At least 50 kids suffering from rare diseases and needing immediate treatment are among 237 patients awaiting treatment support via crowdfunding that has, apparently, taken a severe hit in Covid times.
Donations have almost dried up. According to Union Health Ministry data, it has managed to mobilise a meagre amount of just Rs 1.15 lakh, when these diseases require huge amounts of money for treatment. As a result, not a single patient has been treated so far, said Saurabh Singh, co-founder-director of Rare Disease India Foundation (RDIF) on Monday i.e. February 28 which is marked as Rare Disease Day.
He pointed out that the Ministry currently provides under the scheme a one-time grant of Rs. 20 lakh to Group 1 (Disorders amenable to one-time curative treatment) patients. However, in the past several times the ministry had extended support to 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) which are based on the literature sufficient evidence for good long-term outcomes exists.
Urging the Government to address the gaps in providing life-saving treatment to those requiring it, he said that the umbrella scheme of Rashtriya Arogya Nidhi (RAN) should be made available to all Group 3(a) patients with treatable conditions.
Singh suggested that the annual budgetary allocation of Rs. 25 crore under the RAN for 2021-22 that has not been spent could be immediately used to treat patients with diseases under Group (3) such as Gaucher, Pompe, Fabry disease and MPS I and MPS II.
The Ministry should also provide a budgetary allocation of at least Rs.150 crore from the ministry to provide life-saving therapy to patients with Lysosomal Storage Disorders, for which the Drug Controller General of India approved treatment is available for many years now. Singh suggested following the PM Cares model by encouraging and building PSU support.
Nearly 45 patients had died while awaiting treatment since setting up of the portal by the Ministry a few months ago, he said, adding that several developing economies such as Argentina, Brazil, Algeria and Egypt had robust reimbursement mechanisms or had allocated funds from the healthcare budget for the purpose. Jaipur-based Vikas Bhatia , head of MERD India, said, “The government should have facilities of OPD and clinics in each and every district for treatment of rare diseases.”
The commonly reported diseases include Primary immunodeficiency disorders, Lysosomal storage disorders (Gaucher’s disease, Mucopolysaccharidoses, Pompe disease, fabry disease etc.) small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias etc.), Cystic Fibrosis, osteogenesis imperfecta, certain forms of muscular dystrophies and spinal muscular atrophy, etc.