Centre mulls adding more Centres of Excellence for rare disease patients

2-3 new CoEs likely to be added to the current count of 12

The Union Ministry of Health and Family Welfare is discussing adding more Centres of Excellence (CoEs) to its network of centres to improve access to diagnosis and treatment for patients suffering from rare diseases.

 

“The ministry is exploring options to add at least two to three such centres in the coming year,” an official in the know told Business Standard.

 

CoEs are institutions identified by the central government under the National Policy for Rare Diseases (NPRD), 2021, to treat patients suffering from rare diseases actively. There are 12 such centres with a quota of 2,420 rare disease patients from six categories across three groups.

 

The World Health Organisation (WHO) defines a rare disease as a debilitating lifelong disease or disorder with a prevalence of 10 or fewer per 10,000 population. While India lacks such epidemiological data, the NPRD covers 63 rare diseases across three different groups based on the kind of treatment needed.

 

The move to increase the number of centres comes as patients belonging to several rare disease groups have flagged issues ranging from lack of institutional care at CoEs to the exhaustion of the one-time fund of Rs 50 lakh given to patients.

 

An official with a patient advocacy group said that the challenges faced by these patients are deeply concerning, particularly due to the inadequacy of the Rs 50 lakh one-time funding model, which fails to support sustained treatment for chronic conditions like Lysosomal storage disorders (LSDs). 

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“Many patients, having exhausted this financial aid, are now left in a dire situation with no sustainable funding,” he added.

 

Recently, patients with rare genetic LSDs under Group 3a of NPRD wrote to the Union health ministry, saying that they have been facing severe health issues after exhausting their funds for treatment.

 

Group 3a includes conditions such as LSDs, for which definitive treatment is available, but challenges include optimal patient selection for benefit, very high costs, and lifelong therapy.

 

“There are currently around 454 eligible rare disease patients with LSDs such as Pompe disease (Glycogen storage disease type II), Anderson–Fabry disease, and Mucopolysaccharidosis types I and II in India, who need to be put on immediate life-saving therapy, according to NPRD 2021,” an official from an advocacy group said.

 

Caregivers have also complained that the lack of a centralised, long-term funding mechanism puts lives at risk. The ineffective utilisation of allocated funds by CoEs, coupled with delays in approvals, further worsens the problem.

 

“A centralised, sustainable funding model under NPRD 2021, enhanced accountability in fund utilisation, and legislative efforts to ensure equal prioritisation of all rare diseases is the need of the hour,” the official added.

 

Commenting on the delays in the treatment of their children, a Delhi-based parent of a six-year-old girl suffering from the rare Gaucher’s disease said that his daughter’s health has alarmingly deteriorated due to the stoppage of treatment at All India Institute of Medical Sciences Delhi, since August this year. He added that the last time her treatment stopped, his daughter experienced hearing loss.

 

Similarly, a caregiver from Uttar Pradesh said that his 10-year-old son’s condition has worsened due to the discontinuation of treatment since September. “He has been getting episodes of fever and cough as his health becomes unpredictable. I urge the government to release immediate funds for my son,” he added.