National Policy for Rare Diseases, 2021 – UPSC GS2

Rare Disease:
  • A rare disease is a health condition of particularly low prevalence that affects a small number of people compared with other prevalent diseases.
  • Haemophilia, Thalassemia, sickle-cell anaemia, primary immunodeficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain types of muscular dystrophies are the most common types of rare diseases recorded in India.
National Policy for Rare Diseases, 2021:
  • The National Policy for Rare Diseases, 2021 was notified on the 31st of March 2021.
  • The policy comes over three years after the health ministry formulated a National Policy for Treatment of Rare Diseases in July 2017 but kept it in abeyance.
  • India plans to create a national registry for a database on rare diseases, classify known rare diseases in three groups.
  • In the new policy, ‘rare diseases’ have not been defined but classified into three groups based on expert opinion.
    • Group 1 has disorders controllable by one-time curative treatment, including osteopetrosis and Fanconi anaemia.
    • Group 2 has diseases requiring long-term or lifelong treatment with a relatively lower cost of treatment and benefit has been documented in literature, including galactosemia, severe food protein allergy, and homocystinuria.
    • Group 3 has diseases for which definitive treatment is available, but challenges are to make optimal patient selection for benefit, and very high cost and lifelong therapy, covering diseases such as spinal muscular atrophy (SMA), Pompe disease, and Hunter syndrome.
  • The policy says the government would provide financial assistance of up to Rs 20 lakh to poor patients and those covered under Ayushman Bharat for diseases under Group 1.
  • The state governments would be asked to undertake treatment of diseases covered under Group 2, which largely include disorders managed with special dietary formulae or food for special medical purposes (FSMP) and disorders that are manageable to other forms of therapy.
  • For diseases classified under Group 3, which require life-long expensive treatments, the government would create a digital platform to bring together Centres for Excellence, patients undergoing treatment and corporate donors or prospective voluntary individuals who could help fund treatment.
  • The government has identified eight Centres of Excellence for the treatment of rare diseases.
  • Though the document specifies increasing the government support for treating patients with a rare disease from ₹15 lakh to ₹20 lakh — caregivers say this doesn’t reflect the actual costs of treatment.
  • It is argued that the new policy offers no support to patients awaiting treatment since the earlier National Policy for Treatment of Rare Diseases 2017 was kept in abeyance.
  • Concerns have been raised about the new policy not considering Group 3 patients, who require lifelong treatment support.
  • It has been demanded that at least two centres in every state need to be identified as a centre of excellence to help the patients avoid the time and cost of moving out.

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