RARE DISEASE (Syllabus: GS Paper 3 – Sci & Tech)

Context: Recently, the Union Health Ministry of India revealed the launch of the first four domestically manufactured drugs for rare diseases, significantly reducing medication costs by up to a hundred-fold.

Rare Disease

• About: A rare disease is defined as often debilitating lifelong disease or disorder with a prevalence of one or less per 1,000 population, according to the World Health Organization (WHO).
• Also known as: It is also called Orphan Disease.
• The Union health ministry had formulated and launched the National Policy for Rare Diseases March 30, 2021.
• Type of Disease: Mostly considered genetic, these rare diseases are typically inherited from one generation to the next.
• Rare Disease in India: Haemophilia, Thalassemia, Sickle cell anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders.

National Policy for Rare Diseases (NPRD), 2021

• About: It provides financial assistance for a single treatment, with a cap of Rs. 20 lakh, introduces a crowdfunding mechanism, establishes a registry for rare diseases, and facilitates early detection.
• Aim: To enhance emphasis on domestic research and the local manufacturing of medications.
• Categories
• Group 1: Disorders eligible for a one-time curative treatment, with financial support of up to Rs. 20 lakh provided under the Rashtriya Arogya Nidhi umbrella scheme.
• Group 2: Conditions necessitating long-term or lifelong treatment.
• Group 3: Diseases with available definitive treatment, facing challenges such as optimal patient selection for benefits, very high costs, and the requirement for lifelong therapy.
• Rashtriya Arogya Nidhi: It is a centrally sponsored scheme which aims at providing financial assistance to the patients who live under the poverty line.