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 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.

Rare cancers are generally classified among the larger group of rare diseases, defined in the U.S. as diseases that affect fewer than 200,000 people and in the European Union (EU) as diseases that affect less than one in 2,000. Given that prevalence is affected by mortality, the Surveillance of Rare Cancers in Europe (RARECARE) project has proposed a definition for rare cancers as those that have an incidence of less than 6 per 100,000 in the population. In the U.S., the de facto threshold for a rare cancer based on the literature is one with an incidence of less than 15 per 100,000 in the population. Using incidence rather than prevalence as a definition helps minimize the risk of under-representing the true impact and burden of rare cancers with high mortality rates.

Epidemiology

Approximately 186 cancer types are considered rare. Despite the relatively low incidence of each individual type, rare cancers as a whole account for 27 percent of all new cancer diagnoses in the U.S., and 22 percent of all new cancer diagnoses in the EU. They are also responsible for 20-30 percent of cancer deaths.

When looking at cancer incidence rates by age, the incidence of rare cancers significantly outpaces that of non-rare cancers in the pediatric population. And, survival rates for rare cancers are lower than those for non-rare cancers – 47 percent versus 65 percent – making rare cancers a significant burden and a public health priority.

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