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Generic Drugs for Rare Disease Cure

 

Source: Th

 Context: The Union Health Ministry in India has introduced generic drugs to treat four rare diseases, namely Tyrosinemia-Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet-Lennox Gastaut Syndrome.

 

What are Generic drugs?

Generic drugs are medications that are equivalent to brand-name drugs in terms of active ingredients, dosage, safety, strength, quality, and intended use but are typically sold at a lower cost. Once the patent protection for a brand-name drug expires, other pharmaceutical companies can produce and sell generic versions.

  • The price of these unbranded generic versions of medicine for the four rare diseases will be 100 times lower than the current market value of the branded drugs.

 

What are Rare Diseases?

WHO defines rare diseases as debilitating lifelong conditions with a prevalence of 1 or less per 1000 population. They collectively affect 6-8% of India’s population ( about 8 to 10 crore cases in India), with nearly 80% being genetic.

The National Policy for Rare Diseases (NPRD), 2021, provides financial support up to Rs. 50 lakhs for treatment, exempts Rare Diseases drugs from Basic Customs Duty and includes orphan drugs in the Production Linked Incentive Scheme for Pharmaceuticals.

 

About the four diseases:

Rare Disease Description Impact on the Body
Tyrosinemia-Type 1 An inherited metabolic disorder where the body lacks the enzyme (Fumarylacetoacetate hydrolase) needed to break down the amino acid tyrosine. Can lead to the accumulation of harmful substances, affecting the liver, kidneys, and nervous system.
Gaucher’s Disease An inherited metabolic disorder characterized by a deficiency of the enzyme (glucocerebrosidase) necessary for breaking down certain lipids. Results in the buildup of fatty substances in organs, primarily impacting the spleen, liver, and bone marrow.
Wilson’s Disease A rare autosomal recessive disorder where abnormal copper accumulation occurs, primarily affecting the brain, liver, and cornea. Excess copper can lead to liver and neurological problems, including cognitive decline and psychiatric symptoms.
Dravet or Lennox Gastaut Syndrome (seizures) A genetic epilepsy syndrome marked by severe seizures, typically starting in infancy. It can lead to developmental delays and cognitive impairment. Frequent and prolonged seizures can hinder cognitive development, impacting motor skills, language, and overall cognitive function.