Answer

Rare diseases, often defined as conditions affecting fewer than 1 in 2,500 individuals, collectively impact over 70 million people in India. Despite their low individual prevalence, their cumulative burden is significant. In 2021, India launched the National Policy for Rare Diseases, yet concerns remain around regulatory clarity, affordability of orphan drugs, and incentivizing research.

Positives of Policy and Regulatory Framework in India for Rare Diseases

• Constitutional Backing: The right to health under Article 21 strengthens the legal foundation for rare disease care, ensuring health is treated as a fundamental right.
  For example: In Paschim Banga Khet Mazdoor Samity v. State of West Bengal (1996), the Supreme Court emphasized that the state must provide timely medical treatment to protect the right to life.
• Dedicated National Policy: The National Policy for Rare Diseases (NPRD) 2021 provides a structured approach to classifying and managing rare diseases in India.
  For example: The policy classifies rare diseases into three groups and outlines the mechanism for financial assistance, offering up to Rs 50 lakh for specific treatments.
• Registry for Patients: The National Registry for Rare and Other Inherited Disorders helps in collecting data for effective planning and intervention strategies.
• Judicial Interventions: Indian courts have actively ensured policy implementation and directed the government to act swiftly in individual cases of rare disease treatment.
  For example: The Delhi High Court ordered the formation of a committee to oversee NPRD implementation and highlighted the need for government action in rare disease cases.
• Provision for Local Production: NPRD’s implementation strategy directs MoHFW to coordinate with industrial departments to promote domestic manufacturing of orphan drugs.
  

Limitations of Policy and Regulatory Framework in India for Rare Diseases

• Inadequate Funding Support: The financial cap of Rs 50 lakh per patient is often insufficient for lifelong treatments of high-cost rare diseases like Spinal Muscular Atrophy.
  For example: A child undergoing treatment with risdiplam, costing Rs 72 lakh annually, had to discontinue due to exhausted NPRD funds and lack of extended aid.
• Slow Policy Implementation: Policy measures often come only after court intervention, reflecting bureaucratic apathy and delays in government response.
  For example: The MoHFW approved the NPRD only after a Delhi HC directive in 2021, showing lack of proactive governance.
• Legal Ambiguity on Patents: India’s current framework does not aggressively tackle patent monopolies that block the production of affordable generics for rare diseases.
  For example: Patentees often refuse to market essential drugs like risdiplam in India, making them unaffordable and inaccessible to most patients.
• Lack of Incentives for Innovation: There is minimal government support for R&D in orphan drugs, limiting India’s potential to develop innovative, affordable treatments locally.
• Poor Awareness and Identification: Most rare disease patients remain undiagnosed or unregistered, making it difficult to assess the real burden and provide timely support.

How India Can Balance Affordability, Accessibility, and Innovation

• Enforce Compulsory Licensing: The government should use compulsory licensing provisions under the Patents Act to allow generic production of high-cost rare disease drugs.
  For example: In 2012, India granted Natco Pharma a compulsory license to produce Nexavar, slashing the cost by over 95% for liver cancer patients.
• Public-Private Partnerships (PPPs): Encourage collaborations between government, academia, and industry for affordable R&D and domestic manufacturing of orphan drugs.
  For example: A PPP model like the one used for COVAXIN can be replicated for rare disease drugs, reducing costs and accelerating innovation.
• Tax and Regulatory Incentives: Introduce financial incentives, such as tax deductions, market exclusivity, and fast-track approvals for firms developing orphan drugs.
• Expand Insurance and CSR Funding: Include rare disease treatment in public and private insurance schemes and promote CSR funding from corporates to fill financial gaps.
  For example: In 2022, the Kerala HC directed the Centre to fund rare disease treatment through CSR and crowdfunding, showcasing alternative funding models.
• Strengthen Medical Infrastructure: Invest in diagnostic centres, training for healthcare workers, and public awareness campaigns to ensure early diagnosis and intervention.
  For example: The All India Institute of Medical Sciences (AIIMS) can be upgraded into a national centre for rare diseases, aiding early detection and better treatment outcomes.

In order to provide systemic support for diagnosis, treatment, and research, a strong rare disease policy must go beyond tokenism. By encouraging public-private partnerships, providing incentives for the development of orphan drugs, incorporating rare diseases into primary healthcare, and increasing financial risk protection, India can strike a balance between affordability, accessibility, and innovation, leading to the transformation of care into inclusive and equitable.

DOWNLOAD PDF