exports and patient access. It should add advance market commitments, a promise to buy set volumes at set prices.
5. De-risked supply: Purchases would flow through Centres of Excellence on Rare Diseases (designated treatment hospitals), the Central Government Health Scheme and Jan Aushadhi Kendras. The aim is to de-risk the first investment, not subsidise firms.

Challenges

1. Trial design: Small numbers make it hard to find a patient cohort and fix trial endpoints, the outcomes proving a drug works.
2. Affordable small-scale supply: Making tiny volumes cheaply at high quality stays hard even after approval.
3. Chinese competition: Chinese firms, racing US firms, already have patients and low-cost manufacturing.

Way Forward

1. Registries for trials: The Indian Council of Medical Research (ICMR) should turn rare disease registries into a trial recruitment network.
2. Newborn screening: States should widen newborn screening to catch treatable rare diseases early.
3. Clear Rule 101 guidance: CDSCO should base such approvals on bioequivalence (the copy acts like the original) and pharmacovigilance (tracking harm after launch).
4. Cost-linked support: The Health Ministry should revise the per patient cap for the costliest therapies.

Conclusion

India already makes medicines cheaply for the world, but orphan drug prices and supply are still decided elsewhere. Watch for published approval guidance and a firm government purchase commitment.

Key numbers

1. US orphan drug approvals: 38 before the Act; 1,122 by 2022.
2. Crowdfunding: Listed children’s annual treatment cost over Rs 9,000 crore; Rs 9 crore raised for one Kolkata toddler (mid-2025).
3. Beta-thalassemia: 3.7% in India’s general population (2023 review).

Matching Previous Year Question

“[2024, GS2, 15 marks] In a crucial domain like the public healthcare system, the Indian State should play a vital role to contain the adverse impact of marketisation of the system. Suggest some measures through which the State can enhance the reach of public healthcare at the grassroots level.”