NEW DELHI: The centre on Friday announced that India-made drugs for 14 rare diseases, which afflict 6-8 per cent of India’s population, will be available soon.
Apart from 13 rare diseases, whose treatment will now become cost-effective, drugs for sickle cell, will also be made available, Union Health Minister Mansukh Mandaviya announced on Friday.
According to estimates, India could have 8.4-10 crore of such rare diseases also cases.
All drugs for rare diseases were being imported and ran into lakhs and crores and parents of children afflicted with these rare diseases were totally dependent on crowdfunding.
Now, with the launch of these India-made drugs, the cost will come down a hundredfold. The centre also provides aid of Rs. 50 lakh per year for such diseases, but its reach is very limited.
While the first of the four India-made cost-effective generic drugs will soon be available, four others will be launched next year.
According to NITI Ayog member Dr. V K Paul, out of the 14 rare diseases, they prioritised six diseases, which included Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, Dravet/ Lennox Gastaut Syndrome related seizures, Phenylketonuria and Hyperammonemia.
Paul said four drugs have been approved and will soon be available in the market for four rare diseases, which include Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet/ Lennox Gastaut Syndrome-related seizures.
He added that one drug for Phenylketonuria, two for Hyperammonemia, and another medicine for Gaucher’s Disease is under process for approval.
Terming the launch of these India-made drugs as a “game changer,” Paul said this will bring a paradigm shift in treating rare diseases globally, especially in low-income countries.
Mandaviya said that India was able to develop drugs for these rare diseases because the central government took a special initiative.
“Discussions were held with academia, pharma industries, organisations, CDSO (Central Drugs Standard Control Organisation), and the Department of Pharmaceuticals to launch these medicines,” the minister said.
He said, “India is one of the best cost-effective and efficient drug manufacturers in the world. We are the pharma of the world. We have been able to develop drugs for such rare diseases for which treatment was very expensive. I am sure drugs for these rare diseases will be in demand globally.”
Rare disease is a condition of a particularly low prevalence that affects a small number of people.
“Over 80 per cent are genetic conditions that affect in childhood at varying ages and is lifelong,” he added.
Paul said that the process to develop these drugs began last year in July.
One of the rare diseases for which the generic drug will be available is Tyrosinemia Type 1. In this rare disease, a child dies in 10 years and its treatment runs into crores. The imported drug that is available now is Nitisinone, which costs Rs 2.2- 6.5 crore per year depending on the body weight of the child. Now, the generic version which will be launched by Laurus Labs and Zenara Pharma will bring down the cost to Rs 2.5 lakh per annum.
The other rare diseases for whose Indian pharma companies are working are Cystic Fibrosis, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy (DMD), Achondroplasia, Pompe’s disease, Mucopolysaccharidosis, and Nieman Pick disease. The last one is sickle cell disease for which the government has launched a national programme.
Paul said the generic drugs for these rare diseases will be available in Centre for Excellence Centres and also Jan Aushadhi Kendras.