Fierce Pharma September 25, 2023
Since rare disease research gained traction in the 1980s, pharmaceutical investment in orphan drugs has accelerated. Orphan drugs have represented 50% or more of new molecular entities approved by the FDA in recent years, and orphan drug sales will constitute nearly one third of the global drug pipeline’s value by 2026, according to a 2022 report by Evaluate Ltd. We are continuously learning about the 7,500+ conditions that affect nearly 400 million people worldwide, 95% of which have no FDA-approved treatments and about 80% of which are genetic.
Although 70% of rare diseases start in childhood, the standard newborn screening process only tests for a select number of them. Without specific testing, many serious rare diseases...
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