Relief to patients with rare diseases; production of four generic cost-effective medicines begins in country
New Delhi. The production of four generic cost-effective medicines has begun in the country to bring down the exorbitant cost and provide relief to patients with rare diseases. These drugs are now available in the market for the treatment of four types of rare diseases which are Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease and Dravet-Lennox Gastaut Syndrome, told Union Health Ministry.
NITI Aayog, Member, Health Dr. V.K. Paul said in New Delhi that currently there are eight drugs out of which four are available in the market while the other four are under the process of approval. He informed that these drugs are being made by manufacturers. For the disease Tyrosinemia Type 1, which is characterised by jaundice, liver failure and liver cancer, the drug Nitisinone has been developed which was earlier used to be imported and costs 2.2 crore rupees per year depending on the body weight of the child but this will cut down the cost to 2.5 lakh rupees per annum. The decision is likely to drop prices of treatment anywhere between one-tenth and one-hundredth of current market costs, said the Ministry.
Medicine Eliglustat has been developed for the treatment of Gaucher’s disease, which causes liver or spleen enlargement and acute fatigue. For Wilson’s disease which causes RBC hemolysis, the indigenous drug is Trientine and for the treatment of Dravet or Lennox-Gastaut Syndrome, the fourth medicine which has been made available is Cannabidiol oral solution. Earlier, all drugs used for treating rare diseases were so far being imported and were expensive.
The other four drugs that are under the process of approval are for Phenylketonuria, Hyperammonemia and Gaucher’s disease. A rare disease is a health condition of particularly low prevalence that affects a small number of people. It collectively afflicts 6 to 8 per cent of the population in any country at any given time. According to the Health Ministry, India could have 8.4 crore to 10 crore such cases and nearly 80 per cent of these diseases are genetic in nature. The Government has prioritised 13 rare diseases along with sickle cell anemia.
