3-yr-old dies waiting for rare disease funds from Centre
Times of India | 4 April 2025
123 Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the Central govt's National Rare Disease Policy (NRDP). The policy provides one-time financial assistance of up to Rs 50 lakh per child with a rare disease for treatment at designated centres of excellence (CoE).
For Bengal, the CoE is located at the state-run IPGMER (SSKM). Sources said that there are over 250 children in Bengal whose names are registered in the designated portal to receive this facility.
Doctors said that the three-year-old Anushree Dhar from Barrackpore was diagnosed with Gaucher about a year ago. This condition is a rare genetic disorder that causes a buildup of fatty substances in the body. This buildup can lead to enlarged organs, bone problems, and other symptoms. The treatment for this condition is enzyme replacement therapy (ERT).
Sources said the girl's name was put up in the portal about eight months ago. But the girl died on Thursday before the treatment could start.
Neonatologist Suchandra Mukherjee, department head as well as the nodal officer of the CoE at IPGMER, said that all baseline investigations were done for this child for the ERT to begin, and even the medicines were ordered. "Among the rare diseases, Gaucher has a better outcome than other diseases. Currently, 21 children are being treated for this condition under the NRDP, apart from children with MPS, another rare disease. We were all set to begin the treatment of this child too. Unfortunately, the parents informed us that the child died," Mukherjee told TOI. The CoE is also planning to start the treatment of children with SMA (spinal muscular atrophy), another rare disease.
The NRDP categorises rare diseases into three groups - Group 1: disorders amenable to one-time curative treatment, Group 2: diseases requiring long-term/lifelong treatment, and Group 3: diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost, and lifelong therapy. At present, 63 rare diseases across the three groups are included in the NRDP.
Doctors working for rare diseases said long waiting times in registration and delays in starting treatment are putting the lives of many children with rare diseases on the edge. At present, the names of around 250 children from Bengal are on the portal for rare disease treatment, but only about 28 children are getting treatment under the policy at the CoE at IPGMER.
"We understand that funds are limited and there is a need to triage so that these children get medical assistance without losing any time. We are more than willing to help in this if the CoE asks us," said geneticist Dipanjana Datta, state coordinator for Organisation for Rare Disease India (ORDI).
Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the Central govt's National Rare Disease Policy (NRDP). The policy provides one-time financial assistance of up to Rs 50 lakh per child with a rare disease for treatment at designated centres of excellence (CoE).
For Bengal, the CoE is located at the state-run IPGMER (SSKM). Sources said that there are over 250 children in Bengal whose names are registered in the designated portal to receive this facility.
Doctors said that the three-year-old Anushree Dhar from Barrackpore was diagnosed with Gaucher about a year ago. This condition is a rare genetic disorder that causes a buildup of fatty substances in the body. This buildup can lead to enlarged organs, bone problems, and other symptoms. The treatment for this condition is enzyme replacement therapy (ERT).
Sources said the girl's name was put up in the portal about eight months ago. But the girl died on Thursday before the treatment could start.
Neonatologist Suchandra Mukherjee, department head as well as the nodal officer of the CoE at IPGMER, said that all baseline investigations were done for this child for the ERT to begin, and even the medicines were ordered. "Among the rare diseases, Gaucher has a better outcome than other diseases. Currently, 21 children are being treated for this condition under the NRDP, apart from children with MPS, another rare disease. We were all set to begin the treatment of this child too. Unfortunately, the parents informed us that the child died," Mukherjee told TOI. The CoE is also planning to start the treatment of children with SMA (spinal muscular atrophy), another rare disease.
The NRDP categorises rare diseases into three groups - Group 1: disorders amenable to one-time curative treatment, Group 2: diseases requiring long-term/lifelong treatment, and Group 3: diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost, and lifelong therapy. At present, 63 rare diseases across the three groups are included in the NRDP.
Doctors working for rare diseases said long waiting times in registration and delays in starting treatment are putting the lives of many children with rare diseases on the edge. At present, the names of around 250 children from Bengal are on the portal for rare disease treatment, but only about 28 children are getting treatment under the policy at the CoE at IPGMER.
"We understand that funds are limited and there is a need to triage so that these children get medical assistance without losing any time. We are more than willing to help in this if the CoE asks us," said geneticist Dipanjana Datta, state coordinator for Organisation for Rare Disease India (ORDI).