Patients with rare diseases and defense groups appeal to the PM Modi to obtain sustainable funds for treatment

A vision of the Indira Gandhi Children’s Institute in Benagluru, where facilities for rare diseases are offered. | Photo credit: the Hindu

Several families of children suffering from rare diseases and patient defense groups (PAG) have appealed to Prime Minister Narendra Modi and the Minister of Health of the Union, JP Nadda, to eliminate the limit of providing unique treatment of treatment of up to ₹ 50 Lakh for patients.

According to the National Rare Diseases Policy (NPRD), 2021, patients diagnosed with ultra -rich chronic disorders, such as lysosomal storage disorders (LSD), receive funds from up to ₹ 50 LAKH for treatment.

Read too: India’s struggle against rare diseases

Rare diseases, particularly chronic genetic disorders, impose potentially mortal challenges, which often affect children disproportionately. To the alarming, 30% of children diagnosed with rare diseases do not survive beyond their fifth birthday, if the condition is not diagnosed and treated.

Karnataka 20

Approximately 38 patients from three centers of excellence (COES), including 20 Karnataka, have been fleeing from one pillar to another after exhausting their unique financial support from ₹ 50 Lakh.

Prasanna Shirol, co -founder of the Organization for Rare Diseases of India (Ordi), said that many families face catastrophic financial loads, without alternative funds or support to continue with the treatment of life savings.

The mother of a nine -year -old patient of Karnatak ₹ 50 Lakh background sold out. “Because of this, my son’s growth has slowed down drastically, since he has no control over his daily activities. Sometimes he eats in excess, sometimes he doesn’t eat at all, ”he said.

Karnataka’s four -year -old Gaucher disease patient also had a similar concern. His daughter’s treatment has also stopped abruptly after the bottom was exhausted.

Gaps in the implementation

Highlighting the gaps in the implementation of the NPRD, 2021, who have left many patients without adequate support, families and patients have written open letters to the prime minister and the Minister of Trade Union Health.

“These challenges include the interruption of the treatment after the single financing support up to ₹ 50 Lakh is exhausted, the restricted access to the therapies that save lives such as enzyme replacement therapies for conditions notified such as Pompe, MPS and Fabry, and delay In the deployment of the deployment of the deployment of the enzyme national program of ₹ 974 million rupees for the treatment of rare diseases, ”said the letter.

The Superior Court of Delhi, in its order of October 4, 2024, had ordered the Ministry of Health and Family Welfare of the Union to release funds immediately for the treatment of patients with rare diseases who had exhausted the limit of ₹ 50 Lakh . In addition, the Court ordered the establishment of the National Fund for Rare Diseases (NFRD) with an allocation of ₹ 974 million rupees for fiscal year 2024-2025 and 2025-2026. However, the Ministry has not taken measures, leaving families with complete despair, Shirol said.

Pags and patient families have recommended that the government establish a transparent and sustainable financing framework. This is to guarantee uninterrupted treatment for all eligible patients, particularly for those conditions, which have demonstrated clear clinical results and have been administered to patients in India for a long time.

Their other recommendations include the resolution of delays in the acquisition and disbursement of funds in the COES, the inclusion of the deficiency of the acidic sphingomyelinase (ASMD) as a condition notified under NPRD 2021 to ensure that eligible patients have access to the therapy of life savings.