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.1 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...
Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy. Adaptive...
Rare cancers account for almost a quarter of all new cancers worldwide though there is no universally adopted definition for rare cancers. In the U.S., rare cancers are defined as those with fewer than 15 cases per 100,000 per year, whereas in the EU, they are defined as six cases per 100,000 per year. Historically,...
The study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Careful consideration of clinical trial design and regulatory pathways can help increase the likelihood of success in rare oncology clinical trials. Researchers studying rare cancers must call on expertise in both...
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. Moreover, the trend toward increasing international cooperation among big pharma, biotech, and academia...
A variety of factors can make it difficult to conduct traditional full-scale clinical trials for new treatments of rare cancers. Consequently, because so little information is available, treatments for rare oncology patients are inadequate or nonexistent. Luckily for both patients and researchers, the FDA and the European Medicines Agency (EMA) have introduced regulations that expedite review and approval of certain investigative drugs. Expedited FDA...
A rare cancer is often defined as one with an incidence of less than 15 per 100,000. The patient populations of these conditions are so small that traditional clinical trial design strategies can be unfeasible. But, with such a low incidence, just why is rare oncology research so important? Why Rare Oncology Needs Innovation Keep in mind...