Parents’ worries mount as govt support remains elusive for costly treatments
Ashish and CA Kirti parents of baby Akirti a SMA type 1 patient
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 paper.
The delay in implementing this policy has left parents of nearly 999 children in Group 3 of the rare disease category, registered on the government's rare disease crowdfunding portal, worried about the uncertainty surrounding their child's well-being and the unaffordable, expensive treatments.
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KEM hospital cost estimation letter
"The 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 & Family Welfare Rare Disease Cell.
Over 7,000 rare disease
"There are approximately 7,000 known rare diseases worldwide, affecting around 8% of the global population, with 75% 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 patients.
No option but
The 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 '2.86 lakh collected for the 999 patients registered in the Group 3b category.
PIL and court intervention
"We 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 & Director Patient Advocacy, CureSMA Foundation of India.
Alpana also noted, "Crowdfunding cannot be a sustainable mechanism for life-saving or life-altering medicines. Lasting institutional and policy changes through government intervention are necessary to help SMA patients and their families."
Drugs not affordable
"There are three FDA-approved treatments for Spinal Muscular Atrophy (SMA) – 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 life-saving medicines," added Alpana.
"Where you live should not determine if you live. Just because our children are born in low/middle-income countries, they should not be denied the right to live. The lack of access to life-saving medicines is resulting in the loss of precious lives," emphasised Alpana.
Can’t afford R75 lakh per annum drugs
Baby 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 middle-class family.
Aakriti’s life at risk
CA Kirti Singh, Aakriti's 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 haven't received any response. We have also resorted to crowdfunding, but it's far from covering the hospital's cost estimate."
What KEM said
KEM hospital issued a 'Cost Certificate' letter dated August 7, 2023, which outlines the details of Aakriti's treatment and the estimated costs associated with Risdiplam therapy. Aakriti Singh, diagnosed with Spinal Muscular Atrophy Type-1, is under treatment at KEM Hospital. She needs lifelong, life-saving treatment with Risdiplam, which costs approximately '6.20 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 Aakriti's family seek financial assistance for Risdiplam therapy.
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