Hyderabad: A webinar organized by the Federation of Asian Biotech Associations-US Chapter highlighted the socio-economic and emotional toll on the 70 million Indians affected by rare diseases (RDs). It sought lessons from global practices, particularly the US. The session titled ‘Global Burden of Rare Diseases: Issues and Challenges’ was addressed by Prof. Ramaiah Muthyala, Founder, Institute for Rare Diseases of India, Hyderabad.
Prof. Muthyala compared India’s nascent RD ecosystem to the US, where the Orphan Drug Act of 1983 spurred progress through incentives, leading to the approval of more than 1,000 treatments. However, these treatments remain prohibitively expensive, such as Zolgensma for spinal muscular atrophy at $2.1 million per patient. In India, costs are compounded by dependence on imports, with drugs like Trientine for Wilson’s disease costing ₹1.6 crore annually, unaffordable for most Indian families earning less than $300 a month.
India faces critical gaps, including an average delay of 7 to 20 years in diagnosis and fragmented infrastructure. Only 14,994 cases are recorded in the National Registry of the Indian Council of Medical Research, much less than the estimated burden. Although the National Policy for Rare Diseases (2021) showed promise, between 2021 and 2024 only 48.7 percent of resources were used for Centers of Excellence.
Despite the challenges, India’s production of 450 active pharmaceutical ingredients through initiatives such as Make in India and the ₹ 15,000-crore Product Linked Incentive Scheme offers hope for reducing costs and improving access. The webinar brought together leading scientists, industry pioneers, and healthcare professionals to address the challenges of RDs in India. The discussion highlighted critical gaps in treatment, research and patient care, while outlining a collective vision for the future.