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.
- 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.