Delay in roll out of ₹974 crore under rare diseases policy hits patients

Delay in the roll out of the ₹974-crore National Policy for Rare Diseases (NPRD) is having an adverse impact on patients, including children, with patients’ advocacy groups writing to the Health Ministry seeking urgent intervention and immediate access to life-saving drugs. 
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Delay in the roll out of the ₹974-crore National Policy for Rare Diseases (NPRD) is having an adverse impact on patients, including children, with patients’ advocacy groups writing to the Health Ministry seeking urgent intervention and immediate access to life-saving drugs. 

“The funding under NPRD-2021 has run out, and there is no sustainable plan in place yet. Families are helpless, watching their loved ones deteriorate every day. This delay has left patients stranded across Centres of Excellence [CoEs] due to exhaustion of funds. Despite the policy’s intent to support rare disease patients, delays and funding restrictions are leaving hundreds — especially children — without access to life-saving treatment,’’ said the letter addressed to the Health Ministry.

Rare diseases, particularly chronic genetic disorders, impose life-threatening challenges, often affecting children disproportionately. Currently 30% of children diagnosed with rare diseases do not survive beyond their fifth birthday, if the condition is not diagnosed and the patient put on treatment.

Restricted access

These challenges include discontinuation of treatment after the one-time funding support up to ₹50 lakh is exhausted and restricted access to life-saving therapies such as Enzyme Replacement Therapies for notified conditions like Pompe, MPS and Fabry. The letter added that approximately 38 patients from three CoEs have been running from pillar to post after exhausting their one-time financial support of ₹50 lakh. 

The advocacy groups say that many families are facing catastrophic financial burdens, with no alternative funding or support to continue life-saving treatments. This delay has not only caused unnecessary suffering but also undermined the trust of the rare disease community in the government’s commitment to their welfare. 

The group has recommended a sustainable funding mechanism, establishing a transparent framework to ensure uninterrupted treatment for all eligible patients, ensure timely access to treatment and resolve delays in procurement and disbursement of funds at the CoEs.

The group added that the Delhi High Court, in its October 4, 2024 order, directed the Ministry of Health and Family Welfare to immediately release funds for the treatment of rare disease patients who had exhausted the ₹50-lakh cap. Additionally, the court mandated the establishment of the National Fund for Rare Diseases (NFRD) with an allocation of ₹974 crore for FY 2024-25 and 2025-26. However, despite these judicial directives, there has been no action taken by the Ministry, leaving families in complete despair.