Rare disease treatment a pain after Rs 50 lakh aid exhausts

India’s rare disease policy promises treatment worth Rs 50 lakh per patient battling with 62 kinds of diseases listed under the National Rare Disease Policy (NRDP) 2021. 

But 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. 

The policy has divided the diseases into three groups: Group 1 which requires procedures such as Bone Marrow Transplant (BMT) or organ transplantation.

Group 2 which requires long term treatment but at low cost and group 3, requiring lifelong treatment at high cost. 

Prasanna Shirol, co-founder and executive director of Organisation for Rare Disease of India (ORDI) told to DH, “The problem here is that 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 will lead to funds exhausting soon”. 

“In group 2, it is manageable. But 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,” he said. 

There are no updates if the support will be revived, said Shirol.

Another association requesting anonymity said, “Rare disease is a very unfortunate fate. In the 62 diseases listed, a few of the diseases have treatments, other diseases do not have treatment in India and the rest do not have any treatment at all. But for diseases with no treatment, the government should at least provide some support or interventions”. 

The patients diagnosed with rare diseases that DH spoke to 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. 

Dr Sanjeeva G N, professor of paediatrics and nodal officer of Centre Of Excellence for Rare Diseases, said, “As for the funding amount, there are no problems. The problem is with group 3 as there is nothing we can do after treatment worth 50 lakh is over. The disease relapses once the treatment is stopped”.