Prioritize Indigenous manufacturing & multi-ministerial coordination to tackle rare diseases: Dr Vinod K. Paul
New Delhi: Dr. Vinod K. Paul, Member (Health), NITI Aayog, highlighted that rare diseases affect nearly 90 million Indians, posing a significant health and societal challenge. Speaking at the National Conference on ‘Rare Diseases’ organized by FICCI, NITI Aayog, and IMPF, he stressed the urgent need for indigenous manufacturing, robust registries, and multi-ministerial coordination, as approved therapies exist for only a small fraction of over 7,000 rare diseases.
Dr. Paul emphasized the importance of faster diagnosis, equitable access to treatment, and improved quality of life. He noted that since 2023, 13 high-burden disorders have been prioritized, with affordable drugs made available for seven diseases. He also underscored the need for collaboration among academia, industry, and government to develop quality, affordable treatments, guided by the principle of “leave no one behind.”
Dr Anil S. Bonde, Member of Parliament and Chairperson, Indian Medical Parliamentarians’ Forum laid focus on rare diseases, though individually small, 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 & Fertilizers, Government of India emphasized 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.
Amitabh Dube, Co-Chair, FICCI Pharma Committee & MD, Novartis India, opened the deliberations by stressing the extraordinary struggles patients face, emphasizing access as the key challenge.
Dr Jasvantsinh Parmar, Member of Parliament & Joint Convenor, IMPF chaired a session on Centres of Excellence and emphasized their critical role in delivering specialized 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 emphasized. Under “Centers of Excellence: Service Delivery and Partnerships,” operationalizing 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 centralized procurement to enhance affordability. It was reinforced that rare care can be realized 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’s rare disease ecosystem.
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