At the National Conference on Rare Diseases, organized by FICCI in partnership with NITI Aayog and the Indian Medical Parliamentarians’ Forum (IMPF), leaders from government, industry, and academia underscored the urgent need for coordinated action to address the growing challenge of rare diseases in India.
Dr Vinod K Paul: 90 Million Indians Affected by Rare Diseases
Dr Vinod K Paul, Member (Health), NITI Aayog, stated that rare diseases collectively affect nearly 90 million Indians, posing a significant health and societal challenge. With only a fraction of the 7,000 rare diseases having approved therapies, he urged India to strengthen indigenous drug manufacturing, build robust registries, and enhance multi-ministerial coordination.
He emphasized that collective resolve must deliver faster diagnosis, equitable treatment, and better quality of life. Referring to efforts since 2023, Dr Paul noted that 13 high-burden disorders were prioritized. He added that significant progress was made in making five drugs for seven diseases available at drastically reduced prices. He called for deeper collaboration across academia, industry, and government so that research can translate into affordable, lasting cures, guided by the principle of “leave no one behind.”
Parliamentarians Stress Policy Responsibility
Dr Anil S. Bonde, Member of Parliament and Chairperson, IMPF, highlighted that although rare diseases affect small patient groups individually, they collectively impose a severe medical, social, and financial burden. He underlined the duty of policymakers to ensure no family feels invisible and reaffirmed Parliament’s role in keeping rare diseases on the national health agenda.
Government Calls for Innovation with Compassion
Mr Amit Agrawal, Secretary, Department of Pharmaceuticals, Ministry of Chemicals & Fertilizers, stressed that addressing rare diseases is not just a medical challenge but a moral responsibility. He called for compassion, innovation, and bold partnerships to support orphan drug development and advanced therapies and also emphasized the need for innovative diagnostics.
He highlighted that government-led initiatives act as catalysts in building a supportive ecosystem and reminded stakeholders that affordability must remain central. Policies, he noted, must balance research incentives with ensuring breakthroughs are accessible to all patients.
Industry Voices Patient Struggles
Opening the deliberations, Mr Amitabh Dube, Co-Chair, FICCI Pharma Committee & MD, Novartis India, highlighted the extraordinary struggles patients face. He noted that access to therapies remains the greatest challenge in rare disease care.
Centres of Excellence as Critical Hubs
Dr Jasvantsinh Parmar, Member of Parliament and Joint Convenor, IMPF, chaired a session on Centres of Excellence (CoEs), highlighting their critical role in specialized care, research, and training. He urged the adoption of hub-and-spoke models to expand the reach of CoE services. He also called for stronger integration of patient advocacy groups to improve their effectiveness.
Thematic Sessions Define Priorities for Action
The conference featured three thematic sessions, each setting clear priorities for strengthening India’s rare disease response:
- Research, Development, and Advanced Therapies – Speakers called for expedited regulatory pathways and stronger global and domestic collaborations. They also emphasized the rapid translation of research into therapies.
- Centres of Excellence: Service Delivery and Partnerships – Participants emphasized hub-and-spoke models and national treatment protocols. They also highlighted the importance of meaningful engagement of patient groups.
- Financing, Procurement, and Access Mechanisms – Proposals included pooled funding from government, CSR, insurance, and philanthropy. They also suggested outcome-based pricing and centralized procurement to improve affordability.
Building a Consensus for Rare Care in India
As per the press release, the deliberations reinforced that rare disease care can succeed only when therapies are affordable. Early diagnosis must be widespread, and partnerships need to be sustainable. The conference brought together policymakers, parliamentarians, industry, clinicians, and patient advocates to create a platform for consensus-building. It also outlined a roadmap to strengthen India’s rare disease ecosystem.