Infusion therapy on Rare Disease patient begins in Kerala
Patients, doctors hope that the Kerala model of innovative financing could be replicated by other states
Two toddlers – aged 2 and 3 years respectively – and both diagnosed with Pompe disease, a form of Lysosomal Storage Disorders (LSDs), have been cleared to start their life-saving Enzyme Replacement Therapy (ERT) at the Government Medical College, Kozhikode. While the first patient from Kannur will receive her infusion therapy on Friday, the second patient, a resident of Mannarkkad, begins her infusion therapy in November.
The infusion therapy was started following an order by a division bench of the Kerala High Court – Chief Justice S Manikumar and Justice Shaji P. Chali — on August 14th this year. The court was hearing a writ petition filed by the Lysosomal Storage Disorders Support Society (LSDSS), a patient advocacy group, set up to fight the cause of rare disease patients across the country. Both these patients were supported by Sanofi Genzyme under its Charitable Access Program and provided the short-term bridging therapy considering their medical condition.
According to Manoj Manghat, Kerala state co-ordinator of LSDSS, “This is a unique financing model where not just the Union Government, but the state of Kerala and the Kerala High Court Advocates’ Association too contributed towards a corpus fund, set up to start treatment for these two children (girls). This innovative financing model is a step in the right direction, and could be explored by other states to provide sustainable funding support to LSD patients, awaiting treatment without any institutional support so far.”
While the Centre allotted an amount of INR 1.5 crore, the state sanctioned another INR 50 lakh for the treatment of these two rare disease patients. Interestingly, members of the Kerala High Court Advocates’ Association, following an appeal by the High Court, also mobilised an amount of INR 5 lakh for the treatment of the rare disease patients.
Health experts and patients are now hopeful that the Kerala government, under its ambitious Kerala Social Security Mission (KSSM), will create a dedicated fund pool to support the treatment of all LSD patients in the state.
“The delay in the finalization of the National Policy on Rare Diseases 2020 and the absence of sustainable funding mechanism for those diagnosed with Group 3 disorders like LSDs has caused immense distress to the patients and many lives are already lost. Majority of patients are unable to mobilise funds to support their treatment, and any further delay in the formulation of the National Policy with sustained funding mechanism will only shorten their survival prospects,” explained Manoj, appealing to the Prime Minister Narendra Modi to support the cause of the patients.
According to Dr. Mohandas Nair, Head of Department, Pediatrics, Kozikhode Medical College, “Rare Diseases like LSDs are complex and heterogeneous, and requires a sustainable support mechanism for diagnosis and treatment. The genetic and Rare Diseases are serious chronic, debilitating and fatal disorders, often requiring long term, specialized treatments/management and sometimes causing severe disabilities.”
According to Dr. Muhammed Asheel, Executive Director, Kerala Social Security Mission (KSSM), “This is a commendable effort by the Kerala High Court to raise money for Rare Disease patients who have been ignored for a long time. However, we need a solid premise for sustainable treatment funding in the National Policy for Rare Diseases 2020 and attention also needs to be paid to other specifics such as cost of medicines and special exemptions in the policy.”
The High Court, while passing the order, also issued directions to the Kerala State Legal Services Authority (KeLSA) to explore possibility of raising funds for providing treatment to the children who suffer from rare diseases. Assistance of Childline and such other NGOs can also be sought for by the member secretary, KeLSA for the purpose of raising adequate corpus, the court further ruled.