From the Laboratory to Bedside: Expediting Development of Novel Cancer Treatment

Study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Current strategies and processes for general oncology drug development don’t always apply to rare oncology, a field that today accounts for up to 20 percent of new cancer diagnoses. To optimize drug development, study designs should maximize the percentage of patients on effective treatment and minimize overall sample size to limit patient exposure to drugs or doses that have no effect. This can be achieved through use of adaptive design techniques and more rigorous oversight of patient eligibility to define the optimal trial population. Researchers should consider alternative statistical principles — for example, estimating survival when further lines of therapy are anticipated — along with interim analyses and proper futility management. Biomarkers and surrogate endpoints of efficacy, such as response duration and progression-free survival, also need special consideration and may be required as part of the regulatory review process. Collecting information beyond survival is becoming more important, with payers increasingly requiring quality survival be demonstrated. Development plans should encompass expanded access to bridge the gap between clinical trials and market approval.