The much awaited Rare Diseases Policy of the Health and Family Welfare (MoHFW) has proposed financial support of up to ₹15 lakh under the Rashtriya Arogya Nidhi for the treatment of rare diseases that require one-time treatment, such as stem cell transplant and organ transplants.

The draft policy states the scheme will cover only patients to whom the PM Jan Arogya Yojana (PM-JAY) or Ayushman Bharat, which provides cashless health cover of ₹5 lakh per annum per family, is applicable.

Rare diseases categorised under Group I by MoHFW —such as severe combined immunodeficiency, Fanconi anaemia, disorders of the liver like maple syrup urine disease, urea cycle disorders, Fabry’s disease, and conditions found suitable for stem cell, liver or kidney transplant — will receive funding under the scheme.

However, rare diseases that require lifelong expensive treatment ranging from ₹10 lakh to ₹1 crore annually will not be funded by the Centre. These include Gaucher disease, Hurler syndrome, Hunter syndrome, Pompe disease, Fabry disease before damage to kidney, spinal muscular atrophy and so on. that But, for these, MoHFW has proposed an alternative funding mechanism by setting up a digital platform for voluntary and corporate donations.

“Keeping in view the resource constraints and competing health priorities, it will be difficult for the government to fully finance the treatment of rare diseases,” said the draft policy.

The MoHFW formulated a National Policy for the Treatment of Rare Diseases in July 2017, but had to withdraw it due to implementation challenges. A corpus of ₹100 crore was announced to fund medical care for patients, but this never found a mention in the MoHFW’s budget.

“Issues such as cost effectiveness of interventions for rare diseases versus other health priorities, sharing of expenditure between the Centre and States and flexibility of the State governments in changing their policies were raised on the 2017 policy. So an expert committee was constituted in November 2018 to reframe the policy,” stated the new draft.

There are 7,000-8,000 rare diseases, but less than 5 per cent have treatments and therapies.

Countries such as the US, the UK, Germany, Canada, Malaysia, Australia and Singapore provide subsidies for patients to buy drugs for rare diseases.

“Due to high costs, the government has not been able to provide these drugs for free,” the MoHFW draft said.

Dearth of data

The draft also there is a dearth of data in India on how many people suffer from diseases that are considered rare. So far, 450 such diseases have been recorded in India, including genetic ones. These include haemophilia, thalassemia, sickle-cell anaemia, primary immuno deficiency in children, auto-immune diseases, lysosomal storage disorders such as Pompe, Hirschsprung, Gaucher’s, cystic fibrosis, hemangioma and certain forms of muscular dystrophies. “The Indian Council of Medical Research is making a registry of rare diseases in the context of Indian conditions,” the draft said.

The MoHFW has prepared the draft ahead of a Delhi High Court hearing on January 14, in which the Centre has been dragged into a case asking for to frame policies to support and fund expensive patient treatments.