News & Views

A patient's right to obtain medication that will be ineffective against his/her terminal illness and will cause his/her death should also encompass the right of that patient to be prescribed medication that might be ineffective and might cause his/her death. Indeed, the 'right to die' should embody the 'right to try' experimental agents for the treatment of cancer.

Escalating costs of cancer treatments are threatening access to high-quality care. This economic trend comes at a time of unprecedented opportunity for translational research, but an alarmingly low level of patient participation in clinical trials. Patients' concerns about costs are a barrier to trial enrolment, and addressing these concerns is a moral and practical imperative if we are to accelerate progress against cancer.

The rapidly rising costs of cancer care, driven in particular by the high prices of new drugs, are increasingly challenging health-care systems across the world. To ensure accessibility to novel antitumour drugs, novel paradigms are needed at several levels, not only economically, but also in terms of research and new study designs.

Imatinib has revolutionized the treatment of chronic myeloid leukaemia (CML). In 2016, generic imatinib will be introduced into the US market. We analyse the potential impact of this new product on patient care and optimal CML therapy, and comment on the effect that distorted cancer drug pricing in the USA will have on treatment for patients with limited therapeutic options.

A study reveals that, from 2009–2014, a considerable percentage of public speakers at Oncologic Drugs Advisory Committee meetings had disclosed or undisclosed financial associations with the drug company seeking product approval. Amid calls for increased public engagement in health care and health-care research, steps must be taken to minimize the conflicts of interests of those who claim to speak for patients and the public.

Apatinib significantly improves both the progression-free survival (PFS) and overall survival in patients with advanced-stage gastric cancer who are refractory to two or more lines of chemotherapy. In the context of previous phase III trials of angiogenesis inhibitors for this disease, we discuss the role of apatinib, and the advantages and limitations of VEGFR-2 blockade in the advanced disease setting.

Accurate and efficient expedited investigational new drug (IND) reporting is a crucial component of clinical research. The FDA, pharmaceutical companies, institutional review boards, and clinical investigators should develop dynamic standardized electronic forms with preferred, predetermined terms to harmonize their practices and to help optimize the quality of clinical research and maximize patient safety.

The recent FDA approval of MM-398 as a second-line treatment of metastatic pancreatic cancer, based on a 1.9-month overall survival benefit observed in the NAPOLI-1 trial, adds a new therapeutic option for this notoriously difficult-to-treat disease; however, by discouraging clinical trial enrolment, this approval might have negative consequences for the development of novel agents, which remain an essential unmet need.

A survey on the official prices of 31 cancer drugs across 18 countries, published by Vogler and colleagues, has revealed substantial differences in price for the same drug in different countries. Herein, we discuss inequalities in the access to cancer care and raise some challenging questions.

Prostate-specific antigen (PSA)-based screening approaches are associated with reduced prostate-cancer mortality, but can lead to overdiagnosis, unnecessary biopsies and overtreatment. Two solutions for this problem exist: to abandon PSA-based screening completely, or to improve the accuracy of PSA-based screening methods to solve the benefit-to-harm equation. Herein we explore these solutions by examining three recent publications.

The recently updated breast cancer screening guidelines from the American Cancer Society are less aggressive than previous versions and clearer about overdiagnosis. However, a lack of attention was placed on the differences in effect estimates between trials at high and low risk of bias, and the authors failed to quantify the most serious harm.

Henderson and colleagues previously highlighted the need for more-rigorous standards of preclinical experimental design and reporting metrics. They now build on their earlier work with a meta-analysis of preclinical experiments that examined the efficacy of sunitinib. Their results demonstrate how suboptimal preclinical study designs can prompt unwarranted clinical expectations.

Surgeons should promote the best standard of surgical care through evidence-based results from prospective trials. European surgical investigators, in this issue of the journal, highlight the difficulties in patient accrual to surgical trials, patient and physician biases, and referral and quality assurance hurdles. We expand on these points and suggest some solutions.

The results of three recent studies demand that more attention be placed on defining the most-appropriate approach to population-based breast-cancer screening, in particular regarding the potential harms of increasing overdiagnosis. Two of these studies report that more-sensitive detection of breast neoplasms is possible by 3D tomography and by MRI, but the third paper raises the question of whether this increased sensitivity is desirable.

A study assessing the impact of the 21-gene recurrence score assay in routine clinical practice on the use of adjuvant chemotherapy in women with early stage ER-positive breast cancers showed that adjuvant chemotherapy use decreased in high-risk patients, but increased in low-risk patients. I discuss these results and highlight how this reflects more-selective administration of chemotherapy.

In the RADIANT study, no difference in disease-free survival was observed for patients with non-small-cell lung cancer (NSCLC) treated with erlotinib versus placebo in the adjuvant setting. Further biomarker studies are awaited to determine whether patients with NSCLC can benefit from adjuvant therapy with tyrosine kinase inhibitors.

In two recent phase III trials, investigators evaluated the addition of docetaxel to androgen-deprivation therapy for non-castrate prostate cancer. On the basis of the CHAARTED-trial findings, we can firmly conclude that this combination can be used in the metastatic setting. The results of the GETUG 12 trial are less informative, although some benefit for patients with high-risk localized prostate cancer was demonstrated.

A recent objective study has demonstrated that the use of adjuvant platinum-based intraperitoneal chemotherapy in patients with small-volume residual advanced-stage ovarian cancer remains limited, despite the publication of several phase III trials demonstrating superior overall survival associated with this approach. Several factors might explain this far less than satisfactory state of affairs.

Dose-expansion cohorts (DECs) enable investigators to identify potentially effective drugs, for specific patient populations, in a single trial by assessing antitumour activity as early as possible. We discuss how the objectives, design and interpretation of DEC have evolved, and how DECs are changing the landscape of early drug development.

Treatment with pembrolizumab, an anti-PD-1 antibody, improved progression-free survival compared with investigator-choice chemotherapy in a phase II trial in patients with advanced-stage melanoma previously treated with ipilimumab. Two subsequent independent trials have confirmed that anti-PD-1 therapy is a better option than either chemotherapy or ipilimumab in the frontline setting.