Concerns over the benefits of the National Policy of Rare Diseases (NPRD) not reaching to patients

Rajya Sabha member from the Nationalist Congress Party raised concerns over the benefits of the National Policy of Rare Diseases (NPRD) not reaching patients even after several months since its introduction.

Concerns

• 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 crowd­funding platform revealed that around 190 patients diagnosed with ultra­rare genetic conditions could be immediately put on lifesaving therapy.

National Policy for Rare Diseases (NPRD), 2021

The Government has launched National Policy for Rare Diseases (NPRD), 2021 in March, 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 has 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.

• Five Nidan Kendras have been set up for genetic testing and counselling services.

Centres of Excellence

• In order to provide facilities for treatment and care of patients suffering from rare diseases,eight (08) Centres of Excellence have been notified, which are premier Government tertiary hospitals with facilities for diagnosis, prevention and treatment of rare diseases.