UPSC Syllabus: Prelims: Social Issues | GS Paper II – Social Issues
- Caregivers to patients with ‘rare diseases’ and affiliated organisations are dissatisfied with the National Policy for Rare Diseases, 2021.
- Though the document specifies increasing the government support for treating patients with a ‘rare disease’— from ₹15 lakh to ₹20 lakh — caregivers say this doesn’t reflect actual costs of treatment.
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. However, different countries have their own definitions to suit their specific requirements and in context of their own population, health care system and resources.
|In the US, rare diseases are defined as a disease or condition that affects fewer than 200,000 patients in the country (6.4 in 10,000 people).|
India, like many other developing countries, currently has no standard definition of rare diseases and data on prevalence. Since there is no epidemiological data, there are no figures on burden of rare diseases and morbidity and mortality associated with them.To overcome this, a hospital based National Registry for Rare Diseases has been initiated by ICMR by involving centers across the Country that are involved in diagnosis and management of Rare Diseases. This will yield much needed epidemiological data for rare diseases.
So far only about 450 rare diseases have been recorded in India from tertiary care hospitals. The most common rare diseases include:
- Sicklecell Anaemia
- Primary Immuno Deficiency in children
- auto-immune diseases
- Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and
- Certain forms of muscular dystrophies.
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 ‘Rashtriya Arogya Nidhi Scheme’. The treatment will be limited to the beneficiaries of ‘Pradhan Mantri Jan Arogya Yojana’.
- 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.
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 – ‘Rashtriya Arogya Nidhi’ – 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.
|Case Study: What USA Does?
In United States of America, development of drugs for rare disease is sought to be encouraged through the Orphan Drugs Act, which incentivises industry by way of market exclusivity, grants to researchers and tax incentives on expenditure incurred during evaluation of drugs for their therapeutic potentials.