India's Patients Are Its Strongest Pharma Asset: Trading Trial Cohorts for Fair Orphan-Drug Prices

India's Real Advantage in Rare-Disease Drugs Is Its Patients Developing a

drug for a rare disease is difficult for a simple reason. Before a company can show that a drug works, it has to find enough people who have the disease. When a disorder affects one person in tens of thousands, recruiting a trial cohort can take years. India has an unusual advantage here and it comes from its population genetics.

Why some "rare" diseases are less rare in India Many rare disorders are recessive.

A child develops the disease only if they inherit a faulty copy of a gene from both parents. In a large, mixed population, two carriers of the same rare variant seldom have children together, so the disease stays rare. Endogamy, the practice of marrying within a community, changes those odds.

If a variant entered a closed community generations ago, it keeps circulating within that community instead of being diluted across the wider population. Two carriers are then much more likely to meet. A simple way to picture it: a rare card is more likely to be drawn twice from a small, closed deck than from a huge shuffled pile.

The analogy has limits. Genes are not drawn at random and endogamy raises risk only for variants already present in that group. But it captures the mechanism. The data reflects this. In particular ethnic groups, beta-thalassemia has been reported at 10.5%, against 3.7% in the general population.

Patients are also increasingly organised: one study follows 70 people with GNE myopathy and Rett syndrome groups maintain a registry of 400+ patients.

The problem and the bargain that could fix it Despite these advantages, Indian patients rarely take part in international orphan-drug trials.

Even when a drug is approved, its price puts it far out of reach. Lenmeldy costs $4.25 million, while the National Policy for Rare Diseases covers only ₹50 lakh. The Kolkata family had to raise ₹9 crore, eighteen times that cap. The authors propose an exchange based on benefit-sharing, a principle discussed in the negotiations on the WHO Pandemic Agreement:

• India contributes trial cohorts, low-cost small-batch manufacturing and a clearer approval route under Rule 101, which lets CDSCO skip repeat local trials for drugs approved in specified countries.
• India gets the right to set prices within India and the Global South. Developers remain free to price elsewhere.
• The state reduces the risk through production-linked incentives and advance market commitments, with the government agreeing in advance to buy set volumes at set prices through Centres of Excellence, CGHS and Jan Aushadhi Kendras.

Where

India stands globally On manufacturing, India is already ahead of most countries. Its generics fill 47% of US generic prescriptions, it supplies 15% of US biosimilars and it now makes CAR-T therapies to international standards.

Crucially, Indian manufacturers are willing to produce small volumes, which many Western plants avoid. On drug discovery and trials, India lags. The US and China are competing hard in this field and China already combines access to patients with low-cost production.

India has both of those advantages too. What it lacks is a policy that turns them into leverage in negotiations with drug developers. The exam lesson is that access to patients is a bargaining asset, not just a public-health burden. The practical question for Indian policy is whether the country treats its cohorts as something to offer for free or as the basis for negotiating fair prices.