Time to deterioration of symptoms or function using patient-reported outcomes in cancer trials.

Time-to-event endpoints for patient-reported outcomes, such as time to deterioration of symptoms or function, are frequently used in cancer clinical trials. Although time-to-deterioration endpoints might seem familiar to cancer researchers for being similar to survival or disease-progression endpoints, there are unique considerations associated with their use. The complexity of time-to-deterioration endpoints should be weighed against the information that they add to the tumour, survival, and safety data used to inform the risks and benefits of an investigational drug.

Association of Patient-Reported Physician Awareness of Complementary Medicine, Medical Care Experience and Care Quality.

The aim was to examine the association of patient-reported physician awareness of biological CAM use and patient perceptions of care experience and quality with a population-based study of patients with incident lung and colorectal cancer. This was a secondary data analysis using regression models. Outcomes of interest were patient reports of medical care experience and quality ratings.

The estimand framework and its application in substance use disorder clinical trials: a case study.

Relapse rates among individuals with substance use disorder (SUD) remain high and new treatment approaches are needed, which require evaluation in randomized controlled trials (RCTs). Measurement and interpretation challenges for SUD RCT data are often ignored or presented only in statistical analysis plans. Since different analytic approaches may result in different estimates and thus interpretations of the treatment effect, it is important to present this clearly throughout the trial.

The use of proxies and proxy-reported measures: a report of the international society for quality of life research (ISOQOL) proxy task force.

Aims: Proxy reports are often used when patients are unable to self-report. It is unclear how proxy measures are currently in use in adult health care and research settings. We aimed to describe how proxy reports are used in these settings, including the use of measures developed specifically for proxy reporting in adult health populations.

Does Knowledge of Treatment Assignment Affect Patient Report of Symptoms, Function, and Health Status? An Evaluation Using Multiple Myeloma Trials.

Objectives: Unblinded trials are common in oncology, but patient knowledge of treatment assignment may bias response to questionnaires. We sought to ascertain the extent of possible bias arising from patient knowledge of treatment assignment.

Methods: This is a retrospective analysis of data from 2 randomized trials in multiple myeloma, 1 double-blind and 1 open label.

Patient and proxy reports regarding the experience of treatment decision-making in cancer care.

Objective: Shared decision-making, including the elicitation of patient preferences regarding treatment decisions, is considered part of high-quality cancer care. However, patients may not be able to self-report due to illness, and therefore proxy reports may be used. We sought to determine the difference between proxy and patient reports about patient decisions and preferences among patients who received or were scheduled for chemotherapy using data from a large, population-based survey of patients with incident lung or colorectal cancer.

Exploration of baseline patient-reported side effect bother from cancer therapy.

Background: Patient reports of expected treatment side effects are increasingly collected as part of the assessment of patient experience in clinical trials. A global side effect item that is patient-reported has the potential to inform overall tolerability. Therefore, the aim of this study was to examine the completion and distribution of such a global single-item measure of side effect burden in five cancer clinical trials.

US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January, 2008, and December, 2017.

With the advent of patient-focused drug development, the US Food and Drug Administration (FDA) has redoubled its efforts to review patient-reported outcome (PRO) data in cancer trials submitted as part of a drug's marketing application. This Review aims to characterise the statistical analysis of PRO data from pivotal lung cancer trials submitted to support FDA drug approval between January, 2008, and December, 2017. For each trial and PRO instrument identified, we evaluated prespecified PRO concepts, statistical analysis, missing data and sensitivity analysis, instrument completion, and clinical relevance.

Global Variation in Opioid Use in Prostate Cancer Trials.

Importance: Regional variation in opioid use may be attenuated when pharmaceutical-sponsored trials include care that is often standardized by protocols. Understanding such variation is important for global trials that sometimes include time to opioid use as an end point.

Objective: To identify whether regional and country-level variation in opioid use exists among prostate cancer clinical trials across the world.

Analyzing patient-reported outcome data when completion differs between arms in open-label trials: an application of principal stratification.

Purpose: Cancer trials are often open-label and include patient-reported outcomes (PROs). Previous work has demonstrated that patients may complete PRO assessments less frequently in the control arm compared with the experimental arm in open-label trials. Such differential completion may affect PRO results.

Patient reported outcomes in anti-PD-1/PD-L1 inhibitor immunotherapy registration trials: FDA analysis of data submitted and future directions.

Background: Patient-reported outcome measures can be used to capture the patient's experience with disease and treatment. Immunotherapy agents including the anti-programmed death receptor-1/programmed death-ligand-1 inhibitor therapies have unique symptomatic side effects and patient-reported outcome data can help to characterize the benefits and burdens associated with therapy.

Methods: We reviewed registration trials in the Food and Drug Administration database for five anti-programmed death receptor-1/programmed death-ligand-1 inhibitor therapies to characterize trial design and patient-reported outcome assessment strategy (cutoff 31 December 2017).

Blinding and Patient-Reported Outcome Completion Rates in US Food and Drug Administration Cancer Trial Submissions, 2007-2017.

Background: Patient-reported outcomes (PROs) are commonly included in submissions to the United States Food and Drug Administration (FDA). Open-label designs are frequent in cancer trials. Between-arm differences in PRO missingness may affect results.

The Association of Proxy Care Engagement with Proxy Reports of Patient Experience and Quality of Life.

Objective: To assess the association of proxy-specific covariates with proxy-reported patient cancer care experience, quality rating, and quality of life.

Data Sources/study Setting: Secondary analysis of data from the Cancer Care Outcomes Research and Surveillance (CanCORS) study.

Study Design: Cross-sectional observational study.

Proxy and patient reports of health-related quality of life in a national cancer survey.

Background: Proxy respondents are frequently used in surveys, including those assessing health-related quality of life (HRQOL). In cancer, most research involving proxies has been undertaken with paired proxy-patient populations, where proxy responses are compared to patient responses for the same individual. In these populations, proxy-patient differences are small and suggest proxy underestimation of patient HRQOL.

The Effect of a "Class Smoke Free Pledge" on Breath Carbon Monoxide in Arabic Male Adolescents.

Introduction: Arabic male adolescents have a high smoking prevalence. Introduction of "Class smoke-free" pledges have been successful amongst European adolescents but have not been evaluated using objective valid measures. We tested the impact of adding a smoke free pledge strategy to a proven peer-led asthma and smoking prevention program on breath carbon monoxide level (BCO) in male high-school students in Jordan.

Systematic review of caregiver responses for patient health-related quality of life in adult cancer care.

Purpose: In surveys and in research, proxies such as family members may be used to assess patient health-related quality of life. The aim of this research is to help cancer researchers select a validated health-related quality of life tool if they anticipate using proxy-reported data.

Methods: Systematic review and methodological appraisal of studies examining the concordance of paired adult cancer patient and proxy responses for multidimensional, validated HRQOL tools.

Differences between Proxy and Patient Assessments of Cancer Care Experiences and Quality Ratings.

Objective: To assess the impact of proxy survey responses on cancer care experience reports and quality ratings.

Data Sources/study Setting: Secondary analysis of data from Cancer Care Outcomes Research and Surveillance (CanCORS). Recruitment occurred from 2003 to 2005.

Does Resilience Mediate Carer Distress After Head and Neck Cancer?

Background: Caring for patients with head and neck cancer (HNC) can have significant negative psychological and practical impact; however, some carers seem able to cope effectively. Little research has investigated this resilience among carers.

Objective: The objective of this study was to investigate the resilience levels among carers of patients with HNC.

Nurse-led supportive care management: a 6-month review of the role of a nurse practitioner in a chemotherapy unit.

Objectives: This case study evaluates the oncology nurse practitioner (NP) role in a chemotherapy unit.

Background: The NP works in the cancer centre of a major metropolitan public hospital. The NP role was established in the chemotherapy unit in 2007.

The effect of cancer stage and treatment modality on quality of life in oropharyngeal cancer.

Objectives/hypothesis: To examine changes in health-related quality of life among oropharyngeal cancer patients by stages and across treatment types among advanced cancer patients.

Study Design: Individual prospective cohort study.

Methods: All newly diagnosed patients with oropharyngeal cancer treated with curative intent were routinely assessed.

Advancing the educational and career pathway for clinical trials nurses.

Clinical trials nurses play a pivotal role in the conduct of clinical research, but the educational and career pathway for these nurses remains unclear. This article reports findings from a survey of nurses working in cancer clinical trials research in Australia. Most participants held postgraduate qualifications (42 of 61); however, clinical trials education was primarily attained through short professional development courses.

Recruiting and retaining general practitioners to a primary care asthma-intervention study in Australia.

The need for more evidence-based interventions in primary care is clear. However, it is challenging to recruit general practitioners (GPs) for interventional research. This paper reports on the evaluation of three methods of recruitment that were sequentially used to recruit GPs for a randomised controlled trial of an asthma communication and education intervention in Australia.

The information needs of patients with head and neck cancer and their caregivers: a short report of instrument development and testing.

Purpose: The study aimed to assess the reliability and scaling assumptions of an information needs questionnaire for patients with head and neck cancer (HNC) and their caregivers.

Background: Patients with HNC have specific information needs but no tools exist that can simultaneously assess the needs among these patients and their caregivers.

Methods: The Head and Neck Information Needs Questionnaire (HaNiQ) was adapted from a validated information needs questionnaire for breast cancer patients.

An exploratory analysis of active and low energy behaviour in Australian adolescents.

Adolescents engage in a complex range of low energy-consuming and active behaviours not easily captured by single behavioural measures. This study aims to describe the associations among adolescent active and low energy behaviours. Data were obtained from adolescents aged 12-16 years (n=1532) participating in a nationally representative survey.