Despite the fact that rare cancers have not been as appealing for their commercial potential as higher-incident cancers such as breast, colorectal, lung, and prostate cancer, the pressing unmet need among many rare cancers attracts drug developers. Many rare cancers offer developers an opportunity to become dominant players in small markets. Dramatic advancements in the treatment of rare cancers such as chronic myeloid leukemia (CML) and multiple myeloma represent examples of how developing therapies for these niche indications can lead to products with blockbuster sales. With regulatory mechanisms in place in the United States to incentivize pharmaceutical and biotechnology companies to pursue drug development for rare cancers, developers are tapping into the commercial potential of these niche indications.
Markets covered: United States.
Epidemiology: U.S. diagnosed incident cases for 18 rare cancers, including 26 subpopulations for 4 rare cancers.
Primary research: Interviewed experts:
- Three medical directors, U.S. health plans
- Tables: Therapies in the pipeline that will address the following unmet needs: the treatment of advanced/metastatic disease, therapies for specific patient subtypes, and better treatment options for elderly patients.
- Figures: Incidence of rare cancers by age group; U.S. sales of select monoclonal antibodies for rare cancers; U.S. sales of branded therapies prescribed for CML; U.S. sales of Celgene products used in the treatment of multiple myeloma; U.S. sales of Roche/Genentech products for NHL.