In the news

• World Rare Diseases Day (February 29) was recently celebrated.

What are Rare Diseases?

• Global Perspective: Rare diseases are defined by the World Health Organization (WHO) as often debilitating lifelong diseases or disorders with a prevalence of 1 or less, per 1,000 population.
• National Context: While India lacks a standardized definition, the Organisation of Rare Diseases – India suggests defining a disease as rare if it affects 1 in 5,000 people or less.

National Policy on Rare Diseases, 2021: Highlights

• Comprehensive Approach: This Policy offers a holistic framework encompassing prevention, management, and treatment strategies tailored to the unique needs of patients.
• Financial Support: Recognizing the financial burden on patients, the policy aims to lower the exorbitant costs of treatment through targeted interventions and support mechanisms.
• Research Focus: Emphasizing indigenous research, the policy lays the foundation for bolstering research initiatives in the field of rare diseases, fostering innovation and discovery.

Other Initiatives in India

• National Hospital-Based Registry: A pivotal component of the policy, the establishment of a national registry of rare diseases promises to provide invaluable epidemiological data, informing targeted interventions and resource allocation.
• Early Screening and Prevention: The creation of Nidan Kendras aims to enhance early detection and prevention efforts, crucial for improving patient outcomes and reducing disease burden.
• Capacity Building: Strengthening secondary and tertiary health facilities at Centres of Excellence underscores the commitment to enhancing healthcare infrastructure and service delivery.

Challenges and Imperatives

• Defining Rare Diseases: Despite significant progress, India lacks a standardized definition of rare diseases, necessitating clarity to guide policy and resource allocation effectively.
• Funding Utilization: Concerns arise over the underutilization of allocated funds, highlighting the urgency to streamline resource allocation and enhance accountability mechanisms.
• Patient Advocacy: Rare diseases patient advocacy groups play a pivotal role in advocating for timely access to treatment and sustainable funding support, urging policymakers and healthcare providers to prioritize patient-centric initiatives.

Way Forward

• Sustainable Funding: Ensuring sustainable funding support for rare diseases treatment is paramount to safeguarding patient well-being and fostering equitable access to care.
• National Registry Implementation: Accelerating the establishment of a hospital-based national registry is imperative to harness the power of data-driven decision-making and advance rare diseases research.
• Multidisciplinary Care: The creation of comprehensive care centers, coupled with initiatives to support caregivers, represents a crucial step towards enhancing patient outcomes and fostering a supportive healthcare ecosystem.

Conclusion

• As India commemorates World Rare Diseases Day, it stands at a pivotal juncture in its journey towards rare diseases care and advocacy.
• By embracing a collaborative and patient-centric approach, India can surmount existing challenges, paving the way for a future where every individual affected by a rare disease receives the care and support they deserve.

Try this PYQ from CSP 2014:

Consider the following diseases

1. Diphtheria
2. Chickenpox
3. Smallpox

Which of the above diseases has/have been eradicated in India?

(a) 1 and 2 only

(b) 3 only

(c) 1, 2 and 3

(d) None

Get an IAS/IPS ranker as your 1: 1 personal mentor for UPSC 2024

Attend Now