Rajya Sabha MP from the Nationalist Congress Party Fauzia Khan on Friday raised concerns over the benefits of the National Policy of Rare Diseases (NPRD) not reaching any patient with rare diseases even after several months since its introduction.
The Union Ministry of Health and Family Welfare notified the NPRD in March 2021. In May, it increased the funding support to ₹50 lakh per patient with diseases classified as rare for their treatment.
However, the MP alleged that the “unending delay and lack of urgency” on the part of the Centres of Excellence (CoE), designated as per the policy, has claimed several young lives and endangered the survival prospects of 415 patients, largely children, diagnosed with rare diseases.
These diseases included Lysosomal storage disorders, Gaucher disease, Pompe disease, MPS 1 and 2 and Fabry disease. An analysis of data from the national crowd-funding platform revealed that around 190 patients diagnosed with ultra-rare genetic conditions could be immediately put on life-saving therapy, she added.
She said that majority of these patients have been diagnosed with Gaucher disease, for which therapy approved by Drug Controller General of India is available for many years. She pointed out that many CoEs were yet to seek financial support as per the policy for treating the patients. She urged the Ministry to issue urgent instructions to all the CoEs to commence treatment for these patients.