In medical conditions, rare diseases present unique challenges to patients, caregivers, and medical professionals. These conditions, also known as orphan diseases, affect a small percentage of the population. While each rare disease is distinct, they collectively pose various treatment obstacles. This article delves into the world of rare diseases and orphan drugs, exploring the hurdles faced and the innovative approaches to overcome these challenges.
Understanding Rare Diseases and Orphan Drugs
Defining Rare Diseases
Rare diseases, by definition, afflict a limited number of individuals within a population. In the United States, a disease is classified as rare if it affects fewer than 200,000 people. This small patient pool can make research, diagnosis, and treatment particularly daunting.