Comment

Scientific Advice meetings are a mechanism to improve communications between drug developers and regulators during the drug-development process. While standard practice for industry, the benefits provided by these meetings are under-utilised by academia. In the context of drug repurposing, can scientific advice, as part of a proposed new R&D tax credits scheme, help to unblock some of the obstacles in the way to clinical adoption?

In studies investigating the combination of two or more anticancer drugs that are already approved for independent use, or 'maintenance' regimens, the use of progression-free survival as the end point for approval is inadequate; sequential treatment with the same agents or existing salvage therapies, respectively, might provide an equivalent survival benefit, with lower toxicity, cost, and treatment burden, therefore, the use of an overall survival end point is essential to justify such interventions.

Patients with resectable solid tumours can harbour minimal residual disease (MRD) after initial treatment, which is a potential source for subsequent metastatic relapse. The interaction between disseminated tumour cells (DTCs) and the new microenvironment in which they reside determines whether DTCs remain dormant or progress into overt metastases. We highlight the promise of liquid biopsies to inform on MRD.

Does a patient with advanced incurable disease have a right not to hear the bad news? We think not. Failing to disclose a poor prognosis undermines patient autonomy and increases the likelihood of poor end-of-life care.

Over the past decade, many anticancer drugs have been approved for use only in combination regimens and only in palliative settings, despite having negligible single-agent activity in the same disease. We examine whether these agents provide any tangible clinical benefits and are worthy of continued development, or whether R&D efforts would be better focused elsewhere.

The 69 National Cancer Institute-designated Cancer Centers are premier academic institutions that place significant value on research integrity and an ethic that rigorous evidence should guide patient care and define expectations. Recent patient-focused advertising has strayed from these values, obscuring valid reasons for seeking care at these centres.

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.