Funding treatment of rare diseases

Source: The post “Funding treatment of rare diseases” has been created, based on “Funding treatment of rare diseases” published in “BusinessLine” on 31st January 2026.

UPSC Syllabus: GS Paper-3-Science and technology

Context: Spinal Muscular Atrophy (SMA) is a rare genetic disorder that causes progressive muscle weakness and can be life-threatening, especially in children. In India, despite being classified as a rare disease, a significant number of patients suffer due to limited access to affordable treatment.

Issues Related to Treatment of SMA

1. High Cost of Treatment: The cost of gene therapy is around ₹17 crore, while branded medicines like Evrysdi cost nearly ₹72 lakh annually, making treatment unaffordable for most families.
2. Limited Coverage Under NRDP: The National Rare Diseases Policy, 2021 provides only ₹50 lakh as one-time assistance, which is insufficient for long-term treatment.
3. Inadequate Implementation: Only a few patients receive financial support, and many remain on waiting lists despite registration.
4. Underutilisation of Legal Provisions: Section 100 of the Patent Act, which allows government use of patented medicines for public interest, has not been invoked.
5. Weak Institutional Response: Centres of Excellence are slow in prescribing affordable generic medicines.
6. Financial Constraints: The government’s crowdfunding fund remains extremely low compared to patient needs.
7. Judicial Uncertainty: Several cases are pending before courts, leading to delays in treatment.

Constitutional and Legal Dimensions

1. Article 21 guarantees the right to life, which includes the right to health.
2. Failure to ensure affordable treatment amounts to deprivation of life.
3. The government has constitutional and legal responsibility to protect citizens’ health.

Way Forward

1. The government should procure generic versions of medicines and provide them free of cost to patients.
2. Section 100 of the Patent Act should be invoked in public interest when required.
3. Financial assistance under NRDP should be enhanced and made flexible.
4. Budgetary allocation for rare diseases must be increased.
5. Indigenous research and manufacturing of rare disease medicines should be promoted.
6. Centres of Excellence must be strengthened and monitored for effective delivery.
7. Courts should issue clear guidelines to protect patients’ right to treatment.

Conclusion: The plight of SMA patients reflects serious gaps in India’s healthcare system for rare diseases. Ensuring affordable treatment is not merely a social obligation but a constitutional duty. Through policy reforms, legal intervention, and public investment, India can uphold the right to life and dignity of SMA patients.

Question:  Spinal Muscular Atrophy (SMA) highlights the challenges of access to affordable healthcare in India. Discuss the issues related to treatment of SMA patients and suggest measures to ensure their right to life and health.

Source: BusinessLine