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National Policy For Rare Diseases 2021 : Important Points

National Policy For Rare Diseases, 2021:

Caregivers to patients with ‘rare diseases’ and affiliated organizations are dissatisfied with the National Policy for Rare Diseases, 2021.

  • The policy specifies increasing the government support for treating patients with a ‘rare disease’— from ₹15 lakh to ₹20 lakh. But, caregivers say this doesn’t reflect actual costs of treatment.
  • Advocacy groups, however, have expressed concerns about the lack of funding support in the policy for patients diagnosed with life-threatening rare, genetic disorders.

 National Policy for Rare Diseases, 2021:

  • Patients of rare diseases will soon be eligible for a one-time treatment under the Ayushman Bharat Pradhan Mantri Jan Arogya Yojana (AB-PMJAY).
  • Beneficiaries for financial assistance would not be limited to below poverty line (BPL) families, but extended to about 40% of the population, who are eligible as per 23 norms of Pradhan Mantri Jan Arogya Yojana (PMJAY), for their treatment in Government tertiary hospitals only.
  • The policy has categorized rare diseases into three groups – disorders amenable to one-time curative treatment; those requiring long-term or lifelong treatment; and diseases for which definitive treatment is available but challenges are to make an optimal patient selection for benefit.

Rare disease:

  • A rare disease also referred to as an orphan disease, is any disease that affects a small percentage of the population.
  • Most rare diseases are genetic, and are present throughout a person’s entire life, even if symptoms do not immediately appear.
  • The commonly reported rare diseases include:
    • Primary immunodeficiency disorders,
    • Lysosomal storage disorders (Gaucher’s disease, Mucopolysaccharidoses, Pompe disease, fabry disease etc.)
    • small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias, etc.),
    • cystic fibrosis, osteogenesis imperfecta, certain forms of muscular dystrophies, and spinal muscular atrophy.