Lives hang in the balance: Government funding delays put kids at risk!

It has been eighteen months since KEM was selected as a centre of excellence one of the eleven across India for treating rare diseases in four states Maharashtra Madhya Pradesh Chhattisgarh and Gujarat However the funding of 5 crore for infrastructure and 50 lakh under the national policy for rare diseases is still pending and remains only on paperThe delay in implementing this policy has left parents of nearly 999 children in Group 3 of the rare disease category registered on the governments rare disease crowdfunding portal worried about the uncertainty surrounding their childs wellbeing and the unaffordable expensive treatmentsKEM hospital cost estimation letterThe central government increased the grant for treating rare diseases Group 1 2 and 3 from 20 lakh to 50 lakh as stated in the office memorandum dated May 19 2022 issued by the Union Ministry of Health amp Family Welfare Rare Disease CellOver 7000 rare diseaseThere are approximately 7000 known rare diseases worldwide affecting around 837 of the global population with 7537 of these patients being children In recent decades new therapies for rare disorders have emerged offering hope for survival through innovative techniques and advanced scientific technology However these therapies are often prohibitively expensive and require lifelong treatment placing a tremendous burden on affected families and physicians stated a statement on the government digital portal for crowdfunding and voluntary donations for rare disease patientsNo option butThe Government of India has established a crowdfunding portal as per the National Rare Disease Policy 2021 Still it has raised a meagre amount with only 286 lakh collected for the 999 patients registered in the Group 3b categoryPIL and court interventionWe are continuously engaging with the government of India requesting their support through PIL and Court Intervention In a recent court hearing on November 2 2023 the Honourable Justice asked government representatives to submit a report about their discussions with pharmaceutical companies which they committed to do by December 7 said Alpana Founder Trustee amp Director Patient Advocacy CureSMA Foundation of IndiaAlpana also noted Crowdfunding cannot be a sustainable mechanism for lifesaving or lifealtering medicines Lasting institutional and policy changes through government intervention are necessary to help SMA patients and their familiesDrugs not affordableThere are three FDAapproved treatments for Spinal Muscular Atrophy SMA ndash Risdiplam Zolgensma and Spinraza Out of these only Risdiplam has been launched in India but it remains inaccessible to patients due to its high cost These drugs are available to a select few SMA patients worldwide but not to the majority of Indian patients due to the lack of an insurance or reimbursement model for these lifesaving medicines added AlpanaWhere you live should not determine if you live Just because our children are born in lowmiddleincome countries they should not be denied the right to live The lack of access to lifesaving medicines is resulting in the loss of precious lives emphasised AlpanaCanrsquot afford R75 lakh per annum drugsBaby Aakriti who celebrated her first birthday on November 3 was diagnosed with SMA Type 1 at four months old and is undergoing treatment at KEM Hospital Her treatment includes six doses of gene therapy under a sampling program free of cost However they are still waiting for the promised government support of 50 lakh under the National Rare Diseases Policy 2021 to cover the annual cost of 75 lakh which is unaffordable for a middleclass familyAakritirsquos life at riskCA Kirti Singh Aakritis mother expressed her concerns We are unsure of what will happen to Aakriti after these six doses are over Considering the cost and uncertainty of the treatment her life is at risk We have requested support from the state and central governments but havent received any response We have also resorted to crowdfunding but its far from covering the hospitals cost estimateWhat KEM saidKEM hospital issued a Cost Certificate letter dated August 7 2023 which outlines the details of Aakritis treatment and the estimated costs associated with Risdiplam therapy Aakriti Singh diagnosed with Spinal Muscular Atrophy Type1 is under treatment at KEM Hospital She needs lifelong lifesaving treatment with Risdiplam which costs approximately 620 lakh for a 60 mg bottle with an annual expense of around 75 lakh This cost is expected to rise as Aakriti grows This certificate aims to help Aakritis family seek financial assistance for Risdiplam therapy