- Recently the Union Ministry of Health and Family Welfare published a national policy for treatment of rare diseases, listing 450 diseases as rare but not providing a detailed roadmap on treatment.
- 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.
- 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
Details about the National Policy
- 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.
- 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 also intends to kick-start a registry of rare diseases that Indian Council of Medical Research will maintain.