Rare-Disease Drugs: Making India the Next Frontier in Affordable Healthcare

1. Scale of the Problem

• Around 8,000 rare diseases are known globally, collectively affecting hundreds of millions of people.
• India has reported 1,004 rare genetic disorders so far.
• Since individual patient populations are very small, such medicines are called “orphan drugs”, as conventional markets provide limited commercial incentives.
• Existing medicines address only around 5% of rare diseases, leaving most patients without effective treatment.
• India provides financial assistance of up to ₹50 lakh per patient under its rare-disease support framework, but this can be inadequate because some therapies cost several crores.

2. Why Rare-Disease Drugs Remain Expensive

• Pharmaceutical companies face two major challenges:
• Finding sufficient patients for clinical trials.
• Establishing reliable clinical endpoints for extremely rare conditions.
• Small patient populations make recovery of R&D costs difficult.
• Advanced treatments such as gene therapy and personalised therapies can involve very high upfront costs.
• Even after approval, manufacturing medicines in small quantities and maintaining specialised distribution networks can increase costs.
• The US Orphan Drug Act, 1983 addressed the problem through tax incentives, grants, fee waivers and market exclusivity, contributing to a substantial increase in orphan-drug approvals.

3. India's Potential Advantage

• India's population of over 1.4 billion provides a large absolute patient pool for studying even very rare conditions.
• Certain communities with higher levels of endogamy can have greater prevalence of particular genetic disorders, creating opportunities for genetic research.
• India's pharmaceutical ecosystem provides:
• Large-scale manufacturing capacity.
• Skilled scientific manpower.
• Lower production costs.
• Experience in generics and biosimilars.
• India supplies a significant share of global generic medicines and has extensive manufacturing facilities approved by international regulators.
• Indian facilities have also developed advanced therapies such as CAR-T cell therapy, demonstrating growing capabilities beyond conventional generics.

4. Regulatory and Policy Opportunity

• Rule 101 of the New Drugs and Clinical Trials Rules, 2019 provides a regulatory pathway for certain drugs already approved in specified countries, allowing them to avoid repeating an Indian clinical trial in eligible cases.
• Greater clarity and transparency in the approval process could encourage companies to conduct rare-disease research and manufacturing in India.
• India can promote collaboration between multinational pharmaceutical companies, Indian firms, hospitals, research institutions and patient groups.
• Government procurement can reduce market uncertainty and make investment in orphan drugs more commercially viable.

5. Way Forward: From Subsidy to Sustainable Access

• Introduce PLI-type incentives for orphan-drug manufacturing.
• Provide tax incentives and support for technology transfer from national laboratories and global companies.
• Use Advance Market Commitments (AMCs), where the government commits to purchasing a specified quantity at an agreed price.
• Strengthen distribution through Centres of Excellence for Rare Diseases, CGHS and Jan Aushadhi Kendras.
• Encourage exports of affordable rare-disease medicines to low- and middle-income countries.
• Develop a benefit-sharing model so that Indian patients who contribute data and participate in trials can obtain affordable access to the resulting therapies.
• The broader objective should be to de-risk initial investment rather than permanently subsidise companies, allowing a competitive rare-disease pharmaceutical ecosystem to emerge.