By Rupa Doshi and Sameena Sharif
Rare cancers account for 27 percent of all new cancer diagnoses in the U.S. and 22 percent of all new cancer diagnoses in the EU. With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment options, researchers, clinicians, and patients may be seeking approaches to accelerate the development and approval of novel therapies. In this blog post, we review the regulatory programs available to expedite treatments for rare disorders and serious diseases.