As our understanding of the genetic and molecular basis of cancer advances, rare oncology research is accelerating at an unprecedented pace. In fact, in 2014, more than 40 percent of U.S. Food and Drug Administration’s orphan drug designations were for rare cancers.1 Moreover, the trend toward increasing international cooperation among big pharma, biotech, and academia is making rare oncology research more efficient than ever. In this white paper, we provide insight on the major issues being raised in the rare oncology space today, including patient perspectives on rare cancer research, innovative trial designs, the regulatory landscape, and pending legislation that may impact how studies are conducted.