India to manufacture expensive medicines for 13 rare diseases: Govt

India will soon commercially produce medicines for 13 “rare diseases” substantially bringing down the treatment cost for such illnesses - sometimes 90-100 times less – as standard therapies based largely on imported medicines cost a few crores per year, top health officials said here on Friday.

In the first phase, four such medicines have received the marketing approval while another four drugs are under process for approval. These eight medicines will be needed to treat six rare diseases, most of them being genetic in nature.

In the next stage, the Union Health Ministry and the Department of Pharmaceuticals would explore avenues with local pharmaceutical companies to indigenously manufacture expensive and patented gene and enzyme replacement therapies for another six rare diseases, VK Paul, Member (Health) NITI Ayog said here.

While there are many rare diseases, the Union government has picked up the 13 commonest ones for manufacturing indigenous drugs.

In addition, commercial production of paediatric formulation for sickle cell disease – one of the focus areas of the Narendra Modi government in healthcare – has also been included in the programme, facilitated by the government.

“As per the World Health Organisation, any disease whose prevalence is less than one per 1,000 are classified as rare diseases. Cumulatively they may account for 6-8% of the population and there may be 8-10 crore such patients in India. But it is only an estimate as there is no survey for rare diseases,” Paul said.

The treatment for most of these diseases are hugely expensive. For instance the medicines of Tyrosinemia type-1 (disrupts metabolism of tyrosine, damaging kidney, liver and nerves) for a child weighing 10 kg will cost around Rs 2.2 crore in a year while treating Gaucher’s Disease (accumulation of a type of lipid in cells, damaging several organs) in an adult will annually cost Rs 1.8-3.6 crore.

“The indigenous medicines will lower the treatment cost to Rs 2.5 lakh for Tyrosinemia type-1 and Rs 3-6 lakh for Gaucher’s disease. This is within the government’s ability to provide a support of Rs 50 lakh per year to such patients,” Union Health Minister Mansukh Mandaviya said.

Similarly for Wilson’s Disease (excess copper build up in the body), the current therapy costs around Rs 2.2 crore for a kid weighing 10 kg. The home made drugs will lower the treatment cost to Rs 2.2 lakh.

The fourth medicine that received the regulatory approval is for Dravet-Lennox Gastaut Syndrome (types of epilepsy with prolonged seizures). The treatment cost will drop to around Rs 1-5 lakh per annum from the current level of Rs 7-34 lakh.

Four other made-in-India medicines are likely to receive the marketing approval next year.

The companies have also been encouraged to produce hydroxyurea syrup for children below five suffering from sickle cell anaemia. The syrup currently available in the market costs around Rs 70,000 for a 100 ml bottle, whereas the home-grown one will cost around Rs 400. The commercial supply is expected to begin by March 2024.

“This will be a game changer as it was a challenge to make oral suspension of hydroxyurea for a paediatric dose,” said Paul, a former professor of paediatrics at the All India Institute of Medical Sciences, Delhi.

Mandaviya said that the government started working on the plan in July 2022 and had multiple discussions with the industry.