'National policy for rare diseases draft finalised' says Union Minister

Union Minister Ashwini Kumar Choubey addresses during a programme in Patna IANS
A draft 'National Policy for Rare Diseases' was finalized, Union Minister Ashwini Kumar Choubey, on Friday informed Parliament.

The MoS Health, in his written reply to a question asked in Lok Sabha, said "the draft has been placed on the website of the Ministry of Health and Family Welfare with a view to elicit comments and views of stakeholders, including the States and Union Territories and the general public by February 15.

The draft policy provides for lowering the incidence of rare diseases based on an integrated preventive strategy encompassing awareness generation and screening programmes and, within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases which are amenable to one-time treatment.


Choubey informed the House that number of persons suffering from diseases considered rare globally, was lacking in India and also there was limited data available on rare diseases. Therefore for the purpose of the policy the term 'rare diseases' shall construe three groups of disorders, identified and categorised by experts based on their clinical experience. The draft policy focused on prevention of rare diseases as a priority for all the three groups of rare diseases identified by experts.

The three groups under which diseases fall in rare categories have been identified as Group 1: Disorders amenable to one time curative treatment. This includes disorders amenable to treatment with Hematopoietic Stem Cell Transplantation (HSCT) and disorders amenable to organ transplantation.

Group 2 pertains to diseases requiring long term or lifelong treatment having relatively lower cost of treatment and benefit, has been documented in the literature and annual or more frequent surveillance is required. This has been subdivided in two more groups-Disorders managed with special dietary formulae or Food for special medical purposes (FSMP) and Disorders that are amenable to other forms of therapy (hormone/ specific drugs) .


Group 3 encompasses diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy.

For disorders like Wolman Disease, Hypophosphatasia, Neuronal ceroid lipofuscinosis, Cystic Fibrosis, Duchenne Muscular Dystrophy for which the cost of treatment is very high and either long term follow up literature is awaited or has been done on small number of patients was categorised as subgroup of group 3 of the rare diseases, said the minister.

"Diseases like Lysosomal Storage Disorders (LSDs), the Enzyme replacement Therapy (ERT) is presently not available", the minister informed the house.


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