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.
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.
- 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.
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:
- 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.
- 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