The Butterfly collaboration

Researchers and scientists of patient advocacy organizations report identifying approximately 10,000 rare diseases with 80% of these rare medically conditions involving genetics. An estimated 400 to 700 million people worldwide are believed to have at least one rare disease with approximately 25-30 million people living with a rare condition in the United States and 30 million people living with a rare condition in the European Union. 

Rare diseases disproportionately affect the pediatric community due to 71 percent of rare diseases impacting people before their 18th birthday. Children born with rare diseases experience a shortened projected lifespan and significant, often life threatening, gaps in healthcare.

Even though only 10% of rare diseases have an FDA-approved drug therapy treatment, 90% of pediatric patients with rare diseases in the U.S. survive into adulthood. This is in large part due to medical advancements and the Orphan Drug Act of 1983 that incentivizes research and development of treatments and cures for rare diseases. As progress continues to be made to extend the life expectancy of children affected by rare diseases, it is vital that the existing gaps in healthcare transitions from pediatric to adult care be addressed for the overall well-being and survivability of the aging rare disease community. 

The Butterfly Collaboration, a rare disease-focused campaign developed in partnership with Curant Health, has identified some of the more life-threatening issues rare disease patients experience during the period in healthcare when pediatric patients are forced to transition into adult care in its first publication titled: