National Rare Diseases Committee

National Rare Diseases Committee

Q. Why is it in News/Context?

·   The Delhi High Court has set up a five-member panel to implement the Centre’s rare diseases policy in an efficient manner and ensure that its benefits reaches patients.

Q. What is National Rare Diseases Committee?

·  The Delhi HC recently constituted a five-member ‘National Rare Diseases Committee’ to implement the National Rare Disease Policy-2021 in an efficient manner and to “ensure that the benefits of the policy reach the ultimate patients of rare disease”.

·   The National Rare Diseases Committee will also look into the case of these patients, enrolled with the All India Institute of Medical Sciences (AIIMS), Delhi, and the manner in which their treatment can begin.

Q. What are Rare Diseases?

·  WHO defines rare disease as often debilitating lifelong disease or disorder with aprevalence of 1 or less, per 1000 population

The Indian Scenario

·   So far only limited number of diseases has been recorded in India from tertiary care hospitals that are globally considered as rare diseases though ambit may encompassfrom 7000 to 8000 disorders.

·  The commonly reported diseases include Primary immunodeficiency disorders, Lysosomal storage disorders (Gaucher’s disease, Mucopolysaccharidoses, Pompe disease, fabry disease etc.) small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias etc.), Cystic Fibrosis,osteogenesisimperfecta, certain forms of muscular dystrophies and spinal muscularatrophy,etc.

Q. What are salient features of National Policy for Rare Disease 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.

·  The Policy aims at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy encompassing awareness generation, premarital, post-marital, pre-conception and post-conception screening and counselling programmes to prevent births of children with rare diseases, and within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases.

Initiatives for treatment support for patients of rare diseases under the Policy are as follows: -

· Financial support up to Rs. 20 lakhs under the Umbrella Scheme of RashtriyaArogayaNidhishall be provided by the Central Government for treatment, of those rare diseases that require a one-time treatment (diseases listed under Group 1). Beneficiaries for such financial assistance would not be limited to BPL families, but extended to about 40% of the population, who are eligible as per norms of Pradhan Mantri Jan ArogyaYojana, for their treatment in Government tertiary hospitals only. 

·   State Governments can consider supporting patients of such rare diseases that can be managed with special diets or hormonal supplements or other relatively low-cost interventions (Diseases listed under Group 2).

·   Keeping in view the resource constraints, and a compelling need to prioritize the available resources to get maximum health gains for the community/population, the Government will endeavour to create alternate funding mechanism through setting up a digital platform for voluntary individual and corporate donors to contribute to the treatment cost of patients of rare diseases.

·  Voluntary crowd-funding for treatment: Keeping in view the resource constraint and competing health priorities, it will be difficult for the Government to fully finance treatment of high-cost rare diseases. The gap can however be filled by creating a digital platform for bringing together notified hospitals where such patients are receiving treatment or come for treatment, on the one hand, and prospective individual or corporate donors willing to support treatment of such patients.

·   Conferences will be organized with corporate sector companies to motivate them to donate generously through digital platform. Ministry of Corporate Affairs will be requested to encourage PSUs and corporate houses to contribute as per the Companies Act as well as the provisions of the Companies (Corporate Social Responsibility Policy) Rules, 2014 (CSR Rules). Promoting health care including preventive health care is included in the list in the Schedule for CSR activities.

·   Treatment cost of the patient will be first charge on this fund. Any leftover fund after meeting treatment cost can be utilized for research purpose also.

At present financial assistance to poor patients, living below threshold poverty line and also to the population, who are eligible as per norms of Pradhan Mantri Jan ArogyaYojana under Ayushman Bharat, suffering from specified rare diseases for their treatment at Government Hospitals or Institutes having super specialty facilities / Government tertiary hospitals is being provided under the Umbrella Scheme of RashtriyaArogyaNidhi (RAN).

·   National Policy for Rare Diseases, 2021 provides for National Consortium for Research and Development on therapeutics for Rare Diseases with an expanded mandate to include research & development, technology transfer and indigenization of therapeutics for rare diseases. It will be convened by Department of Health Research (DHR) with ICMR as a member.

·    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 (08) Centres of Excellence (CoEs) have been identified for diagnosis, prevention and treatment of rare diseases.

·        Five NidanKendras have been set up for genetic testing and counselling services.

Ø Lady Hardinge Medical College (LHMC), Delhi

Ø Nizam’s Institute of Medical Sciences (NIMS), Hyderabad, Telangana

Ø All India Institute of Medical Sciences (AIIMS), Jodhpur

Ø Army Hospital Research & Referral, Delhi

Ø Nil RatanSircar (NRS) Medical College and Hospital, Kolkata 

·   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 conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices.

·  Department of Pharmaceuticals has initiated the implementation of Production Linked Incentive Scheme for Pharmaceuticals. The Scheme provides for financial incentives to manufacturers selected under the Scheme for domestic manufacturing of various product categories, which also include Orphan drugs.

·      Department of Revenue, Ministry of Finance gives full waiver of Basic Customs Duty (BCD) and Integrated Goods and Services Tax (IGST) to drugs imported (personal use only) for treatment of Spinal Muscular Atrophy (SMA) rare disease, thereby making the medicines for SMA rare disease more affordable.

·   In addition, Department of Revenue, Ministry of Finance 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.