India's Rare Disease Patients Face Treatment Halt as Rs 50 Lakh Aid Cap Exhausted
Over 100 children with rare diseases in India have exhausted the Rs 50 lakh financial assistance limit under the National Policy for Rare Diseases 2021, forcing treatment halts and prompting urgent appeals for continued support.
More than 100 children with rare diseases in India have exhausted the Rs 50 lakh financial assistance limit provided under the National Policy for Rare Diseases (NPRD 2021), forcing a complete halt in life-saving care and pushing them back to ground zero. Eight patients have already died while waiting for treatment support, according to the Lysosomal Storage Disorders Support Society (LSDSS).
Nearly 2,000 rare disease patients across India are currently awaiting treatment, including around 450 eligible patients suffering from life-threatening Lysosomal Storage Disorders who require timely therapy. The LSDSS president has appealed to the Prime Minister to immediately allocate the remaining Rs 271 crore out of Rs 299 crore to all Centres of Excellence and direct them to initiate treatment for eligible patients while ensuring continuity of care for children whose funds have been exhausted.
The NPRD launched by the Ministry of Health and Family Welfare in March 2021 categorised rare diseases into three groups: Group 1, disorders amenable to one-time curative treatment; Group 2, diseases requiring long-term or lifelong treatment with relatively lower costs; and Group 3, diseases where definitive treatment is available but involves challenges such as optimal patient selection, very high costs and lifelong therapy. A total of 63 rare diseases including lysosomal storage disorders such as Fabry, Pompe and MPS 2 are being severely impacted.
For Group 1 patients, many government hospitals do not have facilities and they are asked to go to private hospitals. The treatment is expensive in private hospitals, which leads to funds exhausting soon. Group 3 faces bigger problems as it includes Lysosomal Storage Disorder (LSD), which requires long-term treatment. In 2016, Karnataka became the first state to announce financial support for people with LSD, but in 2024 the support was stopped. There are no updates if the support will be revived.
Lysosomal Storage Disorders are ultra-rare genetic metabolic disorders marked by an abnormal build-up of toxic substances in the body's cells due to enzyme deficiencies. These include Gaucher disease, Pompe disease, Fabry disease and others. Set up in 2010, the LSDSS is the first patient organisation in India to bring Lysosomal Storage Disorders and rare diseases into national policy discussions.
A nodal officer at a Centre of Excellence for Rare Diseases stated that as for the funding amount, there are no problems. The problem is with Group 3 as there is nothing that can be done after treatment worth Rs 50 lakh is over. The disease relapses once the treatment is stopped.
The chairperson of the Indian Medical Parliamentarians' Forum had written to the Prime Minister citing data from the National Rare Disease Crowdfunding Portal and patient networks of around 600 registered Group 3(a) patients in India. Twelve Centres of Excellence were identified for the diagnosis, prevention and treatment of rare diseases under the NPRD.
Patients diagnosed with rare diseases said after they were diagnosed, they had to let go of their previous lifestyle, forcing many into unemployment and debt. They said they were happy for the support, but that it was not enough. For families battling life-long genetic disorders, the cap often runs out before the treatment does, leaving them halfway to either crowdsource or abandon therapy.