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.
About rare cancers
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 2 and in the European Union (EU) as diseases that affect less than one in 2,000.3 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.4 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.5 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.
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
…and much more!