India is making significant progress in addressing rare disease treatment gaps, with five medications for seven conditions now available in the market and four additional drugs expected to launch soon, according to NITI Aayog member (health) Dr VK Paul.
Speaking at a recent event, Dr Paul highlighted that generic medications launched by local companies have generated substantial cost savings for patients suffering from rare diseases. The progress represents a major advancement in India's rare disease treatment landscape, where patients have historically faced limited therapeutic options and high treatment costs.
Current Treatment Landscape
The National Policy for Rare Diseases currently encompasses 63 rare diseases, as recommended by the Central Technical Committee for Rare Diseases (CTCRD). Among these, seven diseases have seen significant therapeutic progress, including Thalassemia, Wilson's disease, and Cystic Fibrosis, with five corresponding medications now commercially available.
NITI Aayog had strategically shortlisted 13 disorders as priority conditions in 2023, focusing development efforts on diseases with the greatest patient impact and therapeutic potential.
Government Support and Manufacturing
The government's Production Linked Incentive (PLI) scheme for pharmaceuticals has successfully supported the production of drugs treating eight rare diseases, according to Department of Pharmaceuticals Secretary Amit Agrawal. The initiative positions rare diseases and orphan drugs as strategic priority areas deserving enhanced policy support.
This manufacturing support addresses a critical gap in India's pharmaceutical ecosystem, where rare disease treatments were previously dependent on expensive imports or unavailable entirely.
Market Impact and Patient Burden
Rare diseases affect over 300 million people globally, with an estimated 70-90 million patients in India alone, according to Novartis MD & Country President Amitabh Dube. This substantial patient population underscores the importance of India's expanding rare disease treatment pipeline.
The availability of locally manufactured generic alternatives has created "immense savings" for patients, addressing one of the primary barriers to rare disease treatment access in the country.
Industry Challenges and Future Outlook
Despite the progress achieved, significant challenges remain in rare disease management. Dube emphasized the need for continued efforts to create "an ecosystem that encourages and enables R&D, reduces regulatory delays and values innovation so treatments can reach Indian patients faster."
The industry leader also advocated for sustainable financing solutions to ensure patient access to emerging advanced therapies, particularly those exceeding ₹50 lakh in cost. This financial consideration remains crucial as newer, more sophisticated treatments enter the market.
The combination of government policy support, manufacturing incentives, and industry collaboration appears to be creating momentum in India's rare disease treatment sector, potentially transforming outcomes for millions of affected patients across the country.
