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:
- What are ‘Rare diseases’?
- Pressing Issues regarding ‘Rare diseases’
- National Rare Diseases Committee
- 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
