Indigenous manufacturing, strong registries, multi-ministerial coordination must prioritised to treat rare diseases: NITI Aayog

New Delhi [India], August 20 (ANI): Dr Vinod K Paul, Member (Health), NITI Aayog, on Wednesday, stated that rare diseases are estimated to collectively affect close to 90 million Indians, making them both a health and societal challenge. He emphasised 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.

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.

Reference was made to the efforts undertaken since 2023, wherein it was shared that NITI Aayog, experts, and regulators prioritised 13 high-burden disorders, with significant progress achieved in making five drugs for seven diseases available at drastically reduced prices.

He also highlighted the importance of collaboration across academia, industry, and government so that quality and affordable treatments can be developed and research can be translated into lasting cures. He further 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, laid focus on rare diseases, though individually small, that 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.

Mr Amit Agrawal, Secretary, Department of Pharmaceuticals, Ministry of Chemicals & Fertilisers, Government of India, emphasised that addressing rare diseases must be seen not merely as a medical challenge but as a moral responsibility. He stated that compassion, innovation, and the courage to forge new partnerships are called for in this context.

Reference was made to government-led initiatives, which were described as catalysts for building an ecosystem that supports orphan drug development, advanced therapies, and innovative diagnostics. He further underlined that affordability must remain central, with policies being designed to encourage research while ensuring that breakthroughs are ultimately made accessible to patients in need.

Mr Amitabh Dube, Co-Chair, FICCI Pharma Committee & MD, Novartis India, opened the deliberations by stressing the extraordinary struggles patients face, emphasising access as the key challenge.

Dr Jasvantsinh Parmar, Member of Parliament & Joint Convenor, IMPF, chaired a session on Centres of Excellence and 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 CoE service delivery.

The conference was structured around three thematic sessions that set priorities for rare disease action. Research, Development and Advanced Therapies, the need for expedited regulatory pathways and stronger global and domestic collaborations was emphasised. Under Centers of Excellence: Service Delivery and Partnerships, operationalising hub-and-spoke models, establishing national protocols, and integrating patient groups were highlighted. The session on Financing, Procurement and Access Mechanisms 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 made available and affordable, supported by early diagnosis, awareness, and sustainable partnerships. By convening government, parliamentarians, industry, clinicians, and patient advocates, a platform for consensus-building was established to drive coordinated action and strengthen India rare disease ecosystem. (ANI)

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