Context:

• Indian pharmaceutical firms have initiated the production of medications for 4 rare diseases, significantly reducing costs of drugs. 

Made in India Drugs for Rare Diseases

• Medications: The manufacturing started for at least four conditions including Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet-Lennox Gastaut Syndrome. They  are likely to become available in early 2024.
  • At present, medicines are available at the centres of excellence for rare diseases. 
  • Government is looking at the feasibility of making it available at Jan Aushadhi stores. 
• PLI Scheme: The government is working on a production-linked incentive (PLI) scheme for these medicines and is also promoting research on indigenous therapies for these conditions.
• Other Medications: Government also urged the industry to produce an oral solution for hydroxyurea. 
  • It is needed for the treatment of children up to the age of 5 years suffering from sickle cell disease.

What is the Rare Disease?

• World Health Organisation (WHO) defines rare disease as a  lifelong disease or disorder with a prevalence of 1 or less, per 1000 population. 
• Common Rare Diseases: They include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Hirschsprung disease, and certain forms of muscular dystrophies.
• Rare Disease in India: Around 6% to 8% of the Indian population is estimated to have a rare disease.
• Rare Disease Policy: Ministry of Health and Family Welfare has formulated a National Policy for Treatment of Rare Diseases (NPTRD) in July, 2017. 
  • The policy aims at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy.

News Source: Indian Express