Generic drugs to treat four rare diseases launched

In a bid to provide relief to rare disease patients across India, the Union Ministry of Health has made available generic medicines to support the care and treatment of four diseases: tyrosinemia type 1, Gaucher disease, Wilson disease, and Dravet-Lennox-Gastaut syndrome. I did it like this. This means that the cost of these drugs will be reduced by 60 to 100 times the current market price.

The ministry is also working to make available treatments for rarer diseases, such as phenylketonuria and hyperammonemia, over the coming months. “Approval of these drugs is awaited,” said health expert VK Paul, member of NITI Aayog. He added that the initiative would also reduce costs for patients from billions to just a few hundred thousand a year. In addition, a formulation for the treatment of sickle cell disease will be made available to children.

A rare disease is a health condition that affects a small number of people and has a particularly low prevalence. The ministry said India could have between 8.4 and 10 million similar cases, as 6-8% of the population in any country is affected at any given time. Almost 80% of these diseases are genetic in nature.

Priority disease

“Special efforts were made to bring in these medicines, and consultations were held with academia, the pharmaceutical industry, organizations, the Medicines Control Agency and the Ministry of Medicines. Thirteen rare diseases were prioritized. We specifically worked on syrups for sickle cell disease and children under five who cannot be given tablets,” said Health Minister Mansukh Mandaviya.

“This project is a purely non-profit project, with the sole motive of providing services to people in urgent need of assistance. “We have been trying to support our patients financially and this is a more sustainable measure not just for India but for patients around the world,” he said.

Dr. Paul added that the ministry is also working with companies that sell patented rare disease drugs. “We are actively looking at how best to treat these drugs for patients who urgently need them,” he explained.

Several companies have come forward to participate in the venture, including Biophore India, Laurus Labs, Azico Biophore, MSN Pharmaceuticals, and Akums Drugs and Pharmaceuticals.