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Clinical pharmacologist Harry Shirkey noted more than 50 years ago that children are often “therapeutic orphans” in biomedical research. Today, we see a continued five- to 10-year lag of pediatric biomedical advances behind the adult populations.
In pediatric healthcare, approximately 90% of healthcare resources are utilized by 5 to 10% of children who suffer complex and chronic medical conditions. In many cases, these 5,000 pediatric-only rare conditions, or “orphan diseases,” lack a market large enough to drive intervention, which permeates both the pharmaceutical and biomedical device industries.