Although randomized trials provide the most reliable evidence of a drug's safety and efficacy, there are situations where randomized trials are not possible or ethical. In this article we discuss when and how single-arm trials can be used to support full approval of oncology drugs. These include situations in which an unprecedented effect on tumor response is observed in a setting of high unmet medical need, clinical trial patients have been well characterized, enabling a target population to be clearly defined, experience exists in a sufficient number of patients to allow adequate assessment of the risk:benefit relationship, and a proper historical context can be provided for analysis. We also discuss how response rates might be considered predictive of long-term outcomes or clinically meaningful in and of themselves in certain contexts.
Annals of Oncology, the journal of the European Society for Medical Oncology and the Japanese Society of Medical Oncology, provides rapid and efficient peer-review publications on innovative cancer treatments or translational work related to oncology and precision medicine.Main focuses of interest include: systemic anticancer therapy (with specific interest on molecular targeted agents and new immune therapies), randomized trials (including negatives ones), top-level guidelines, and new fields currently emerging as key components of personalized medicine, such as molecular pathology, bioinformatics, modern statistics, and biotechnologies. Radiotherapy, surgery and pediatrics manuscripts can be considered if they display a clear interaction with one of the fields above or are paradigm-shifting.With a large international editorial board of experts who are leaders in their fields, Annals of Oncology aims at delivering the best communication on the fast moving, and continually evolving, global oncology landscape.
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