India is making significant progress in tackling rare diseases, with five new drugs already available for seven illnesses and four more therapies in the pipeline, according to Dr. VK Paul, Member (Health), NITI Aayog.
Speaking at an event, Dr. Paul highlighted that locally developed generic medications have led to substantial cost savings for patients battling rare diseases. Currently, 63 rare diseases are covered under the National Policy for Rare Diseases, based on recommendations from the Central Technical Committee for Rare Diseases (CTCRD).
He noted that major strides have been made in treating conditions such as Thalassemia, Wilson’s disease, and Cystic Fibrosis, while new therapies are being developed for additional disorders. In 2023, NITI Aayog identified 13 priority rare diseases to fast-track treatment options.
Amit Agrawal, Secretary, Department of Pharmaceuticals, said the government’s PLI scheme has supported domestic production of drugs for eight rare diseases, reflecting its strategy to prioritize orphan drugs.
Adding perspective, Amitabh Dube, MD & Country President, Novartis India, stressed that rare diseases impact over 300 million people globally, including 70–90 million in India, and called for sustainable financing models to make advanced therapies (often costing above ₹50 lakh) accessible to patients.