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.

Abstract

More than 10,000 rare diseases affect more than 30 million Americans, nearly 70% of which manifest in childhood. The drug development pipeline boasts hundreds of candidates for pediatric-onset rare disease, but little is known about the impact of potential approvals. To quantify US projected product approvals, patients treated, and product revenues for pediatric-onset rare disease treatments through 2033. Four-stage model consisting of a Markov Chain Monte Carlo simulation of US Food and Drug Administration approvals, calculation of eligible patients per clinical trial criteria, and projection of adoption and list price revenues, all using publicly available data. By 2033 the pipeline will yield approximately 45 new product approvals, a 14% growth in annual treated patients, and an incremental $10.7B in list price drug revenues ($28.2B: 2023; $38.9B: 2033) prior to any health care cost offsets, caregiving impacts, long-term social benefits, or other benefits from treating the additional patients. 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.