RARE DISEASE - HEALTH
News: Delhi High Court steps in for patients of rare diseases, sets up panel
to implement Centre’s policy
What is in the news?
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Recently, 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 reach patients.
Key takeaways from the news:
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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.
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Under the National Policy
for Rare Diseases, 2021, hundreds of patients, mostly children, are registered
with the ‘Digital Portal for Crowdfunding and Voluntary Donations for Patients
of Rare Diseases’ by the Union Health Ministry.
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However, the Government has been unable to
fully finance their treatment due to high costs, resource constraints, and
competing health priorities.
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The court ordered the
panel to explore procurement of therapies and drugs, and the creation of an
associated logistical framework to administer treatment.
What is a rare disease?
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Rare Disease (RD) is
defined by the World Health Organization (WHO) as an often debilitating,
lifelong disease or disorder with a prevalence of 1 or less per 1,000
population.
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However, there is no
universal definition as different countries define rare diseases differently,
with India recording about 450 rare diseases that affect at least 70 million people.
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Example:
Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular
dystrophy, spina bifida, hemophilia etc.
Issues in handling rare disease:
1. High cost:
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Treatment for rare
diseases requires higher cost for medicine, diagnosis etc.
2. Resource constraints:
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Lack of treatment
facilities, medicines in India is one of the issues in the rare disease.
3. Delayed Diagnosis:
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Rare diseases are often
difficult to diagnose due to their complex nature and limited understanding.
4. Competing health priorities:
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Competing health
priorities on other health issues often leads to delay in the treatment of rare
disease.
5. Inadequate Research and Development:
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Research and development
in the field of rare diseases in India are relatively limited.
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There is a lack of dedicated research institutes,
funding, and clinical trials focused on rare diseases.
6. Lack of coordination:
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Lack of central coordination among the centers of
excellence delays the progress in the research and development in the area
of rare disease.
7. Lack of Awareness:
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One of the primary
challenges is the lack of awareness about rare diseases among the general
public, healthcare professionals, and even policymakers.
8. Delayed Diagnosis:
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Rare diseases are often difficult to diagnose due to their
complex nature and limited understanding.
Rare Disease Policy:
1. Aim:
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Increase focus on
indigenous research and local production of medicines.
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Lower the cost of treatment
of rare diseases.
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Screen and detect rare
diseases early for prevention.
2. Categorization of the disease:
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Group
1: Disorders amenable to one-time curative treatment.
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Group
2: Diseases requiring long-term or lifelong treatment.
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Group
3: Diseases with available treatment but challenges in
patient selection, high cost, and lifelong therapy.
3. Financial Support:
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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)
outside the Umbrella Scheme of Rashtriya
Arogaya Nidhi.
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Financial support of up
to Rs. 20 lakhs under Rashtriya Arogya Nidhi for rare diseases listed under
Group 1.
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Rashtriya Arogya Nidhi
provides assistance to patients with major life-threatening diseases,
regardless of their poverty status.
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Voluntary
crowdfunding for treatment through a digital platform
for individual and corporate contributions.
4. Centers of Excellence:
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Designation of eight
health facilities as 'Centres of
Excellence'.
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One-time
financial support of up to Rs. 5 crore
for upgrading diagnostic facilities.
5. National Registry:
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Creation of a national
hospital-based registry of rare diseases.
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Ensuring comprehensive
data and definitions for research and development purposes.
WAY FORWARD:
1. Awareness Promotion:
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Government should promote
awareness about the rare disease among healthcare professionals, the general
public etc.
2. Financial assistance:
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The government can play a
significant role by providing financial
support for treatment, offering subsidies for orphan drugs, and
establishing dedicated centers of excellence for rare diseases.
3. Domestic Drug availability:
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The Government through incentives like PLI schemes can
increase drug manufacturing among the private sectors.
4. Coordination with NGO:
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Government should provide
necessary assistance to NGOs to enable them to promote awareness, identify
patients etc.
5. International Collaboration:
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Collaborating with
international rare disease organizations, research institutions, and
pharmaceutical companies can facilitate knowledge exchange, access to
expertise, and participation in global research initiatives.