Context:
On Rare Diseases Day, concerns have emerged over ₹271 crore of unspent funds under the National Policy for Rare Diseases (NPRD), even as thousands of patients remain untreated or face interruptions in treatment. The issue has drawn attention to implementation gaps in the policy, with patients awaiting intervention by the Supreme Court to ensure continuity of care.
Key Highlights:
Implementation Challenges in NPRD
- Despite ₹299 crore allocated for rare disease treatment in 2025–26, only ₹30.79 crore has been utilized so far.
- Around ₹271 crore remains unspent, highlighting administrative and implementation delays.
Treatment Interruptions
- Many patients who had begun treatment under NPRD have exhausted the ₹50-lakh funding cap.
- Approximately 100 children who started treatment have reached this cap, leaving their treatment incomplete.
- 8 patients have died while awaiting further financial support.
Patients Awaiting Treatment
- Nearly 2,000 rare disease patients in India are awaiting treatment.
- Around 450 patients suffer from life-threatening Lysosomal Storage Disorders (LSDs) that require continuous treatment.
Case Examples Highlighting the Crisis
- Nayan, diagnosed with MPS-2 (Hunter Syndrome), suffers from stunted growth despite medical care.
- Alishba Khan, a Gaucher disease patient, experienced deteriorating health after government funding stopped in August 2024.
Supreme Court Intervention
- The Supreme Court is expected to examine the issue, especially regarding:
- Continuation of financial support
- Ensuring uninterrupted treatment for rare disease patients.
Significance
- Reveals systemic gaps in policy implementation despite financial allocation.
- Raises concerns about healthcare equity for vulnerable patients with rare diseases.
- Highlights the need for sustainable funding mechanisms for lifelong treatment.
Relevant Prelims Points:
- Rare Disease
- A disease that affects a small percentage of the population.
- Often chronic, genetic, and life-threatening.
- Many require lifelong treatment and specialized care.
- National Policy for Rare Diseases (NPRD), 2021
- Implemented by the Ministry of Health and Family Welfare.
- Objectives:
- Early diagnosis and prevention
- Financial assistance for treatment
- Promotion of research and development
- Provides financial support up to ₹50 lakh per patient for certain rare diseases.
- Categories of Rare Diseases under NPRD
- Group 1: Diseases that can be treated with one-time curative treatment.
- Group 2: Diseases requiring long-term or lifelong treatment with relatively lower cost.
- Group 3: Diseases needing lifelong treatment with very high cost, often requiring specialized therapies.
- Lysosomal Storage Disorders (LSD)
- A group of inherited metabolic disorders caused by enzyme deficiencies in lysosomes.
- Leads to accumulation of toxic substances inside cells.
- Examples:
- Gaucher disease
- Hunter syndrome (MPS-2).
- Rare Diseases Day
- Observed annually on the last day of February to raise awareness about rare diseases.
Relevant Mains Points:
Challenges in Rare Disease Management in India
- High treatment costs
- Many therapies such as enzyme replacement therapy (ERT) cost several crores annually.
- Limited diagnostic capacity
- Rare diseases are often misdiagnosed or diagnosed late.
- Funding and Implementation Gaps
- Despite allocations, delays in fund disbursement and bureaucratic hurdles disrupt treatment.
- Lack of domestic drug manufacturing
- Most treatments rely on expensive imported drugs.
Ethical and Governance Issues
- Ensuring equitable access to healthcare for patients with rare diseases.
- Balancing limited public resources with high treatment costs.
Importance of Strengthening NPRD
- Supports early diagnosis, treatment access, and patient care.
- Encourages research, innovation, and domestic drug development.
Way Forward
- Improve efficient utilization of allocated funds.
- Remove or revise the ₹50-lakh treatment cap for lifelong diseases.
- Strengthen rare disease registries and diagnostic networks.
- Promote domestic production of orphan drugs.
- Enhance public–private partnerships in research and treatment.
UPSC Relevance:
- Prelims: National Policy for Rare Diseases (NPRD), Lysosomal Storage Disorders, Rare Diseases Day.
- Mains: Public health policy, healthcare financing, challenges in rare disease management in India.