MPs' forum seeks action on lack of funds for treating rare diseases

A bipartisan parliamentary forum has warned that funding shortages and administrative delays under India's rare disease policy are disrupting life-saving treatment

A bipartisan parliamentary group has sought immediate action against lack of funds causing treatment disruptions for children and adults facing Lysosomal Storage Disorders (LSD), a group of ultra rare diseases covered under the National Policy for Rare Disease (NPRD).

 

What are LSDs and how many patients are affected under India’s rare disease policy? 

LSDs, which are inherited metabolic conditions caused by a deficiency of specific enzymes, are covered under Group 3a of the NPRD. There are around 600 Group 3a patients registered for treatment under NPRD, according to data on the National Crowdfunding Portal set by the health ministry.

 

 

What concerns has the parliamentary forum raised? 

In its memorandum submitted to the Prime Minister and the Union Health Minister JP Nadda, the Indian Medical Parliamentarians’ Forum (IMPF) claimed that over 60 children and young adults had already died due to delays in therapy initiation or breaks in treatment, whereas nearly 100 are at imminent risk of discontinuation as funds stall.

 

What triggered this renewed call for action? 

This comes after several caregivers and advocacy groups for patients with 3a conditions had written a letter to Nadda last year, citing the inordinate delays being faced by patients eligible for treatment under the NPRD.

 

How does the policy currently support patients? 

Notified in 2021, the rare disease policy allows patients to be given a one-time financial assistance of Rs 50 lakh for enzyme replacement therapy (ERT).

 

“ERT for these conditions has been approved by the DCGI and used globally for over a decade, with clear evidence of improved survival, mobility, cognition, respiratory function and overall quality of life,” the memorandum added.

 

What gaps remain in the implementation of the rare disease policy? 

However, experts indicate that serious implementation gaps remain four years after the policy’s notification, leading to several patients losing their lives due to a lack of treatment.

 

ERT costs in India range from Rs 10 lakh to over Rs 1 crore annually, depending on the specific disease and the patient's weight. “Over 60 patients have exhausted one-time funds under the policy,” the forum stated.

 

What changes is the forum urging the Centre to consider? 

Calling the one-time fund ceiling of Rs 50 lakh insufficient without a mechanism for continuation funding, the forum asked the Centre to either waive or extend the funding cap for existing Group 3a LSD patients.

 

To counter this, the forum has recommended creation of a continuum-of-care funding framework by directing the Health Ministry, in consultation with the Ministry of Finance and NITI Aayog, to design a sustainable mechanism for uninterrupted, long-term therapy support for high-risk Group 3a patients.

 

“This could include a distinct pathway within NPRD for approved therapies with strong evidence of clinical efficacy, ensuring that once a patient is initiated on ERT, treatment is not abruptly withdrawn due to administrative caps,” they added.

 

What administrative challenges are patients facing at CoEs? 

People in the know added that except for insufficient one-time funds, the patients are facing administrative delays at the Centres of Excellence (CoEs), where centres are not fully utilising existing funds by on-boarding eligible patients.

 

“A strengthened oversight framework, with clear timelines and accountability for CoEs and implementing agencies, will help ensure that every rupee reaches the intended patient,” the memorandum added.