For the first time, ‘Made in India’ drugs launched for rare diseases

The Centre has come up with a game-changer special initiative by manufacturing four types of homegrown ‘Made in India’ drugs for rare diseases for the first time.
The Central Government has prioritised 13 rare diseases and sickle cell disease. The most awaited initiative was taken in July 2022 after discussions held with the academia, pharma industries, organisations and CDSCO department of pharmaceuticals.
“After the discussion with pharma companies, scientists, drug regulators and academia we decided to initiate manufacture to deliver the drugs. We prioritized drugs for 13 rare diseases and for sickle cell disease also,” official sources from the Union Health Ministry stated.
“This is a revolutionary change with a huge cost difference. If a drug is costing 2.5 crore then in India it will cost 2.5 lakh,” sources said.
All drugs used for treating rare diseases were so far being imported and were expensive. The Union Health Ministry informed that the new drugs will bring down costs by up to 100-fold.
“2 mg tablet that costs 5 lac from Sweden will be available 6500 in India,” sources further said.
A rare disease is a health condition of a particularly low prevalence that affects a small number of people. According to the data shared by the Union Ministry of Health and Family Welfare India could have 8.4-10 crore cases and 80 per cent are genetic conditions.
Currently, there are eight types of generic drugs and among them, four types of drugs are available in India and another four types of drugs will be available next year. The available generic drugs are for Tyrosinemia Type, Gaucher’s Disease, Wilson’s Disease and Dravet or Lennox Gastaut Syndrome- seizures.
The available priority drugs like Nitisinone (capsules) are used for the treatment of Tyrosinemia Type 1 and the cost difference of this drug is 2.2 crore per annum if purchased from other countries but an Indian company is manufacturing this in 2.5 lakh per annum. The drug Eliglustat recommended for the treatment of Gaucher’s Disease will cost India Rs 3-6 lakhs per annum.
The medicine Hydroxyurea which is used for the treatment of Sickle Cell Anemia is also available at low cost. India is boosting domestic production of Hydroxyurea syrup for sickle cell anaemia that will be available at a low price from next year.
India reports approximately 65 cases of Tyrosinemia type 1 rare disease, 235 Gaucher’s disease and a maximum number of cases of Spinal Muscular Atrophy.
India has initiated low-cost drugs for rare disease treatment after studying successful models from other countries