Latest news with #NationalPolicyforRareDiseases
Time of India
19-07-2025
- Health
- Time of India
Health minister seeks Rs 50L aid from CM for patient
Mangaluru: Minister for health and family welfare Dinesh Gundu Rao urged chief minister Siddaramaiah to release a special sanction of Rs 50 lakh to a patient from Sullia, Chandini GD., who is suffering from a rare disease. The minister stated in a request letter to the CM, that it would require more than Rs 50 lakh for the treatment of the 34-year-old Chandini, wife of Purushothaman C from Gandhinagar in Sullia. She is suffering from right atrial mass, hyper IGE syndrome with mast cell activation syndrome, dock 8 mutation seizure disorder, hypothyroidism, hiatus hernia with motility disorder. Since the treatment involves a huge cost, the minister requested the CM that it would be better if the finance department sanctioned the treatment cost. "Chandini hails from a poor family and she is suffering from a rare disease. There is no option to bear the cost of her treatment under health department schemes. A request to sanction treatment cost under Chief Minister's Relief Fund was made. Accordingly, Rs 1 lakh was already released. Since the treatment of Chandini would require more than Rs 50 lakh, I request you to consider this as a special case and sanction the funds through the finance department," Rao stated in his letter to the CM. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Is it legal? How to get Internet without paying a subscription? Techno Mag Learn More Undo MLC Ivan D'Souza brought to the notice of the govt during the legislature session about Chandini, in March this year. The MLC requested the govt to bear the entire cost of Chandini's treatment. The health minister suggested taking Chandini to visit Indira Gandhi Institute of Child Health (IGICH) for advanced treatment. The treatment method is available under the National Policy for Rare Diseases (NPRD), according to the minister. Chandini, a dance teacher, underwent several surgeries, hospitalisations, and intensive care treatments for about 30 years.
Time of India
01-06-2025
- Health
- Time of India
Rare disease patients continue to struggle despite govt policies
New Delhi: Despite the establishment of the National Policy for Rare Diseases (NPRD) in 2021 and a nationwide financial assistance programme, over 300 patients, predominantly children diagnosed with Lysosomal Storage Disorders (LSDs) such as Gaucher, Pompe, Fabry, MPS I, and II, are left without essential medical care. Tired of too many ads? go ad free now Among them, 70 are from Delhi. Since 2022, nearly 50 patients—including six from Delhi—have succumbed to these diseases. The Lysosomal Storage Disorders Support Society (LSDSS) reports that 50 patients across India exhausted their one-time funding under the policy. In Delhi, 7 patients are in critical condition due to financial constraints. Alishba Khan, a young girl battling Gaucher disease, illustrates the dire consequences of limited funding. Her father, Maqsood Alam, recounts the devastating decline in her health following the discontinuation of her medication in Aug 2024. "She suffers from severe abdominal pain, an enlarged spleen, bone pain, weakness, and decreased haemoglobin level. She is unable to attend school and requires constant supervision," Alam shared, his voice filled with anguish. Having already lost four children, he continually appeals to the High Court for continued treatment, fearing for Alishba's survival. While doctors at AIIMS offer significant support, they are constrained by limited funding, he said. Recognising the critical need, the Delhi High Court on Oct 4, 2024, directed the Ministry of Health and Family Welfare to extend funding beyond the Rs 50 lakh cap for patients with rare diseases. The court also mandated the establishment of a Rs 974 crore national fund for rare diseases for the fiscal years 2024-25 and 2025-26. Tired of too many ads? go ad free now However, the Ministry challenged this directive in the Supreme Court, where the matter remains pending. The Lysosomal Storage Disorders Support Society (LSDSS) has been vigorously campaigning for urgent action. In a recent appeal to Union Health Minister JP Nadda, the LSDSS highlighted that despite allocating over Rs 205 crore to 12 Centres of Excellence (CoEs)—including AIIMS Delhi and MAMC—more than 300 eligible patients remain untreated, and at least 50 patients have died while awaiting treatment. LSDSS National President Manjit Singh emphasised that many of these patients are children with conditions for which DCGI-approved therapies exist. " In the Budget Session of Parliament, the Ministry reaffirmed that no eligible patient should be denied life-saving therapy. However, the disconnect between funding and actual treatment on the ground continues to undermine this commitment," the letter stated. The society requested the ministry to direct CoEs to immediately accept eligible patients, ensure compliance with NPRD 2021 guidelines, prioritise patients with approved therapies, establish a rapid response task force for timely treatment initiation, and authorise continued treatment support beyond the Rs 50 lakh cap based on clinical merit. Singh acknowledged the govt's historic support for rare disease patients, requesting leadership to ensure this support translates into timely, life-saving access. Officials at MAMC explained that the crowdfunding portal has technical constraints preventing status updates from 'waiting for treatment' to 'under treatment' after therapy begins. Although the portal displays zero patients receiving treatment, in reality, 10 individuals out of 28 registered cases of LSDs are currently undergoing enzyme replacement therapy (ERT). The administration confirmed that treatment allocation strictly follows approved eligibility standards, considering ERT's expected clinical effectiveness and requires approval from the Centre of Excellence (CoE) committee, comprising various medical specialists. They noted that patients with significant neurological symptoms receive lower priority, as current evidence suggests ERT offers minimal benefits in such instances. The focus remains on treating patients with a higher likelihood of substantial clinical benefits. Regarding the national policy's one-time financial support limit of Rs 50 lakh per patient, the MAMC officials indicated their inability to provide treatment beyond this amount without specific instructions from senior authorities. When contacted, Dr Neerja Gupta from the genetic unit of paediatrics at AIIMS said she doesn't have the details on this matter offhand.
Time of India
15-05-2025
- Health
- Time of India
Bone marrow transplant facility for MPS at city hosp
1 2 3 Kolkata: The Centre of Excellence (CoE) for rare diseases in eastern India, situated at IPGMER, has begun the procedure to set up a bone marrow transplant (BMT) facility for children suffering from MPS (Mucopolysaccharidoses), a group of rare and inherited metabolic disorders. IPGMER is among the 12 health institutes across the country designated as CoE by the health ministry for the treatment of rare diseases. "Another good treatment option for children suffering from two categories of MPS - 1 and 2 is BMT when they are below two years of age. We are moving forward with the procedure to have such a BMT unit for children suffering from this rare disease," said neonatology head Suchandra Mukherjee, the nodal official at the IPGMER CoE, on World MPS Day on Thursday. At present, only two CoE for rare diseases at AIIMS New Delhi and PGI Chandigarh have BMT facilities for this rare disease. While there is no cure for MPS, treatments such as enzyme replacement therapy (ERT) and stem cell transplantation are available to slow down the disease progression. On average, a child is given ERT twice a month. The treatment is costly and beyond the reach of most the National Policy for Rare Diseases, a patient with any recognised rare disease, including MPS, can get one-time financial aid of Rs 50 lakhs for treatment. However, many times, the funds get exhausted, leaving these patients in the lurch. In fact, of the 25 children with MPS, including a few from the neighbouring states, are registered with the SSKM CoE for treatment. The funds for four children have already been exhausted, forcing them to take up supportive treatment. "Early diagnosis is key to a better treatment outcome. We are holding regular training and sensitisation programmes for paediatricians for awareness. At IPGMER, we are also starting pre-natal screening. This is an inherited disease," added Mukherjee.
