Clinical Research: Phase 1 - Phase 4

Rare Oncology Research in the 21st Century


Sachin Kulkarni
Executive Director, Strategic Development, Oncology & General Medicine
Premier Research

Juliet M. Moritz
Executive Director, Strategic Development, Rare Diseases
Premier Research

Study of rare cancers tends to look more like rare disease research than standard oncology research. This webinar will highlight some of the challenges in designing and conducting trials for rare cancers and explore positions adopted by various authors and research consortia on how to best design and conduct these trials, including a review of pending legislation — the 21st Century Cures Act — and how it might affect rare oncology research if passed.

Considerations include recruiting patients with rare cancers, maximizing the value of data points in clinical trials involving small populations, potential use of adaptive design in these trials, and the appetite for risk among researchers and patients.

Typically in oncology trials, and similarly in rare disease trials, the investigational product is tested only on affected subjects rather than on healthy volunteers. Testing new drugs on this vulnerable population requires consideration of the study design so the fewest possible subjects are exposed to the investigational product, and so the study yields information that supports viable research decisions.

As the cost of clinical trials rises along with the cost of bringing new drugs to market, research should include alternative study design approaches and emphasize early indicators such as biomarkers, natural history data, and preclinical data. This approach also reduces the patient burden and may accelerate drug development timelines.