National Policy on Rare Diseases

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.

A rare disease is a health condition of low prevalence that affects a small number of people compared with other prevalent diseases in the general population. It is estimated that globally around 6000 to 8000 rare diseases exist with new rare diseases being reported in the medical literature regularly. However, 80% of all rare disease patients are affected by approximately 350 rare diseases. Lysosomal storage disorders, Gaucher disease, Pompe disease, MPS 1 and 2 and Fabry disease are some of the rare diseases. 

In March, 2021, the Government has launched National Policy for Rare Diseases (NPRD), 2021,  for the treatment of rare disease patients. The salient features of NPRD, 2021 are as under: The rare diseases have been identified and categorized into 3 groups namely Group 1, Group 2 and Group 3. 

• Group 1: Disorders amenable to one-time curative treatment. 
• Group-2: Diseases requiring long term/lifelong treatment having relatively lower cost of treatment and benefit have been documented in literature and annual or more frequent surveillance is required. 
• Group 3: Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy. 

Provision for financial support of up to Rs. 50 lakhs to the patients suffering from any category of the Rare Diseases and for treatment in any of the Centre of Excellence (CoE) mentioned in NPRD-2021, outside the Umbrella Scheme of Rashtriya Arogaya Nidhi. 

In order to receive financial assistance for treatment of rare disease, the patient of the nearby area may approach the nearest Centre of Excellence to get him assessed and avail the benefits. 

Eight Centers of Excellence (CoEs) have been identified for diagnosis, prevention and treatment of rare diseases. Five Nidan Kendras have been set up for genetic testing and counseling services. 

The NPRD, 2021 has provisions for promotion of research and development for diagnosis and treatment of rare diseases; promotion of local development and manufacture of drugs and creation of a conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices.

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