India commemorated Rare Disease Day 2025 on February 28, emphasizing the need for better policies and patient care.

National Policy for Rare Diseases (NPRD) 2021

• About: The National Policy for Rare Diseases (NPRD) 2021 was introduced after decades of advocacy, providing a framework for treatment and support.
• Efforts: A network of 12 Centres of Excellence (CoEs) across 11 states was established to treat eligible patients.

Challenges in Implementation

• Lack of Funding: Treatment for rare diseases is extremely expensive, making access difficult for most patients. No structured funding mechanism exists to support long-term care.
  • One-time financial assistance of ₹50 lakh for LSD patients is inadequate.
  • Nearly 40 LSD patients have exhausted this support, leaving them without treatment.
  • 30% of children with rare disorders die before their fifth birthday due to lack of treatment.
• Gaps in Access: India has over 450 rare diseases, but treatment is available for less than 50% of them. Lysosomal Storage Disorders (LSDs) are classified under Group 3 (a) disorders, requiring lifelong management.
• Implementation Roadblocks: The Drugs Controller General of India (DCGI) has approved Enzyme Replacement Therapy (ERT) as an effective treatment.
  • MoHFW empanelled 12 CoEs to treat rare disease patients, with 577 LSD patients registered for immediate treatment.
• Lack of structured Monitoring: Despite available therapy and funding, only a fraction of patients receive treatment due to a lack of structured monitoring.
• Delays: Delays in institutional interventions persist despite parliamentary backing.
• Non-Utilization of Funds: ₹143.19 crore sanctioned to 12 CoEs by MoHFW remains underutilized, delaying access to therapies. Lack of accountability and transparency at CoEs exacerbates inefficiencies.
• Exclusion : Conditions like Acid Sphingomyelinase Deficiency (ASMD) are not covered under NPRD 2021, despite clinically approved therapies.
  • 32 ASMD patients in India remain without government funding.

Way Forward

• Sustainable Funding: Establish a dedicated rare disease fund exceeding the current ₹50 lakh limit. Ensure continuous financial assistance for long-term treatments.
• National Programme on Rare Diseases: Implement the ₹974-crore initiative under NPRD 2021 to close treatment gaps. Prioritize children, especially those with LSDs, for timely intervention.
• Equitability: Expedite fund disbursal to CoEs to prevent treatment delays. Implement streamlined operations at CoEs for faster patient care.
• Strengthen Implementation:  Establish a robust monitoring system to track fund utilization and patient treatment. Create an independent oversight body with real-time tracking for accountability.

Conclusion

Urgent reforms are needed to ensure that rare disease patients receive consistent, timely, and equitable access to life-saving treatments. Delays in policy execution equate to denying patients their right to life, particularly innocent children who deserve a future free from uncertainty.

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