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Lead: India Cuts Costs for Rare Disease Treatments by Up to 95%
New Delhi, August 2025 — In a landmark policy shift, the Indian government’s Production Linked Incentive (PLI) scheme has slashed the annual cost of crucial rare disease treatments by up to 95%, dramatically improving access for Indian families once facing insurmountable financial barriers. The announcement came during the Rare Diseases Conference 2025 at FICCI auditorium, where senior officials detailed how select therapies—once costing crores annually—can now be accessed for just a fraction of the price.
Background: The Rare Disease Challenge in India
Rare diseases, though individually uncommon, collectively affect an estimated 5% of India’s population—approximately 70 to 90 million people. For decades, Indian patients with conditions such as Gaucher’s disease and Wilson’s disease have struggled with prohibitively expensive treatments, sometimes exceeding ₹1.8–3.6 crore ($220,000–$440,000) per year.
Amit Agrawal, Secretary, Department of Pharmaceuticals, underlined the public health urgency: “Rare diseases are not just a medical problem, they are a question of inclusion and human dignity. No family should be financially or emotionally devastated because of a rare diagnosis”.
Key Developments: Dramatic Price Reductions Thanks to the PLI Scheme
Introduced as a flagship policy to strengthen India’s pharmaceutical sector, the PLI scheme prioritized rare and orphan drugs in its latest phase. The impact has been immediate and profound:
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Eight therapies for rare diseases are now produced domestically under the scheme, including Eliglustat (Gaucher’s Disease), Trientine (Wilson’s Disease), Nitisinone (Tyrosinemia Type 1), and Cannabidiol (Lennox–Gastaut Syndrome).
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Cost reductions are tangible: The annual price for Gaucher’s disease treatment fell from ₹1.8–3.6 crore to just ₹3–6 lakh—a staggering drop of more than 90%.
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Additional drugs are being developed for conditions such as Thalassemia, Cystic Fibrosis, and more, with four new therapies expected soon.
“The launch of local generic medications has led to immense savings for patients battling rare diseases,” said Dr. VK Paul, Member (Health), NITI Aayog, highlighting that India’s policies now directly align with global priorities for rare disorders.
Expert Perspectives: Industry and Advocacy Voices
While government leaders paint a promising picture, independent experts and patient advocates offer nuanced commentary:
“This is a promising step toward universal health for rare disease patients. However, while small molecule therapies like Eliglustat are now available at much lower prices, patients needing advanced therapies—such as biologics or gene therapy—still face serious access and cost hurdles,” says Dr. Anuja Agarwal, Clinical Geneticist, not affiliated with the PLI scheme rollout.
Industry leaders echo this balanced view. Amitabh Dube, MD & Country President of Novartis India, points out that:
“Rare diseases comprise a silent epidemic affecting over 300 million globally. Sustainable financing models are crucial if we want to make high-cost, advanced treatments accessible to every patient in need.”
Policy Context & Public Health Implications
India’s rare disease strategy has evolved rapidly:
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The National Policy for Rare Diseases (NPRD), launched in 2021, now covers 63 conditions and designates 13 national Centres of Excellence, including new sites at institutions like AIIMS Patna.
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More than 1,100 patients have reportedly benefited from therapy subsidies as of August 2024.
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The current cap on patient financial support stands at ₹50 lakh, but advocacy groups say this remains inadequate for high-cost, novel therapies.
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The government’s new research and innovation framework (PRIP) proposes further incentives for developing orphan and advanced therapies, aiming to bring more novel treatments to the Indian market in the next few years.
Limitations and Counterpoints
Despite significant headway, several challenges persist:
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Therapy Scope: The PLI scheme primarily supports drugs no longer under patent (small molecules), while the greatest need may soon be with innovative biologics and gene therapies.
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Geographic Access: Access to expert care and subsidized medication is mostly limited to major centres. Patients in rural regions remain underserved.
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Financial Cap: With some innovative treatments costing over ₹50 lakh, even after support, many families still face out-of-pocket burdens, especially for therapies not yet included in current support policies.
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Sustainable Supply: Continued investment and regulatory innovation will be required to ensure supply, especially for newer, complex treatments.
What This Means for Patients and Families
For many Indian families, these developments offer genuine hope:
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Conditions once considered “death sentences”—due to prohibitive treatment costs—now have practical, accessible, and affordable solutions.
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The new policies mark a paradigm shift from imported medicines to a self-reliant, domestic pharmaceutical ecosystem.
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Patients and caregivers are advised to consult the official Centres of Excellence for updated lists of supported therapies, and to explore new support mechanisms under the evolving PLI and PRIP frameworks.
“This is only the beginning. As new therapies are developed and more diseases are included under national coverage, the hope is for all rare disease patients in India to eventually have access to effective, affordable care,” says Dr. Suresh Menon, President, Indian Society for Rare Diseases.
Medical Disclaimer
Medical Disclaimer: This article is for informational purposes only and should not be considered medical advice. Always consult with qualified healthcare professionals before making any health-related decisions or changes to your treatment plan. The information presented here is based on current research and expert opinions, which may evolve as new evidence emerges.
References
This article adheres to strict standards of responsible, evidence-based health reporting and aims to empower readers and healthcare professionals with objective, up-to-date information on this key medical policy development.
