PRP Survival Guide

White unicorn

What constitutes a rare disease?

While not as unique as a unicorn, rare and genetic diseases are uncommon to most doctors and, as a whole, represent a large medical challenge. Combine this with the lack of financial or market incentives to treat or cure rare diseases, and you have a serious public health problem.

Here are a few statistics and facts to illustrate the breadth of the rare disease problem worldwide.

(1)   There are approximately 7,000 different types of rare diseases and disorders, with more being discovered each day.

(2)   There are 597 rare skin diseases listed in the Genetic and Rare Diseases Information Center database,

(3)   30 million people in the United States are living with rare diseases. This equates to 1 in 10 Americans or 10% of the U.S. population.

(4)   It is estimated that 350 million people worldwide suffer from rare diseases

(5)   If all of the people with rare diseases lived in one country,  it would be the world’s third most populous country

(6)   In the United States, a condition is considered “rare” if it affects fewer than 200,000 persons combined in a particular rare disease group. International definitions on rare diseases vary. For example, in the UK, a disease is considered rare if it affects fewer than 50,000 citizens per disease

(7)   Approximately 50% of the people affected by rare diseases are children.

(8)   80% of rare diseases are genetic in origin, and thus are present throughout a person’s life, even if symptoms do not immediately appear

(9)   Approximately 50% of rare diseases do not have a disease specific foundation supporting or researching their rare disease. The PRP community is fortunate to have researchers at Thomas Jefferson University, Yale University School of Medicine, an ad hoc coalition in Southern California (UCLA, USC and Kaiser Permanente) and Oregon Health and Science University looking at PRP.

(10)   30% of children with rare diseases will not live to see their 5th birthday

(11)    Rare diseases are responsible for 35% of deaths in the first year of life

(12)    The prevalence distribution of rare diseases is skewed – 80% of all rare disease patients are affected by approximately 350 rare diseases. PRP is lumped into the remaining 6,650 rare diseases.

(13)    According to the EveryLife Foundation, 95% of rare diseases have not one single FDA approved drug treatment

(14)    During the first 25 years of the Orphan Drug Act (passed in 1983), only 326 new drugs were approved by the FDA and brought to market for all rare disease patients combined.