A budget that remains unspent, patients remain untreated, crisis brews, this Rare Diseases Day

Nayan was diagnosed with MPS 2, a rare genetic disorder, in 2016. His growth became stunted, and despite his best efforts to stay active and exercise, the disease does not let him live a normal life. “We had requested ₹1.25 crore from the government but got only ₹50 lakh from the Prime Minister’s Office. That is less than half the amount we need to help Nayan live a normal life,” says Jagdish Papnai, his father.

Alishba Khan, also from Delhi, is in a similar circumstance. Her father, Maqsood Alam, says the 7-year-old with Gaucher disease had begun to stabilise with the treatment, thanks to the financial aid of ₹50 lakh given through the National Policy for Rare Diseases (NPRD). When the funding ceiling was crossed in August 2024, Alishba’s treatment stopped; since then, her condition has deteriorated, and even breathing has become difficult, he said.

On Rare Diseases Day this February 28, there is a strange sort of crisis in India. A notified policy for rare diseases is in place, there are Centres of Excellence (CoEs) established across the country, and a robust budget to assist with treatment. However, about ₹271 crore allocated for rare disease patients remains unused, and many of the children are quietly but steadily losing their quality of life and hope.

A few children have already died waiting for a resolution. “With the Supreme Court of India hearing scheduled for March 2026, patients are caught in a limbo. We are witnessing the devastating reality of treatment interruptions. Every delay in restarting and sustaining therapy has life-altering consequences. We need immediate intervention to ensure continuity of care and prevent further loss of young lives,“ says Saurabh Singh, founder of the Rare Disease India Foundation.

The rare diseases community, which has gained a voice over the years, points out that the current situation is completely untenable, particularly because the money actually exists. For the year 2025–26, ₹299 crore was allocated for rare disease treatment, but as per a reply to an RTI application by Manjit Singh, president of the Lysosomal Storage Disorders Society, only ₹30.79 crore has been utilised by the NPRD so far.  

“Nearly 2,000 rare disease patients across India are currently awaiting treatment, including around 450 eligible patients with life-threatening Lysosomal Storage Disorders (LSD). Alarmingly, approximately 100 children who had begun treatment have already exhausted the ₹50-lakh funding cap and now face a complete halt in life-saving care, pushing them back to square one. Eight patients have already died while waiting for treatment support,” Mr. Singh explains.

“We urgently request the Ministry to immediately allocate the remaining ₹271 crore (out of ₹299 crore) to all Centres of Excellence and direct them to initiate treatment for eligible patients, while also ensuring continuity of treatment for the children whose funds have been exhausted.” Notably, the remaining ₹271 crore will lapse at the end of the financial year on March 31, 2026. 

Members of Parliament across party lines have written to the Health Minister urging his intervention to continue with the funding to ensure uninterrupted therapy. Allowing allocated public funds to lapse while eligible patients remain untreated raises concerns under Article 21 (Right to Life), and indicates a serious disconnect between intent and implementation, the rare diseases lobby points out.