Are Medical Parliamentarians Raising Alarm on Rare Disease Treatments with PM?
Click to start listening
Synopsis
On December 11, the Indian Medical Parliamentarians’ Forum raised a critical alarm regarding the delays in Enzyme Replacement Therapy for rare diseases, calling for urgent intervention from Prime Minister Modi to prevent fatalities among affected children and adults.
Key Takeaways
- Significant delays in accessing ERT for rare diseases pose life-threatening risks.
- IMPF has raised urgent alarms requiring intervention from government authorities.
- Over 60 patients are currently without a treatment path due to financial caps.
- Many affected individuals may not be officially registered, complicating care efforts.
- Immediate action is required to prevent further fatalities.
New Delhi, Dec 11 (NationPress) The Indian Medical Parliamentarians’ Forum (IMPF), composed of 45 parliamentarians with medical backgrounds, voiced serious concerns on Thursday about the significant delays in accessing Enzyme Replacement Therapy (ERT) for both children and adults afflicted by rare and life-threatening diseases, which escalates the risk of mortality.
In a formal communication directed to Prime Minister Narendra Modi and also forwarded to Union Health Minister J P Nadda, the medical professionals expressed urgent concerns over the interruptions in treatment for individuals suffering from Lysosomal Storage Disorders (LSDs).
Lysosomal storage disorders comprise over 40 inherited metabolic disorders distinguished by the abnormal accumulation of various toxic substances in the body’s cells due to enzyme deficiencies. The prevalence of LSDs is estimated to be around 1 in every 7,000-8,000 births and includes diseases like Gaucher disease, Pompe disease, and Fabry disease.
Currently, there is no patient registry for these disorders in India, yet it is estimated that approximately 1 to 1 million individuals might be affected.
The IMPF cautioned that interruptions in treatment, as dictated by the National Policy for Rare Diseases (NPRD) 2021, are posing an immediate danger to numerous children diagnosed with LSDs. Dr. Anil Bonde, the Chairperson of the IMPF and a Rajya Sabha MP, urged for swift and decisive action to avert unnecessary fatalities.
The IMPF, which acts as a knowledgeable action group to influence health policy and highlight public health issues within Parliament, reported that around 60 patients have exceeded the Rs 50 lakh financial cap, leaving them without a feasible option for continued treatment.
They further indicated that delays in funding are placing nearly 100 patients currently receiving ERT at an imminent risk of treatment discontinuation, while over 60 children and young adults have tragically lost their lives due to delays in initiating therapy or interruptions in treatment.
The forum highlighted that even a temporary disruption in ERT can lead to acute metabolic crises, irreversible organ damage, and often, death, rendering every delay a life-threatening situation.
The physicians called for an urgent waiver or extension of the Rs 50 lakh cap for LSD patients to ensure no individual undergoing therapy is compelled to regress or face fatality due to financial constraints. They also advocated for the establishment of a continuum-of-care funding framework under the NPRD to guarantee long-term, uninterrupted treatment.
Point of View
It is crucial that the government acts swiftly to ensure access to necessary therapies. The IMPF’s concerns highlight a significant gap in the healthcare system that needs to be addressed.
NationPress
14/01/2026
Frequently Asked Questions
What is Enzyme Replacement Therapy?
Enzyme Replacement Therapy (ERT) is a medical treatment that replaces an enzyme that is deficient or absent in the body, helping to manage symptoms of certain rare diseases.
What are Lysosomal Storage Disorders?
Lysosomal Storage Disorders (LSDs) are a group of over 40 inherited metabolic disorders caused by enzyme deficiencies, leading to harmful accumulations of substances in the body.
How many people in India are affected by LSDs?
It is estimated that between 1 to 1 million people in India may be affected by Lysosomal Storage Disorders, although there is currently no official registry.
What is the Rs 50 lakh cap?
The Rs 50 lakh cap refers to the financial limit set under the National Policy for Rare Diseases, beyond which patients may struggle to obtain necessary treatments.
What actions are being called for to address this crisis?
The Indian Medical Parliamentarians' Forum is urging for a waiver or extension of the Rs 50 lakh cap and the establishment of a funding framework to ensure continuous access to treatment.
