Editorial 1 : On Rare Diseases, Falling Short
Context: Inaccessible life-saving drugs and treatments
Constitutional Provisions
- Article 21: Supreme Court recognizes the right to health and medical care as a fundamental right.
- Article 41 (DPSP): Mandates public assistance in cases of sickness and disablement.
National Policy for Rare Diseases 2021 (NPRD)
- Approved on March 30, 2021, after Delhi High Court intervention.
- Key Features
- Financial support capped at ₹50 lakh per patient.
- Directs collaboration with departments (e.g. Pharmaceuticals) to facilitate local drug production.
Judicial Interventions
- Delhi High Court: Criticized the government’s helpless approach and formed a 5-member committee (May 2023) to oversee NPRD implementation.
- Kerala High Court: Ordered continued treatment for a patient who exhausted funds. It was put to stay by Supreme Court after ministry appeal.
Challenges in Addressing Rare Diseases
- High Cost of Treatment
- Example: Spinal Muscular Atrophy (SMA) treatment costs exceed ₹72 lakh annually (risdiplam).
- Financial Cap: NPRD’s ₹50 lakh limit is insufficient, forcing patients to discontinue treatment.
- Inadequate Financial Support
- Ministry cites lack of funds to extend assistance beyond ₹50 lakh.
- Registered Patients: 13,479 in National Registry (likely underreported).
- Patent Barriers and Local Production
- Monopoly Exploitation: Patent holders often refuse to market life-saving drugs in India.
- Generic Potential: First generic versions can reduce prices by 90-95% (e.g. risdiplam, trikafta).
- Implementation Delays: Policy delays and funding restrictions leave patients, including children, without treatment.
Government and Judicial Responses
- Ministry’s Approach
- Reluctance to invoke NPRD’s legal/policy measures (e.g. compulsory licensing).
- Prioritized securing stays on court orders rather than addressing patient needs.
- Court Directives and Stays
- Delhi HC: Advised exploring policy options like price negotiations and local manufacturing.
- Supreme Court: Stayed Kerala HC’s order, urging the ministry to act but no follow-through.
Proposed Solutions and Recommendations
- Promoting Local Drug Production
- Utilize NPRD’s Paragraph 11: Collaborate with Departments of Pharmaceuticals and Industry to boost domestic manufacturing.
- Cost Reduction: Local production can slash prices (e.g. SMA drugs).
- Addressing Patent Monopolies
- Compulsory Licensing: Use provisions under Indian Patent Act to bypass monopolies for public health needs.
- Legal Action: Challenge patent holders refusing to market drugs in India.
- Policy Reforms and Funding
- Increase financial allocation for rare diseases.
- Implement Delhi HC’s suggestions: crowdfunding, CSR partnerships, and price negotiations.
Ethical and Legal Concerns
- Equity in Healthcare Access
- Denial of treatment due to cost violates constitutional rights (Articles 21, 41).
- Impact on Families: Children suffer despite available therapies.
- Government Accountability
- Failure to act on court recommendations raises questions about commitment to public health.
- Conflict: Prioritizing fiscal constraints over fundamental rights.
Way Forward and Conclusion
- Urgent Need: Address funding gaps, leverage generic manufacturing, and enforce patent reforms.
- Role of Judiciary: Courts must ensure policy compliance while balancing executive discretion.
- India’s rare disease policy requires holistic reforms, political will, and ethical prioritization of health rights over administrative inertia.