Epidemiology Studies on Rare Childhood Cancers

A major global pharmaceutical manufacturer asked Analysis Group to document the incidence and prevalence of several extremely rare childhood cancers as part of its application to a key regulatory agency to obtain an exemption from conducting clinical trials in children.

Analysis Group, including Senior Advisor Tammy Sisitsky and Managing Principal Mei Sheng Duh, conducted an extensive literature review on each of the rare diseases and used key findings to extrapolate disease incidence and prevalence in children in both the US and EU populations. However, because many of the findings were sufficient to obtain only broad upper bounds, we also conducted an analysis of US SEER cancer registry data to obtain more precise estimates of disease incidence and prevalence in children, and extrapolated these findings to the EU.

Analysis Group combined the findings from the literature and the cancer registries and, for each disease, provided estimates of the number of new pediatric cases per year as well as an estimate of the number of new and existing cases among children. Our research confirmed that all of the childhood cancers studied were extremely rare.