Rare Disease Drugs: Can India Make Treatment More Affordable?

India has built a pharmaceutical industry around the ability to make medicines at lower cost. Rare diseases present a harder question: can the country use that strength to make some of the world’s least accessible medicines more affordable and available?

A medicine can exist and still be out of reach.

That is one of the most difficult realities in rare-disease treatment. For a pharmaceutical company, a drug for a condition affecting only a small number of patients can be an expensive proposition. The research may take years. Clinical trials may be difficult to organise. Manufacturing may have to be done in very small quantities. And after all that investment, there may be too few patients to create a conventional commercial market.

For the patient, however, the calculation is very different. A rare disease does not become less serious because only a few people have it. If an effective treatment exists, the question is whether the patient can actually get it.

This is where India has an unusual opportunity.

The country already has deep experience in producing generic medicines, biosimilars and other pharmaceutical products at relatively low cost. It has a large patient population, an expanding network of rare-disease researchers and patient groups, and manufacturing capacity that can serve both domestic and international markets. India also has a regulatory framework that provides specific routes for certain orphan drugs and other specialised therapies approved in specified countries.

The opportunity, therefore, is larger than simply manufacturing another category of medicines.

It is about building an economic and regulatory system in which rare-disease drugs can be developed, manufactured and supplied without leaving patients behind.

A rare disease is not necessarily a small problem

The word “rare” can be misleading.

An individual rare disease may affect relatively few people. But there are thousands of such conditions. Estimates commonly put the number of known rare diseases at around 7,500 to 8,000, collectively affecting hundreds of millions of people worldwide. India’s burden is difficult to measure precisely because comprehensive national prevalence data remain limited, but research has repeatedly pointed to the country’s large and genetically diverse population as an important factor.

India also has a particular genetic landscape. In some communities, long-standing patterns of endogamy have contributed to a higher prevalence of particular inherited disorders and founder mutations than might be expected from global averages. That does not make rare diseases common in the ordinary sense, but it can make India an important population for studying some of them.

The country is also building the infrastructure needed to understand that population better. The ICMR’s National Registry for Rare and Other Inherited Disorders was established to collect information on patients, disease progression and treatment outcomes, while also helping create patient bases for research and evaluation of drugs and orphan products.

That matters because one of the first problems in rare-disease drug development is not always the laboratory.

It is finding enough patients.

Why the pharmaceutical industry once looked elsewhere

The economics of rare-disease drug development have historically discouraged investment.

A conventional medicine may be developed for millions of potential patients. An orphan drug may have a much smaller population from which to recover the cost of discovery, testing, regulatory approval and manufacturing.

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Governments in several countries therefore changed the economics through policy.

The United States provides the most familiar example. Its Orphan Drug Act of 1983 introduced measures including tax incentives, grants, regulatory fee waivers and market exclusivity for qualifying products. The basic idea was straightforward: if the commercial market was too small to justify the cost of development, public policy could reduce some of the risk.

The results changed the industry.

Before the law, only a small number of drugs for rare diseases had been approved in the U.S. The subsequent expansion of orphan-drug development became one of the reasons other countries and regions, including Japan, Australia and the European Union, introduced their own incentives.

The lesson for India is not that it should copy the American system.

It is that policy can change the commercial calculation around a medicine that would otherwise remain unattractive to develop.

The price of a treatment can become another barrier

There is a second problem.

Once a rare-disease medicine has been developed, the small number of patients can contribute to a very high price. For some therapies, annual treatment costs can run into tens of millions of rupees.

That creates a difficult chain of dependence.

The company needs a price that allows it to recover research and development costs and remain commercially viable. The patient needs a price that a family can actually afford. An insurer or government health system, where one exists, has to decide how much of that cost it can absorb.

In India, the National Policy for Rare Diseases provides financial assistance of up to ₹50 lakh for eligible patients in specified circumstances. But for some advanced therapies, particularly high-cost gene therapies, that amount can be far below the total treatment cost. Recent reporting has also highlighted patients who have exhausted the ₹50 lakh support limit while still requiring treatment.

The result is a system in which families may turn to crowdfunding to bridge the gap.

That is not simply a story about individual hardship. It is also a sign of a larger problem in the economics of rare-disease treatment.

A medicine that is scientifically available but financially unreachable is only partially accessible.

And there is an even bigger gap: many rare diseases still have no suitable drug at all.

The missing medicines are the bigger market problem

Existing rare-disease therapies cover only a small proportion of rare conditions. The overwhelming majority of patients therefore face one of two situations: there is no approved treatment, or the available treatment is so expensive or difficult to obtain that access remains uncertain.

Developing new drugs is particularly difficult in this field.

Researchers need enough patients to conduct meaningful clinical trials. They also need appropriate endpoints, which can be challenging when a disease affects very few people and may progress differently from patient to patient.

India could play a much larger role here.

Its population alone means that even a very low-prevalence condition can produce a patient pool large enough to support research. Patient organisations have also become more active, and disease-specific registries can help researchers identify potential participants.

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Some Indian studies have already demonstrated the value of such patient cohorts. Research groups have followed patients with conditions such as GNE myopathy, while patient organisations working on Rett syndrome and other disorders have built registries that can help connect families and researchers.

The Indian diaspora adds another dimension. Genetic variants found among Indian-origin populations can sometimes be distinctive, creating opportunities for research that may be relevant well beyond India.

Yet Indian patients are not routinely represented in international orphan-drug trials to the extent that India’s population and disease burden might suggest.

That is a gap the country could address.

India could become more than a manufacturing base

The conventional description of India as the “pharmacy of the world” is largely built on generics.

That achievement matters here, but rare diseases require a broader model.

India could potentially participate in the entire chain:

patient identification → clinical research → drug development → regulatory approval → manufacturing → procurement → distribution

That would require collaboration between multinational pharmaceutical companies, Indian manufacturers, research institutions, hospitals and patient organisations.

The government could encourage foreign companies developing rare-disease therapies to establish development programmes in India rather than treating the country only as a future sales market.

There is an important principle behind this.

If Indian patients participate in the research that helps bring a treatment to market, they should have a realistic pathway to access that treatment once it is approved.

Otherwise, India risks becoming a source of patients and research data while the resulting medicine remains financially inaccessible to the same population.

Regulation can remove duplication without removing safeguards

India has already created part of the regulatory pathway needed for this.

Under the New Drugs and Clinical Trials Rules, 2019, certain new drugs already approved in specified countries can be considered for a waiver of local clinical-trial requirements. The framework specifically includes orphan drugs for rare diseases, alongside categories such as gene and cellular therapy products. CDSCO’s 2024 order specifies the relevant countries as the United States, United Kingdom, Japan, Australia, Canada and the European Union.

This can be important for rare diseases because repeating an entire development process in every country can be particularly difficult when the global patient population is already small.

But faster approval cannot mean weaker oversight.

A workable Indian pathway would still need appropriate assessment of quality, safety and efficacy, along with post-market surveillance and pharmacovigilance. The objective should be to eliminate unnecessary duplication in the regulatory process, not to eliminate scrutiny.

Greater clarity for companies about how to apply under the existing framework could also reduce uncertainty and make India more attractive for rare-disease drug development.

Manufacturing may be where India has its strongest advantage

Research is only half of the problem.

Once a rare-disease drug is approved, somebody has to manufacture it consistently, in the right quantities and at a price that does not make access impossible.

This is an area where India already has considerable experience.

Indian pharmaceutical companies supply a large share of the world’s generic medicines and have developed substantial capabilities in biosimilars. The country has also begun producing more advanced therapies, including CAR-T treatments, demonstrating that Indian manufacturing is no longer limited to conventional small-molecule generics.

Rare-disease medicines create a different manufacturing requirement.

The volumes may be small. Production may need to be highly specialised. Distribution may involve a limited number of treatment centres. Conventional high-volume pharmaceutical economics do not necessarily apply.

Indian manufacturers may nevertheless be better positioned than many manufacturers in higher-cost markets to make commercially viable products at smaller volumes.

That is where the country’s existing pharmaceutical capability could become a competitive advantage.

Who should take the initial financial risk?

The government has a role here, but it should be a carefully designed one.

A company deciding whether to manufacture an orphan drug in India is making an investment decision. If demand is uncertain and the patient population is small, the initial risk can be too high.

Government policy can reduce that risk through targeted measures.

These could include tax incentives for investment, production-linked incentives for selected rare-disease medicines, support for technology transfer and process development, and advance market commitments under which the government commits to purchase a defined quantity at an agreed price.

Government procurement could also create a predictable early market.

Medicines could potentially be distributed through designated Centres of Excellence for rare diseases, government health schemes and public pharmaceutical outlets such as Jan Aushadhi Kendras, where appropriate.

The important point is that public support should not simply transfer commercial risk to taxpayers while leaving patients with high prices.

The objective should be to make the initial investment sufficiently predictable for companies to enter the market while linking public support to outcomes such as production, supply, patient access and, where relevant, exports.

That would make the policy less about subsidising an industry and more about building an ecosystem.

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There is another market beyond India

A successful Indian rare-disease manufacturing model would not necessarily have to depend only on domestic demand.

Many low- and middle-income countries face the same problem India does: a rare-disease treatment may exist, but its price makes it inaccessible.

If Indian companies can manufacture selected orphan drugs at lower cost, the country could eventually become a supplier to other emerging markets.

That would create an unusual alignment between industrial policy and public health.

India could build a commercially viable manufacturing base while also increasing access to medicines that are currently concentrated in wealthier markets.

But that outcome would depend on pricing, intellectual-property arrangements, regulatory cooperation and reliable procurement. Manufacturing capacity alone would not solve the access problem.

The real opportunity is bigger than another pharma product

India does not need to prove that it can manufacture medicines.

It has already done that.

The harder question is whether it can apply that capability to a category of medicines for which the usual economics do not work well.

Rare diseases expose the limits of a simple market model. The patient population is small, the scientific challenges are substantial, the development costs can be high and the final price can become extraordinary.

That is precisely why the solution cannot sit with one pharmaceutical company.

It requires patients who are identifiable and represented in research, researchers who can conduct credible trials, companies willing to develop and manufacture therapies, regulators able to move applications without unnecessary duplication, and a government willing to create predictable demand where the ordinary market cannot.

There is also a question of benefit-sharing.

If India’s patients, researchers and genetic diversity contribute to the development of a treatment, the country has a legitimate interest in ensuring that the resulting medicine is not merely approved for India but realistically accessible in India and, where appropriate, across the Global South.

That is the point at which rare-disease policy becomes more than a healthcare issue.

It becomes an industrial policy question, a regulatory question and a question about how the economics of pharmaceutical innovation are designed.

India’s next pharmaceutical opportunity may therefore not be about producing more medicines.

It may be about solving a harder problem:

how to make medicines commercially viable when the market is small, without making them financially impossible for the people who need them.

India has spent decades showing the world that medicines can be manufactured affordably.

Rare diseases offer the country a different test: whether it can turn that manufacturing strength, together with research, regulation and public procurement, into meaningful access for patients whose numbers may be small but whose need is not.

What should India prioritise in rare-disease treatment: bringing down the cost of medicines, strengthening government support, or creating stronger incentives for companies to develop and manufacture them locally? Share your perspective in the comments.