Families of children suffering from rare diseases and patient advocacy groups (PAGs) have urged Prime Minister Narendra Modi and Union Health Minister J.P. Nadda to address the limitations of the current funding policy for rare disease treatments in India. Specifically, they are requesting the removal of the ₹50 lakh cap on one-time treatment support under the National Policy for Rare Diseases (NPRD), 2021.
The policy currently provides up to ₹50 lakh in funding for patients diagnosed with chronic, ultra-rare conditions, such as Lysosomal Storage Disorders (LSDs). However, many families report exhausting this one-time financial support without any provision for continued treatment.
Children Disproportionately Affected by Rare Diseases
Rare diseases, particularly chronic genetic disorders, are life-threatening and disproportionately affect children. Alarmingly, 30% of children diagnosed with these conditions do not survive beyond their fifth birthday if not diagnosed and treated promptly.
In Karnataka alone, approximately 20 patients from three Centres of Excellence (CoEs) have run into financial barriers after depleting the ₹50 lakh support. Many of these patients, suffering from disorders like Gaucher disease, are now facing treatment discontinuation.
Prasanna Shirol, co-founder of the Organisation for Rare Diseases India (ORDI), highlighted the financial burden on families, noting that there is no alternative support once the initial funding is exhausted. Shirol explained that several families are unable to continue life-saving treatments due to the lack of additional financial assistance.
One such case involves the mother of a nine-year-old child from Karnataka diagnosed with Gaucher disease. She described how her child’s treatment abruptly ceased last month once the ₹50 lakh fund was exhausted. “My child’s growth has drastically slowed down, and she has no control over her daily activities. Sometimes she overeats; other times, she doesn’t eat at all,” she said.
Similarly, the father of a four-year-old patient with Gaucher disease expressed concern that his daughter’s treatment was halted once the funding was used up.
Gaps in the Implementation of the NPRD
The implementation of the NPRD has faced several challenges, according to patient families and advocacy groups. In an open letter to the Prime Minister and the Union Health Minister, they pointed out critical gaps, including the abrupt discontinuation of treatments once the ₹50 lakh cap is exhausted, limited access to essential therapies such as Enzyme Replacement Therapies (ERT) for conditions like Pompe, MPS, and Fabry diseases, and delays in the rollout of the proposed ₹974 crore National Programme for Rare Diseases.
Despite a directive from the Delhi High Court on October 4, 2024, to release funds for patients who had exhausted the ₹50 lakh support and establish the National Fund for Rare Diseases (NFRD) with an allocation of ₹974 crore for FY 2024-2025 and 2025-2026, no concrete action has been taken by the Ministry of Health and Family Welfare, leaving families in a state of uncertainty and distress.
A Call for Transparent and Sustainable Funding
In response to these challenges, PAGs and patient families are urging the government to establish a transparent, sustainable funding framework to ensure that patients receive continuous treatment, especially those with conditions that have proven clinical outcomes. They are also calling for improvements in the procurement and disbursement of funds at the CoEs, as well as the inclusion of Acid Sphingomyelinase Deficiency (ASMD) as a notified condition under the NPRD to allow eligible patients access to life-saving therapies.
As rare disease patients and their families await action, the need for long-term, reliable funding remains a critical issue in ensuring that all children and adults affected by rare conditions have access to the treatments they need.
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