The Union Ministry of Health and Family Welfare Monday published a national policy for treatment of rare diseases, listing 450 diseases as rare but not providing a detailed roadmap on treatment.
The policy also intends to kick-start a registry of rare diseases that Indian Council of Medical Research will maintain.
In India, Haemophilia, Thalassemia, Sickle cell anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease and Gaucher’s disease are in the rare diseases list. The latest policy creates three categories of rare diseases — diseases requiring one-time curative treatment, diseases which need long-term treatment but the cost is low, and diseases that require life-long treatment and the cost is high.
The policy states that the Centre will provide assistance of Rs 15 lakh to patients suffering from rare diseases that require one-time curative treatment under the Rashtriya Arogya Nidhi scheme. The treatment is limited to beneficiaries of Pradhan Mantri Jan Arogya Yojana. Some diseases in this category are osteopetrosis, immune deficiency disorders and Lysosomal Storage Disorders.
Manjit Singh, president of Lysosomal Storage Disorder Support Society, said the assistance is “highly inadequate”. “The government claims it will provide financial assistance for children only till the age of two years for lysosomal disorders. Even that amount will not last for more than a month. One-time treatment will not help,” Singh said.
According to Health Ministry, about 95 per cent rare diseases have no approved treatment. “Less than one in 10 patients receives disease-specific treatment,” the policy states.
The Centre first prepared such a policy in 2017, but appointed a committee in 2018 to review it.
Presently, very few pharmaceutical companies manufacture drugs to treat rare diseases and India has no domestic manufacturer. The policy estimates that for a child weighing 10 kg, treatment cost ranges between Rs 10 lakh and Rs 1 crore per year, and the cost rises with age.
The policy has recommended that state governments support patients belonging to the second category of diseases that include Phenylketonuria and Galactosemia, among others. It also recommends crowd funding as a source to fund treatment of rare diseases and advises hospitals to report such cases on digital platforms to gather funds. “The policy is at least beginning of a discussion on rare diseases in the country. But last time they announced a Rs 100-crore corpus fund, now there is no budget. There is no clarity of Centre and State responsibilities and on category III patients,” said Prassana Shirol, founder of Organisation for Rare diseases India.