Dr Vinod K Paul, Member (Health), NITI Aayog, recently stated that rare diseases are estimated to collectively affect nearly 90 million Indians, making them both a health and societal challenge. He said that with only a fraction of the 7,000 rare diseases having approved therapies, indigenous manufacturing, stronger registries, and multi-ministerial coordination must be prioritised.
He was addressing the National Conference on Rare Diseases organised by FICCI, jointly with NITI Aayog and the Indian Medical Parliamentarians’ Forum (IMPF). Dr Paul underlined that collective resolve must ensure faster diagnosis, equitable treatment, and an improved quality of life. Referring to efforts since 2023, he said that NITI Aayog, experts, and regulators prioritised 13 high-burden disorders, with progress achieved in making five drugs for seven diseases available at significantly reduced prices. He also emphasised collaboration across academia, industry, and government so that quality and affordable treatments can be developed and research can be translated into lasting cures. He noted that the principle of “leave no one behind” must serve as the guiding vision.
Dr Anil S. Bonde, Member of Parliament and Chairperson, Indian Medical Parliamentarians’ Forum, said that though rare diseases are individually small, they impose a severe medical, social, and financial burden. He highlighted the responsibility of policymakers to ensure that no patient or family feels invisible and stressed the role of Parliament in keeping rare diseases firmly on the national health agenda.
Amit Agrawal, Secretary, Department of Pharmaceuticals, Ministry of Chemicals & Fertilisers, Government of India, said that addressing rare diseases must be seen not merely as a medical challenge but as a moral responsibility. He stated that compassion, innovation, and new partnerships are needed. He referred to government-led initiatives as catalysts for building an ecosystem that supports orphan drug development, advanced therapies, and innovative diagnostics. He underlined that affordability must remain central, with policies designed to encourage research while ensuring breakthroughs are made accessible to patients in need.
Amitabh Dube, Co-Chair, FICCI Pharma Committee and Managing Director, Novartis India, opened the deliberations by highlighting the struggles patients face and emphasised that access remains the key challenge.
Dr Jasvantsinh Parmar, Member of Parliament and Joint Convenor, IMPF, chaired a session on Centres of Excellence. He emphasised their critical role in delivering specialised care, research, and training. He urged adoption of hub-and-spoke models and greater integration of patient advocacy groups into Centre of Excellence service delivery.
The conference was structured around three thematic sessions that outlined priorities for rare disease action. In the session on “Research, Development and Advanced Therapies,” the need for expedited regulatory pathways and stronger global and domestic collaborations was emphasised. The “Centres of Excellence: Service Delivery and Partnerships” session highlighted operationalising hub-and-spoke models, establishing national protocols, and integrating patient groups. The “Financing, Procurement and Access Mechanisms” session proposed pooled funding from government, CSR, insurance, and philanthropy, along with outcome-based pricing and centralised procurement to enhance affordability.
It was reinforced that rare care can be realised only when therapies are available and affordable, supported by early diagnosis, awareness, and sustainable partnerships. By bringing together government, parliamentarians, industry, clinicians, and patient advocates, the conference created a platform for consensus-building to drive coordinated action and strengthen India’s rare disease ecosystem.