Health ministry adds six rare diseases under NPRD, 2021

New Delhi, March 9, 2023 :

The Union health ministry has included six rare diseases including Laron’s syndrome, Wilson’s disease and hypophosphatemic rickets among others into various groups of disorders under the National Policy for Rare Diseases (NPRD), 2021, enabling the patients of these diseases to avail financial assistance for treatment.

The decision is taken based on the NPRD, launched in March, 2021 for the treatment of rare disease patients by categorising the diseases into three groups.

The Group One include disorders amenable to one-time curative treatment; Group Two include 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; and Group Three comprises of diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy.

According to an office memorandum released by the ministry recently, Laron’s Syndrome, a rare disease in which the body is unable to utilise the growth hormones resulting in short stature, has been added under the Group One.

Wilson’s disease, a rare disorder in which copper is accumulated in vital organs like liver, brain and others; Congenital adrenal hyperplasia (CAH), a group of genetic disorders that affect the adrenal glands; and neonatal onset multisystem inflammatory disease (NOMID) that causes continuous inflammation and tissue damage impacting the nervous system and joints, are included under the Group Two disorders, under the disorders that are amenable to other forms of therapy including hormone and specific drugs.

Under the Group Three, hypophosphatemic rickets, caused due to the defects in the renal handling of phosphorus; and atypical hemolytic uremic syndrome (AHUS), which affects kindly function, were added through the office memorandum.

The financial assistance to the patients suffering from these rare diseases shall be provided as per the provisions envisaged under the NPRD, 2021 and guidelines and procedures issued to all Centres of Excellence (CoEs).

Under the Policy, the 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. The MoHFW has been identifying CoEs for diagnosis, prevention and treatment of rare diseases.

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.

The Ministry has earlier said that in addition to various such measures, department of revenue, ministry of Finance through a notification in February, 2022 has given exemption from Basic Customs Duty to drugs or medicines, which are used in the treatment of rare diseases when imported by Centres of Excellence (CoEs) as specified in NPRD, 2021 or any person or institution on recommendation of any Centre of Excellence listed in NPRD, 2021, certifying that the person (by name) for whom the drugs or medicines are imported, is suffering from a rare disease (to be specified by name) and requires the drugs or medicines for the treatment of said rare disease. PharmaBiz