Indian Medical Parliamentarians’ Forum flags acute crisis in care for children with rare lysosomal disorders

The Indian Medical Parliamentarians’ Forum (IMPF), a cross-party body comprising 45 doctors-turned-lawmakers from both Houses of Parliament, has raised an urgent alarm over what it terms an emerging humanitarian crisis affecting children with ultra-rare Lysosomal Storage Disorders (LSDs). In a detailed submission to the Prime Minister, the forum has warned that treatment interruptions under the National Policy for Rare Diseases (NPRD 2021) have placed dozens of young patients at immediate and preventable risk.

The representation, led by IMPF chairperson Anil Bonde, MP (Rajya Sabha), has sought time-bound intervention to ensure uninterrupted access to Enzyme Replacement Therapy (ERT) — the only life-saving treatment available for a number of Group 3(a) LSDs. A parallel appeal has also been addressed to Union Health Minister J.P. Nadda.

According to the forum, critical indicators point to a rapidly worsening situation. Nearly 60 patients have already exceeded the annual funding ceiling of ₹50 lakh, leaving their treatment unsupported. Close to 100 patients on regular ERT now stand on the brink of discontinuation as funds remain stalled. Significantly, more than 60 children and young adults have already succumbed to delays in therapy initiation or breaks in treatment. Even brief interruptions, the forum notes, can trigger irreversible organ damage or fatal metabolic crises.

Seeking immediate policy correction, the IMPF has urged the Union government to remove or substantially enhance the ₹50 lakh cap for eligible LSD patients, arguing that the current limit does not reflect the lifelong nature of treatment needs. It has recommended a predictable, ring-fenced continuum-of-care financing mechanism and expedited fund utilisation across Centres of Excellence to eliminate administrative delays that jeopardise survival.

The forum has emphasised that timely intervention is essential to prevent avoidable deaths and uphold the commitments of NPRD 2021.