Rare disease patients seek PM’s intervention to secure additional funds for treatment

New Delhi: In a letter to Prime Minister Narendra Modi, the Lysosomal Storage Disorders Support Society (LSDSS) sought for an interim funding mechanism to restore treatment for rare disease patients who have exhausted the Rs 50 lakh financial assistance available under the National Policy for Rare Diseases (NPRD), 2021.

Patients with progressive, potentially life-threatening conditions were facing treatment interruptions after exhausting their sanctioned financial assistance, LSDSS said in the letter addressing Modi, and Union Health Minister, JP Nadda.

It said 164 patients had exhausted the financial assistance available under NPRD, 2021, of whom around 115 were patients suffering from ultra-rare LSDs, including Gaucher and Pompe disease.

Many of these patients are children as young as two, three, four or five years of age, it added.

LSDSS appealed to urgently reinstate treatment for all clinically eligible patients and to establish an interim funding arrangement until the matter is sub judice before the Supreme Court.

It recommended that the Ministry provide funding on a case-to-case basis through the existing NPRD mechanisms.

It added that a continuity-of-treatment mechanism must be established under NPRD to fill a structural gap in the implementation of the policy.

LSDSS said, “A mechanism is urgently required so that a patient requiring lifelong or long-term therapy is not abruptly rendered ineligible merely because the prescribed financial ceiling has been reached.”It also urged that children and patients at high clinical risk must be prioritised for treatment and that all concerned Centres of Excellence must be directed to identify and report such patients immediately.