New research in Journal of Managed Care + Specialty Pharmacy (JMCP) provides insights into the future landscape of treatments for rare diseases that present in childhood, offering a balanced picture of exciting progress for some conditions and serious concerns about growing gaps for the vast majority of pediatric rare diseases.

Using innovative predictive modeling techniques, researchers from the Center for Biomedical System Design in the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center and the EveryLife Foundation for Rare Diseases (EveryLife Foundation) developed a 10-year projection of product approvals, patients treated, and list price revenues for pediatric-onset rare disease therapies through 2033.

Read the article on JMCP.org