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Research & Reports

Searching in Biopharma (1575)

Tapping the Commercial Potential of Rare Cancers

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.

Questions Answered in This Report:

  • Although numerous development challenges have stifled innovation for many rare cancers, the high level of unmet need in these indications offers developers the promise of being able to meaningfully shape the future course of treatment while tapping into this market opportunity. What are the key unmet needs for rare cancers? How important are these unmet needs? What therapies are in development that could address these unmet needs?

  • Developers may be discouraged by the numerous hurdles associated with development of rare cancer therapies, thus leading to sluggish progress for many niche indications. What are the development challenges unique to rare cancers? Are there reimbursement hurdles that may impact market access?

  • Although many developers perceive the low number of patients with rare cancers to be a barrier to achieving commercial success, several indications represent areas where sales potential has been realized. Numerous strategies exist for succeeding in the treatment of rare cancers. What are the possible strategies for tapping into rare cancer markets? What are some examples of rare cancer therapies that have attained commercial success, and what were their strategies?


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

Additional content:

- 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.