Only four out of twelve Centres of Excellence (CoEs) for rare diseases have utilised funds provided to them by the Government of India under the National Policy for Rare Disease (NPRD) in the last three years, according to patient advocacy groups associated with people suffering from conditions notified under Group 3a of NPRD.
According to data accessed by Business Standard, only four CoEs had utilised upwards of 87 per cent of the funds allocated to them since 2021, while the fund utilisation rate for seven other centres ranged from 4 to 57 per cent between 2021-22 and 2023-24.
The twelfth centre, All India Institute of Medical Sciences (Aiims), Bhopal has only been designated as a CoE in November last year.
CoEs are institutions identified by the central government to actively manage patients suffering from rare diseases. Currently, 12 such centres enrol nearly 2,420 rare disease patients from six categories across three groups.
“While the government has set up CoEs to treat patients with rare diseases, patients with Group 3a conditions still experience inordinate delays in procedural formalities across these centres,” an official with an advocacy group for patients with Group 3a conditions said.
According to the rare disease policy enacted in 2021, Group 3a includes conditions such as Lysosomal Storage Disorders (LSDs) for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost, and lifelong therapy.
“There are currently around 454 eligible rare disease patients with LSDs such as Pompe disease, Fabry disease, Mucopolysaccharidosis (MPS) type I and type II in India, to be put on immediate life-saving therapy as per the National Policy for Rare Diseases 2021,” the expert added.
According to data available on the government’s digital portal for crowdfunding and voluntary donations for patients of rare diseases, more than 300 out of 454 LSD patients currently eligible for funding under Group 3a are not getting any treatment in CoEs.
Currently, only 98 LSD patients are on government-approved treatment.
“The delay in the commencement of the treatment process and uncertainties around sustainable funding of Group 3a disorders are turning fatal, with around 28 of these 454 patients reported to have lost their lives in the last few months awaiting treatment support,” an official said.
The groups have asked the government to direct CoEs to ensure efficient utilisation of funds provisioned by the ministry.
This comes after several caregivers and advocacy groups for patients with 3a conditions wrote a letter to Health Minister J P Nadda last month, citing the inordinate delays being faced by patients eligible for treatment under the NPRD.
The letter had stated that several patients across the CoEs have been put off life-saving therapies after the one-time support of Rs 50 lakh was exhausted.
“No urgency being shown by centres,” say distressed parents
A Delhi-based parent whose six-year-old child is diagnosed with MPS-2 said that he has been waiting to get his child on-boarded at the ministry’s designated CoE for treatment, despite his child being eligible to be put on life-saving therapy as per the guidelines of the NPRD.
“My child deserves to lead a near-normal life and I urge the government to intervene in the matter to save the lives of hundreds of other children as well,” he added.
Similarly, a Hyderabad-based parent of an eight-year-old Pompe disease patient said that while his child is eligible for Enzyme Replacement Therapy (ERT) with the available Rs 50 lakh funding, he still has to run from pillar to post to get it.
“My child could be saved if he gets regular ERT sessions, but still I do not see any urgency shown by the hospital despite available funds. We are in distress as we are concerned about our kid’s life, his health is deteriorating,” he added.
Highlighting the need to ensure timely treatment for patients with Group 3a disorders, a medical expert said that patients with LSDs are largely young children and the delay in medical treatment could lead to life-threatening complications.
“These diseases have therapies approved by the Drugs Controller General of India (DCGI) and are available in India with proven clinical outcomes, as well as available government funding. Despite this, it is disappointing to see patients waiting for the inevitable,” he said.
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