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Why in the news?

The National Rare Disease Policy 2021 was recently approved by the Ministry of Health and Family Welfare. The Delhi High Court had previously ordered the Centre to establish a Rare Diseases Committee and a Rare Diseases Fund, as well as to finalize and notify the National Health Policy for Rare Diseases.

What is a rare disease?

• WHO defines rare disease as often debilitating lifelong disease or disorder with a prevalence of 1 or less, per 1000 population. In the USA, a rare or orphan disease is defined strictly according to its prevalence, specifically” any disease or condition that affects less than 200,000 persons in the United States,” or about 1 in 1,500 people.
• European Union (EU) considers diseases affecting fewer than 1 in 2,000 persons to be rare. In Japan a rare disease is defined as affecting not more than 1 in 2500 people. Considering the population of India the suggested threshold for India for a disease to be defined as rare is 1 in 10,000.
• 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.
• Rare diseases include rare cancers, autoimmune diseases, congenital malformations, and infectious diseases amongst others. About half of the rare diseases affect children while the remaining manifest in adulthood. Some examples of rare diseases include Hemangiomas, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, muscular dystrophies and Pompe disease

National Policy for Rare Diseases, 2021:

• It offers financial support for one-time treatment of up to ?20 lakh,
• Introduces a crowd funding mechanism,
• Creates a registry of rare diseases, and
• Provides for early detection.
• The policy has categorised rare diseases in three groups:
  • Group 1: Disorders amenable to one-time curative treatment.
  • Group 2: Those requiring long term or lifelong treatment.
  • 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.

Key Features of the Policy:

• A patient registry of rare diseases is to be constituted under ICMR (Indian Council of Medical Research).
• According to the policy, rare diseases include genetic diseases, rare cancers, infectious tropical diseases, and degenerative diseases.
• Under the policy, there are three categories of rare diseases-
  • Requiring one-time curative treatment include osteopetrosis and immune deficiency disorders, among others.
  • diseases that require long-term treatment but where the cost is low, and
  • Those needing long-term treatments with high cost.
• As per the policy, the assistance of Rs 15 lakh will be provided to patients suffering from rare diseases that require a one-time curative treatment under the ‘RashtriyaArogyaNidhi Scheme’. The treatment will be limited to the beneficiaries of ‘Pradhan Mantri Jan ArogyaYojana’.
• For diseases listed under Group 2, 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.
• Under the policy, certain medical institutes will be certified as Centre of Excellence for rare diseases. It includes AIIMS, New Delhi; Sanjay Gandhi Post Graduate Institute of Medical Science, Lucknow; King Edward Medical Hospital, Mumbai and four others.
• There are certain diseases such as Hurler Syndrome, Gaucher’s disease, Wolman disease for which the annual treatment expenses may vary from Rs 10 lakh to Rs 1 crore. For such diseases, a digital platform is to be set up to raise donations and corporatefunding.
• It proposes an inter-ministerial consultative committee is to be set up at national level. The committee will be led by MoHFW.
• It also aims to create Administrative Committee that will develop guidelines to determine which rare diseases to fund.

Issues with the policy:

• According to the Policy, diseases like LSD have been classified as Group 3 since definitive treatment is available but costs are prohibitive.
• However, no funds have been set aside for immediate and long-term care needs for treatments that have already been approved by India’s Drugs Controller General.
  • Experts point out that the costs to help already-diagnosed patients might be in the range of 80-100 crore annually.
  • If the Centre can extend the cost-sharing agreements that it has worked out with Kerala, Tamil Nadu and Karnataka, with other States too, its share of the annual costs will be halved.

Criticism of the Policy:

• Rare disease advocacy and support groups have called out the policy for several discrepancies including a lack of funding to support families and individuals with a rare disease.
• They have pointed out that the proposed allocation of financial support has been brought down to a maximum of Rs 15 lakh per case under an umbrella scheme – ‘RashtriyaArogyaNidhi’ – for those who have a rare disease that falls in Group 1, and require one-time treatment. This is much lower than the initially proposed corpus fund of Rs 100 crore.
• The policy also passes the buck, and says that crowdfunding can be used to provide financial support.
• It identifies eight hospitals across India as ‘centres of excellence’ and leaves it up to them to set up crowdfunding initiatives for patients that require it, or for families to themselves look at crowdfunding options for treatment.
• The policy caps resources for different diseases. While treatment for one particular disease might amount to Rs 10 lakh, another disease may require Rs 1 crore to cover the cost of medications, which are only bound to increase over time.
• The new policy offers no support to patients awaiting treatment since the earlier National Policy for Treatment of Rare Diseases 2017 was kept in abeyance.
• The new policy has absolutely no consideration for Group 3 patients, who require lifelong treatment support
• The policy has been criticised for acting more like a set of guidelines to understand the disease, rather than specifying actions which must be taken by the government to offer support to those affected.
• Public health and hospitals being a State subject, the Central Government shall encourage and support the States in their endeavour towards screening and prevention of rare diseases. However, while screening has been emphasised as a preventive measure, there is no mention of how and when screening will take place, nor has it been stated how screening will be implemented.

Government Support:

• The government will provide financial support of up to Rs. 20 lakh under the Umbrella Scheme of Rashtriya Arogya Nidhi for treatment of those rare diseases listed under Group 1.
• Moreover, Beneficiaries for such financial assistance would not be limited to BPL families. About 40% of the population, eligible under Pradhan Mantri Jan ArogyaYojana, will also be eligible for assistance.
• Further, for group 2, the State Governments can consider supporting specific patients. It includes a rare disease that can be managed with special diets or hormonal supplements or other relatively low-cost interventions (Diseases listed under Group 2).
• Voluntary Crowd funding: The government has said that it will assist in voluntary crowd-funding for the treatment of Group 3. It is because it will be difficult to fully finance the treatment of high-cost rare diseases of Group 3.

What USA Does?

The Orphan Drugs Act, which incentivizes industry through market exclusivity, grants to researchers, and tax credits on expenditure incurred during drug evaluation for therapeutic potential in the United States, aims to promote the production of drugs for rare diseases.

Way forward:

• Even as the program is being implemented, the Centre will set aside a significant sum to support life-saving treatments.
• Doing so would not only complete a job well started, even if only half completed, but will also reaffirm the government’s commitment to the welfare of every Indian citizen..

Mains oriented question:

What was need of national policy for rare diseases? What are criticism associated with it? (200 words)