Rare disease patients and advocacy groups appeal to PM Modi for sustainable funding for treatment

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Several families of children suffering from rare diseases and patient advocacy groups (PAGs) have appealed to Prime Minister Narendra Modi and Union Health Minister J.P. Nadda to remove the cap of providing one-time treatment support of up to ₹50 lakh for patients.

Under the National Policy for Rare Diseases (NPRD), 2021, patients diagnosed with chronic, ultra-rare disorders such as Lysosomal Storage Disorders (LSDs) are given funding up to  ₹50 lakh for treatment.

Also read: India’s fight against rare diseases

Rare diseases, particularly chronic genetic disorders, impose life-threatening challenges, often affecting children disproportionately. Alarmingly, 30% of children diagnosed with rare diseases do not survive beyond their 5th birthday, if the condition is not diagnosed and treated. 

Approximately 38 patients from three Centres of Excellence (CoEs) including 20 from Karnataka have been running from pillar to post after exhausting their one-time financial support of ₹50 lakh. 

Prasanna Shirol, co-founder of the Organisation for Rare Diseases India (ORDI), said many families are facing catastrophic financial burdens, with no alternative funding or support to continue life-saving treatment.

The mother of a nine-year-old patient from Karnataka suffering from Gaucher disease, a  rare genetic disorder that causes a buildup of fatty substances in the body, said her child’s treatment had stopped abruptly last month after the ₹50 lakh fund was exhausted. “Due to this, my child’s growth has drastically slowed down as she has no control over her day-to-day activities. Sometimes she overeats, sometimes she does not eat at all,” she said.