Rare diseases impact 1 in 10 Americans, and over 400 million people worldwide. The current development and FDA approval process can cost hundreds of millions of dollars – and take an average of 15 years – to develop one therapy. For the 30% of children diagnosed with a rare disease, who won’t live to see their 5th birthday, this wait is unthinkable. Meanwhile, prolonged – and unnecessary – diagnostic delays and inadequate treatment protocols yield increased healthcare costs and irreversible disease progression for individuals living with rare diseases. Even when they are approved, many transformational therapies never each patients because of the current methods we use to assess their value.
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