About The National Rare Diseases Committee

Context:

Recently, the Delhi High Court has taken a proactive step to address the challenges faced by patients with rare diseases by establishing a five-member panel to implement the Centre’s rare diseases policy effectively.

• The panel, known as the National Rare Diseases Committee, aims to ensure that patients enrolled with the All-India Institute of Medical Sciences (AIIMS), Delhi, receive timely treatment and benefit from the policy.

Relevance:

GS II: Government Policies and Interventions

Dimensions of the Article:

1. What are ‘Rare diseases’?
2. Pressing Issues regarding ‘Rare diseases’
3. National Rare Diseases Committee
4. Provisions of the National Rare Disease Policy 2021

What are ‘Rare diseases’?

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.

Most rare diseases are genetic, and are present throughout a person’s entire life, even if symptoms do not immediately appear.

• Haemophilia,
• Thalassemia,
• Sickle-cell anaemia,
• Auto-immune diseases,
• Pompe disease,
• Hirschsprung disease,
• Gaucher’s disease,
• Cystic Fibrosis,
• Hemangiomas and
• Certain forms of muscular dystrophies

Are some of the most common rare diseases recorded in India.

Pressing Issues regarding ‘Rare diseases’

• Rare diseases pose a significant challenge to health care systems because of the difficulty in collecting epidemiological data, which in turn impedes the process of arriving at a disease burden, calculating cost estimations and making correct and timely diagnoses, among other problems.
• There are 7,000-8,000 classified rare diseases, but less than 5% have therapies available to treat them.
• About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease-specific treatment. Where drugs are available, they are prohibitively expensive, placing immense strain on resources.
• These diseases have differing definitions in various countries and range from those that are prevalent in 1 in 10,000 of the population to 6 per 10,000.
• India has said it lacks epidemiological data on the prevalence here and hence has only classified certain diseases as ‘rare.’
• Currently, only a few pharmaceutical companies are manufacturing drugs for rare diseases globally and there are no domestic manufacturers in India except for those who make medical-grade food for those with metabolic disorders.
• Due to the high cost of most therapies, the government has not been able to provide these for free.

National Rare Diseases Committee:

The National Rare Diseases Committee is a panel established by the Delhi High Court to address the challenges faced by patients with rare diseases and implement the rare diseases policy.

Composition:

The committee is composed of five members who are experts in relevant fields, including medical professionals, policymakers, and representatives from healthcare institutions.

Responsibilities and Objectives:

Assessing Cases:

• The committee focuses on patients enrolled with AIIMS in Delhi.
• It evaluates individual cases to understand the medical needs of patients with rare diseases and determine appropriate treatment options.

Implementation of the Policy:

• The committee devises strategies and guidelines for translating the provisions of the rare diseases policy into actionable steps.
• It ensures that the policy is effectively implemented to benefit patients with rare diseases.

Coordination and Collaboration:

• The committee facilitates close coordination between the medical community, therapy providers, and governmental agencies.
• It creates a collaborative environment to address the challenges associated with rare diseases and find collective solutions.

Treatment Accessibility:

• One of the committee’s objectives is to ensure timely access to treatment for patients with rare diseases.
• It explores avenues for procuring necessary therapies and drugs and establishes a logistical framework for the seamless administration of treatment.

Provisions of the National Rare Disease Policy 2021

• Patients of rare diseases will be eligible for a one-tome treatment under the Ayushman Bharat Pradhan Mantri Jan Arogya Yojana (AB-PMJAY).
• Financial support up to Rs20 lakh under the Umbrella Scheme of Rashtriya Arogaya Nidhi shall be provided by the central government for treatment of those rare diseases that require a one-time treatment (diseases listed under Group 1) for their treatment in Government tertiary hospitals only. – (NOT be limited to below poverty line (BPL) families, but extended to about 40% of the population as eligible under the norms of Pradhan Mantri Jan Arogya Yojana (PMJAY))

The policy has categorised rare diseases in three groups:

• Disorders amenable to one-time curative treatment;
• Those requiring long term or lifelong treatment; and
• Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit.

The government has said that it will also assist in voluntary crowd-funding for treatment as it will be difficult to fully finance treatment of high-cost rare diseases.

Criticisms of the National Rare Disease Policy 2021

• Though the document specifies increasing the government support for treating patients with a ‘rare disease’— from Rs 15 lakh to Rs 20 lakh — caregivers say this doesn’t reflect actual costs of treatment.
• The Policy leaves patients with Group 3 rare diseases to fend for themselves due to the absence of a sustainable funding support.
• What the policy doesn’t capture is that these are diseases that last a lifetime adding up to a huge amount of expenditure and many of the patients who can’t afford such treatment will be unable to even make it to the prescribed tertiary hospitals for treatment.

-Source: The Hindu