Current and future challenges in clinical research of rare diseases What are rare diseases? As implied in its name, rare diseases affect small percentages of patients. However, when factoring in the multitude of rare diseases, the overall number of people affected by them is a staggering 475 million worldwide. According to the National Institutes of Health, about 25-30 million Americans have over 6,800 rare diseases, which amounts to 1 in 10 people. Over 50% of individuals with rare disease…