NEW DELHI: The National Policy for Rare Diseases 2021 (NPRD) was notified on March 31. The policy provides financial support of up to Rs 20 lakh, under Rashtriya Arogya Nidhi, for the treatment of rare diseases (RD) listed under group 1 (amenable to one time treatment- either hematopoietic stem cell transplant or organ transplant). Beneficiaries for such financial assistance would not be limited to BPL families, but extended to about 40% of the population, who are eligible as per Pradhan Mantri Jan Arogya Yojana, for their treatment in government tertiary hospitals only.
However, the NPRD does not provide any financial support for patients of Group 3 diseases, which have a definite treatment but is life-long and expensive. It is estimated that for a child weighing 10 kg, the annual cost of treatment for some rare diseases may vary from Rs 10 lakh to more than Rs 1 crore per year with treatment being lifelong and drug dose and cost increasing with age and weight. For such patients, the government will set up a crowd-funding platform where volunteers, both corporate and individuals, can donate money for their treatment.
As per WHO, diseases that affect less than 5 people per 10,000 are referred to as rare, and there are about 7,000-8,000 such diseases. Rare diseases impact about 7 crore Indians. Less than 5% of RDs can be treated and where drugs are available they are prohibitively expensive, placing immense strain on resources. About 80% of RDs are genetic in nature and particularly impact children, causing 35% of deaths before the age of one.
As per the NPRD 2021, the central government will notify selected ‘centres of excellence’ (COE), which will be premier government tertiary hospitals with facilities for diagnosis, prevention and treatment of rare diseases. To begin with, eight such centres have been notified. These COEs will be provided one-time grant subject to a maximum of Rs 5 crore each for infrastructure development for screening, tests, treatment, if such infrastructure is not available.
The policy also states the government’s intent to create more awareness around screening of RDs and also develop R&D for production of expensive medicines needed for treatment of Group 3 RDs.
Families of RD patients and their advocacy groups, however, are unhappy with the lack of financial support for diagnosed Group 3 diseases patients in NPRD 2021. “In the absence of any funding support for life-saving therapies, close to 130 odd patients are left with no option but to wait for the inevitable,” said Manjit Singh, national president of Lysosomal Storage Disorders Society of India.
Patients and their support groups had recently written to the health ministry seeking creation of a seed-funding of Rs 80-100 crore while rolling out the national policy so that life-saving therapies to all those patients with treatable Group 3 disorders, for which DCGI-approved treatment is available, can be provided, thereby reducing any further loss of life.