Clinical trial design for rare cancers: Why a less conventional route may be required

Abstract

Major recent advances in the diagnosis, treatment, prevention and screening of cancer have led to improvements in survival and other outcomes for cancer patients. When the 198 identified rare cancers are taken in aggregate, rare cancers account for 22% of all cancer diagnoses, higher than any single common cancer. Yet median survival for patients with rare cancer patients remains poor as clinical trial and treatment advancements trail those achieved for common cancer. Overcoming the challenges inherent in the study of rare diseases is critical to achieve improvements in outcomes for patients. Combining a redesign of clinical trial protocols with the new paradigm for the treatment of all cancers, may lead to improvements in survival benefits for rare cancers in line with those achieved in the treatment of more common types.