Volume 14 Issue 3, March 2017

In 2016, FDA Oncology approved five new molecular entities and 17 efficacy supplements, including six accelerated approvals, 17 priority reviews, and 11 approvals of breakthrough-designated therapies. The FDA also approved five companion diagnostics, including a liquid biopsy test. One new anti-PD-L1 antibody was approved, along with six supplementary approvals of anti-PD-1/PD-L1 antibodies.

In 2016, four new anticancer drugs were approved by the FDA, and a further 12 existing agents were approved for 14 additional indications. Each one of these drugs is associated with important clinical benefits, but at an average monthly cost of ∼US$9,000. Here, I discuss the cost–benefit considerations related to these treatments and contemplate future economic prospects.

Patients with cancer expect to derive a meaningful clinical benefit from anticancer treatments, especially considering that such therapies are associated with adverse events and, often, substantial financial costs. We have evaluated new anticancer agents approved by the FDA in 2015 and 2016 using the ESMO Magnitude of Clinical Benefit Scale and ASCO Value Framework, and conclude that many agents only offer marginal value.

Tyrosine kinase inhibitors (TKIs) are the most common agents used for the treatment of patients with chronic myeloid leukaemia (CML). Several agents are available for frontline treatment, or after the development of resistance or intolerance to agents previously used. Herein, the authors evaluate differences between available TKIs, and discuss several end points that can be considered when selecting the first and subsequent lines of treatment of patients with CML.

To form metastases, cancer cells must leave the immunosuppressive tumour microenvironment and traffic, predominantly in the circulation, to new tissue sites, where they must then expand. During this process, the tumour cells are open to attack by the immune system. This Review highlights the possible mechanisms used by circulating tumour cells in the blood and disseminated tumour cells in other tissues to evade, escape, or subvert the immune system in order to survive and form metastatic lesions.

Imaging biomarkers (IBs) are used extensively in drug development and cancer research, but important differences exist between IBs and biospecimen-derived biomarkers. A tailored 'roadmap' is required for the development of new IBs to be used either in clinical research or for decision-making in healthcare. In this Consensus statement, a group of experts assembled by CRUK and the EORTC present 14 key recommendations for accelerating the clinical translation of IBs.

Assessment of tumour burden has become an integral part of most oncology clinical trials, and enables the evaluation of the activity and efficacy of new cancer therapeutics in solid tumours. Although RECIST was developed to assess treatment activity in early phase II trials with tumour response as the primary end point, it is now applied across the spectrum from early phase I trials through to confirmatory phase III trials. Several questions regarding the role of RECIST in the rapidly changing landscape of oncology therapeutics, and the challenges faced in its evaluation, are highlighted.