Cancer is a heterogeneous disease with no single treatment that is successful across all cancer types. Identification of specific genetic changes in some cancer types has been crucial in improving therapy outcomes, as it has allowed individualized targeted therapy, broadly called ‘precision medicine’. Together with the other characteristics of a tumour, like location, volume, or spread, genetic factors dictate feasible therapeutic options, which range from surgery, radiotherapy, chemotherapy, to novel targeted agents and immunotherapy. With treatment resistance being a wide-spread issue, the most promising solution seems to be the use of combination therapy with different drugs or even treatment modalities.
However, before a therapeutic approach may reach patients, it needs to go through a rigorous approval process based on the assessment of safety and side-effects. Randomized controlled trials offer the highest level of evidence thanks to the high-quality study design and assigning patients randomly to treatment arms to minimise the bias.
A large number of ongoing clinical trials in various oncology settings will likely change the landscape of cancer therapy even more. The studies of the highest impact, called ‘landmark trials’, significantly change the current clinical practice, challenge the current knowledge or provide the ‘missing link’ evidence that complete our understanding.
This special article collection in BMC Medicine reflects the diversity seen in cancer therapy landscape and includes carefully selected articles from invited experts on oncology landmark trials that had the most substantial influence on the clinical practice or provided some critical evidence that enhanced our understanding in this field. It also features current topics in the trial design.