Facts for Prelims (FFP)
Source: ET
Context: The Delhi HC constituted a five-member ‘National Rare Diseases Committee’ to implement the National Rare Disease Policy-2021 efficiently and to “ensure that the benefits of the policy reach the ultimate patients of rare disease”.
Rare Diseases
- A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.
- There are 6,000-8,000 classified rare diseases, but less than 5% have therapies available to treat them.
- Examples: Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia
- About 95% of rare diseases have no approved treatment and less than 1 in 10 patients receive disease-specific treatment.