Caregivers to patients with ‘rare diseases’ and affiliated organisations are dissatisfied with the National Policy for Rare Diseases, 2021 announced on Wednesday. Though the document specifies increasing the government support for treating patients with a ‘rare disease’— from ₹15 lakh to ₹20 lakh — caregivers say this doesn’t reflect actual costs of treatment.
There are 7,000-8,000 classified rare diseases, but less than 5% have therapies available to treat them. About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease-specific treatment. These diseases have differing definitions in various countries and range from those that are prevalent in 1 in 10,000 of the population to 6 per 10,000. India has said it lacks epidemiological data on the prevalence here and hence has only classified certain diseases as ‘rare.’
Where drugs are available, they are prohibitively expensive, placing immense strain on resources.
Currently few pharmaceutical companies are manufacturing drugs for rare diseases globally and there are no domestic manufacturers in India except for those who make medical-grade food for those with metabolic disorders. Due to the high cost of most therapies, the government has not been able to provide these for free. It is estimated that for a child weighing 10 kg, the annual cost of treatment for some rare diseases, may vary from ₹10 lakh to more than ₹1 crore per year with treatment being lifelong and drug dose and cost increasing with age and weight.
The policy was first prepared by the Centre in 2017 but put on hold. There were “implementation” challenges pointed out by States with the key question again remaining about costs: How would States and Centre share the costs of treatment?; What diseases would be covered? and who would benefit? An expert group was constituted in 2018 to review these questions. The committee submitted its report this January and after a further round of consultation the policy was made public this week.
Three groups
Saliently, a ‘rare disease’ has been categorised into three groups. Diseases in the first group would be eligible for a one-time treatment cost of up to ₹20 lakh provided the beneficiaries conformed to definition of the Pradhan Mantri Jan Arogya Yojana and were treated in government tertiary care hospital. For Group 2 diseases, States could “consider” supporting patients of such rare diseases that could be managed with special diets or hormonal supplements.
The government would notify selected Centres of Excellence at premier government hospitals for comprehensive management of rare diseases. The Centres of Excellence would be provided a one-time grant to a maximum of ₹5 crore each for infrastructure development for screening, tests, treatment.
‘Several lives lost’
“The new policy offers no support to patients awaiting treatment since the earlier National Policy for Treatment of Rare Diseases 2017 was kept in abeyance. In the absence of any funding support, close to 130 patients are left with no option but to wait for the inevitable. Several patients — mostly children — have already lost their lives in the interim period. Unlike conditions under Group 1 and Group 2, patients with Group 3 disorders require sustainable treatment support,” said Manjit Singh, National President, Lysosomal Storage Disorders Support Society (LSDSS).
The Ministry of Health and Family Welfare cited the need to balance competing priorities of public health in “resource constrained” settings.
“Looking at the number of rare disease patients diagnosed and considered eligible for treatment by the respective State technical committees, the immediate requirement of funds to support the immediate treatment needs of the diagnosed patients shouldn’t have exceeded ₹80 crore to ₹100 crore annually. If one is to look at it holistically, the Centre’s contribution would work out to ₹40 to ₹50 crore — if it is able to convince the State(s) for a load-sharing model, as a few States like Kerala, Tamil Nadu and Karnataka have already indicated,” said health economist Prof. Viswanath Pingali.
‘No consideration’
“It is alarming that the Union government has left patients with Group 3 rare diseases to fend for themselves in the National Policy for Rare Diseases 2021. The new policy has absolutely no consideration for Group 3 patients, who require lifelong treatment support. In the absence of a sustainable funding support for Group 3 patients, the precious lives of all patients, mostly children, are now at risk and at the mercy of crowdfunding. The Union government has failed these children who were hoping for help. Even Group 1 is only for few and Group 2 has been openly left for the State government,” said Prasanna Shirol, co-founder and executive director, Organisation for Rare Diseases India, an umbrella organisation.
His daughter Nidhi is afflicted with Pompe disease when she was 7 and now at 22 is in a “semi comatose state.” Mr. Shirol said one room in his house had been converted as an intensive care unit for two decades. “What the policy doesn’t capture is that these are diseases that last a lifetime. It also doesn’t realise how those who can’t afford such treatment will be unable to even make it to the prescribed tertiary hospitals for treatment. As a group, we shall soon be placing our objections to the new policy.”
Patients and their support groups have earlier written to the Health Ministry seeking an immediate seed-funding of ₹80 crore to ₹100 crore while rolling out the national policy so that the life-saving therapy of all those patients with treatable Group 3 disorders such as LSDs can be provided, thereby reducing further loss of life; design and execute a 100-day roll-out plan after the national policy is notified to prioritise treatment of all eligible rare disease patients and prioritise and encourage States with a matching grant, which have demonstrated a proof of concept in providing life-saving therapy to rare disease patients, such as Karnataka, Kerala and Tamil Nadu.
