Pediatric-onset rare disease therapy pipeline yields hope for some and gaps for many: 10-year projection of approvals, treated patients, and list price revenues

The projected approvals over the next decade will undoubtedly be transformational for the patient communities impacted, many of whom have no currently approved treatments. However, the number of newly identified rare diseases is likely to outpace the rate of new therapies to treat them. Resources are needed to accelerate progress as 95% of pediatric-onset rare diseases are projected to still have no approved treatments in the next decade, and even for the 5% that have some options, more is needed.