Rare tumors, when considered as a group, represent a significant burden to society as they may account for up to 25% of the mortality by cancer in nations like the United States. In contrast with the current scenario in highly incident cancer types, little progress has been achieved in the treatment of most rare cancers. The reasons for this apparent stagnation are mostly intrinsic to logistical difficulties in performing large clinical trials in rare diseases and will be addressed further in this article. Because both cancer incidence and clinical research are booming in emerging nations, we also aim to address the current and future role of these countries in research and the drug development process in rare tumor types.
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