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Generic Drugs for Four Rare Diseases

Why is it in the news?

  • The Ministry of Health and Family Welfare has introduced generic drugs for the treatment of four rare diseases.
  • These generic versions of medicines are unbranded and significantly more affordable, with prices around 100 times lower than the current market value of branded drugs.


About the Four Rare Diseases


  • Tyrosinemia-Type 1: An autosomal recessive genetic metabolic disorder characterized by the lack of the enzyme Fumarylacetoacetate hydrolase, which is essential for the final breakdown of the amino acid tyrosine.
  • Gaucher’s Disease: An inherited metabolic disorder characterized by the deficiency of the enzyme glucocerebrosidase, necessary for lipid breakdown.
  • Wilson’s Disease: An autosomal recessive disorder involving abnormal copper accumulation in the body, particularly affecting the brain, liver, and cornea.
  • Dravet or Lennox Gastaut Syndrome – Seizures: A genetic epilepsy syndrome.


Rare Disease

  • Rare diseases, as defined by the World Health Organization (WHO), are often debilitating lifelong disorders with a prevalence of 1 or less per 1000 population.
  • These diseases collectively affect 6-8% of the population in any country and can be genetic in nature.


Challenges Associated with Rare Diseases

  • Late diagnosis due to lack of awareness.
  • Unavailability of treatment options.
  • Prohibitive costs associated with treatment.


Key Initiatives for Rare Diseases

  • National Policy for Rare Diseases (NPRD), 2021: Provides financial support of up to Rs. 50 lakhs to rare disease patients for treatment at designated Centres of Excellence (CoE) mentioned in NPRD.
  • Grants exemption from Basic Customs Duty on rare disease drugs when imported by CoEs or individuals/institutions recommended by CoEs for personal use.
  • Production Linked Incentive Scheme: The Production Linked Incentive Scheme for Pharmaceuticals includes coverage for orphan drugs used in the treatment of rare diseases.

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