• Providing relief to patients with rare diseases across India, the Union Health Ministry has made available generic drugs to support the care and treatment of four ailments– Tyrosinemia-Type 1, Gauchers Disease, Wilson’s Disease, and the Dravet-Lennox Gastaut Syndrome.
• This means that the cost of these drugs will be slashed by anywhere between 60 and 100 times of their current market value.

Disease	Impact	Drop in cost
Tyrosinemia type 1		used to require an annual expenditure of ₹ 2.2 crore to ₹ 6.5 crore, now costs ₹ 2.5 lakh for the same duration. If untreated, a child dies of the disease by the age of 10. The drug for treating the disease is called Nitisinone.
Gaucher’s Disease	results in liver or spleen enlargement, bone pain and fatigue	Eliglustat capsules- cost brought down from ₹ 1.8-3.6 crore per year to ₹ 3.6 lakh
Wilson’s Disease	causes a copper deposit in the liver and psychiatric symptoms	Trientine capsules- from ₹ 2.2 crore to ₹ 2.2 lakh per year
Dravet/ Lennox Gastaut Syndrome	causes complex seizure syndromes	Cannabidiol oral solution, from ₹ 7-34 lakh per year to ₹ 1-5 lakh.

ABOUT RARE DISEASES

• A rare disease is a health condition of particularly low prevalence that affects a small number of people.
• WHO defines rare disease as often debilitating lifelong disease or disorder with a prevalence of 1 or less, per 1000 population.
• There are 6,000-8,000 classified rare diseases, but less than 5% have therapies available to treat them.
• It collectively afflicts 6-8% of the population in any country at any given time, so India could have 8.4 crore to 10 crore such cases, according to the Ministry.
• Nearly 80% of these diseases are genetic, which means that symptoms are seen, and require treatment, at an early age.