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Patient-reported outcomes (PROs) are used in clinical trials to provide evidence of the benefits and risks of interventions from a patient perspective and to inform regulatory decisions and health policy. The collection of PROs in routine practice can facilitate monitoring of patient symptoms; identification of unmet needs; prioritisation and/or tailoring of treatment to the needs of individual patients and inform value-based healthcare initiatives. However, respondent burden needs to be carefully considered and addressed to avoid high rates of missing data and poor reporting of PRO results, which may lead to poor quality data for regulatory decision making and/or clinical care. The collection of patient-reported outcomes (PROs) may capture patients’ assessments of their health status. Here authors highlight PRO-specific issues that should be considered to minimise respondent burden in clinical trials and routine care.

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. Here we use the estimand framework to show how analytical decisions answer different clinical questions of interest, some of which might be uninformative. Challenges including the consideration of intercurrent events, the difficulty in maintaining adequate completion rates, and considerable patient and trial burden from long-term, serial, patient-reported outcome measurements render time to deterioration a problematic approach for widespread use. For trials in which a comparative benefit in symptoms or function is an objective, an analysis at pre-specified relevant timepoints could be a better approach.

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. Of the 37 pivotal lung cancer trials used to support FDA drug approval, 25 (68%) trials included PRO measures. The most common prespecified PRO concepts were cough, dyspnoea, and chest pain. At the trial level, the most common statistical analyses were descriptive (24 trials [96%]), followed by time-to-event analyses (19 trials [76%]), longitudinal analyses (12 trials [48%]), and basic inferential tests or general linear models (10 trials [40%]). Our findings indicate a wide variation in the analytic techniques and data presentation methods used, with very few trials reporting clear PRO research objectives and sensitivity analyses for PRO results. Our work further supports the need for focused research objectives to justify and to guide the analytic strategy of PROs to facilitate the interpretation of patient experience.

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. We searched PubMed, CINAHL, PsycINFO and perused bibliographies of reviewed papers. We reviewed concordance assessment methods, results, and associated factors for each validated tool. Results A total of 32 papers reporting on 29 study populations were included. Most papers were cross-sectional (N=20) and used disease-specific tools (N=19), primarily the FACT and EORTC. Patient and proxy mean scores were similar on average for tools and scales, with most mean differences <10 points but large standard deviations. Average ICCs for the FACT and EORTC ranged from 0.35 to 0.62, depending on the scale. Few papers (N= 15) evaluated factors associated with concordance, and results and measurement approaches were inconsistent. The EORTC was the most commonly evaluated disease-specific tool (N=5 papers). For generic tools, both concordance and associated factor information was most commonly available for the COOP/WONCA (N= 3 papers). The MQOL was the most frequently evaluated end-of-life tool (N= 3 papers). Conclusions Proxy and patient scores are similar on average, but there is large, clinically important residual variability. The evidence base is strongest for the EORTC (disease-specific tools), COOP/WONCA (generic tools), and MQOL (end-of-life-specific tools).

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. In practice, however, proxy responses will only be used when patient responses are not available. The difference between proxy and patient reports of patient HRQOL where patients are not able to report for themselves in cancer is not known. The objective of this study was to evaluate the difference between patient and proxy reports of patient HRQOL in a large national cancer survey, and determine if this difference could be mitigated by adjusting for clinical and sociodemographic information about patients. Methods Data were from the Cancer Care Outcomes Research and Surveillance (CanCORS) study. Patients or their proxies were recruited within 3–6 months of diagnosis with lung or colorectal cancer. HRQOL was measured using the SF-12 mental and physical composite scales. Differences of ½ SD (=5 points) were considered clinically significant. The primary independent variable was proxy status. Linear regression models were used to adjust for patient sociodemographic and clinical covariates, including cancer stage, patient age and education, and patient co-morbidities. Results Of 6471 respondents, 1011 (16%) were proxies. Before adjustment, average proxy-reported scores were lower for both physical (−6.7 points, 95% CI -7.4 to −5.9) and mental (−6 points, 95% CI -6.7 to −5.2) health. Proxy-reported scores remained lower after adjustment (physical: −5.8 points, −6.6 to −5; mental: −5.8 points, −6.6 to 5). Proxy-patient score differences remained clinically and statistically significant, even after adjustment for sociodemographic and clinical variables. Conclusions Proxy-reported outcome scores for both physical and mental health were clinically and significantly lower than patient-reported scores for these outcomes. The size of the proxy-patient score differences was not affected by the health domain, and adjustment for sociodemographic and clinical variables had minimal impact.

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. Methods We systematically searched Medline, PsycINFO, PsycTESTS, CINAHL and EMBASE from database inception to February 2018. Search terms included a combination of terms for quality of life and health outcomes, proxy-reporters, and health condition terms. The data extracted included clinical context, the name of the proxy measure(s) used and other descriptive data. We determined whether the measures were developed specifically for proxy use or were existing measures adapted for proxy use. Results The database search identified 17,677 possible articles, from which 14,098 abstracts were reviewed. Of these, 11,763 were excluded and 2335 articles were reviewed in full, with 880 included for data extraction. The most common clinical settings were dementia (30%), geriatrics (15%) and cancer (13%). A majority of articles (51%) were paired studies with proxy and patient responses for the same person on the same measure. Most paired studies (77%) were concordance studies comparing patient and proxy responses on these measures. Discussion Most published research using proxies has focused on proxy-patient concordance. Relatively few measures used in research with proxies were specifically developed for proxy use. Future work is needed to examine the performance of measures specifically developed for proxies. Systematic review registration PROSPERO No. CRD42018103179

Background: Prostate cancer (PrCa) outcomes are inferior in regional and rural areas compared to metropolitan centres. We evaluated patterns of care in PrCa patients treated in public and private healthcare facilities in regional Tasmania. Methods: This retrospective study used clinicopathological data for 2180 PrCa patients diagnosed between 2015–2022. Descriptive statistics and regression analyses determined associations between treatment facility (public vs. private) and diagnostic and treatment factors. Results: A significantly greater proportion of public patients were from outer regional/remote areas (prevalence ratio (PR) = 1.25, 95% CI: 1.19–1.31), presented with higher-risk disease (PR = 1.56, 95% CI: 1.22–2.00) and underwent active treatment compared to private patients (PR = 1.07, 95% CI: 1.03–1.11). Men treated privately were most likely to have low-risk PrCa (p < 0.001) and be managed with active surveillance (AS, 52.9%). When stratified by disease risk, public patients with intermediate (p < 0.001) or very high-risk/metastatic disease (p = 0.003) were still significantly more likely to receive active treatment than private patients. Furthermore, except for very high-risk/metastatic patients, public patients took significantly longer to commence treatment, ranging between a mean difference of 40 to 59 days depending on risk category. Conclusions: In Tasmania, treatment pathways for PrCa patients differ significantly between public and private healthcare sectors and may contribute to poorer outcomes in regional and remote areas.

Understanding the different facilitators and barriers to screening within cultural and ethnic groups is important for developing appropriate education and outreach programs to underserved groups. Qualitative methods were employed to gain a rich understanding of participant views. In-depth interviews were conducted with 18 Chinese Australian women in their native languages and analysed using content analysis. Knowledge of cervical cancer was low, and few participants understood the benefits and purpose of screening. Having a doctor’s recommendation was a strong motivator, and returning for screening was encouraged by having a female Chinese doctor perform the exam, receiving a reminder letter and the absence of cost for screening participation. However, participation was inhibited by logistical barriers, cultural beliefs and previous painful screening experiences. A range of multifaceted facilitators and barriers must be considered when developing interventions to increase the rates of cervical screening in this population.

Abstract 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. Methods We enrolled male students from four high-schools in Irbid, Jordan. Schools were randomly assigned to receive either TAJ (Triple A in Jordan, n = 218) or TAJ-Plus (with added class smoke-free pledge, n = 215). We hypothesized that students receiving TAJ-Plus would have greater reduction in BCO levels than those only receiving the TAJ intervention. Asthma and smoking status were assessed by self-administered questionnaires. Smoking outcomes were collected using a BCO Monitor. Results Both groups had significant reductions in BCO levels post-intervention (p < .0001), however, decreases were greater in TAJ-Plus group (3.9 ± 0.2 vs. 4.8 ± 0.2, p < .0001). Intervention effects on BCO over time did not vary by smoking status (p = .085), asthma status (p = .602), or a combination of the two (p = .702). Conclusions An added smoke-free pledge strategy to a proven peer-led asthma education program appears to be a promising approach to motivate adolescents to abstain from smoking in Jordan. Future research is required to determine if these results can be extended to Jordanian adolescent females. Implications A commitment by students via a “class smoke-free” pledge can be an added incentive to motivate adolescents in Arabic-speaking countries to abstain from smoking. Social influence approaches in schools can be useful in countering the aggressive tobacco marketing campaigns targeting Jordanian and other Arabic-speaking youth. The combination of “class smoke-free” pledges and an evidence-based peer-led asthma and smoking education can be implemented in schools to influence adolescents with asthma to abstain from smoking.

Aims Many large-scale population-based surveys, research studies, and clinical care allow for inclusion of proxy reporting as a strategy to collect outcomes when patients are unavailable or unable to provide reliable self-report. Prior work identified an absence of methodological guidelines regarding proxy reporting in adult populations, including who can serve as a proxy, and considerations for data collection, analysis, and reporting. The primary objective of this work by the ISOQOL Proxy Task Force was to review documents and clinical outcome assessment measures with respect to proxy reporting and to develop, through consensus, considerations for proxy reporting. Methods We assembled an international group with clinically relevant and/or methodological expertise on proxy use in adult populations. We conducted a targeted review of documentation based on regulatory, non-regulatory, professional society, and individual measure sources. Using a standardized collection form, proxy-related information was extracted from each source including definitions of a proxy, characteristics of a proxy, domains addressable or addressed by a proxy, and observer-reporting. Results The definition of proxy was inconsistent across 39 sources, except regulatory documents which defined a proxy as a person other than the patient who reports on an outcome as if she/he were the patient. While proxy report was discouraged in regulatory documentation, it was acknowledged there were instances where self-report was impossible. Many documentation sources indicated proxies would be well-justified in certain contexts, but did not indicate who could act as a proxy, when proxies could be used, what domains of patient health they could report on, or how data should be reported. Observer-reported outcomes were typically defined as those based on observed behaviors, however there was not a consistent differentiation between proxy and observer reporting. Based on information extracted from these resources, we developed a checklist of considerations when including proxy-reported measures or using proxies in study design, data collection, analysis, interpretation and reporting of proxy reported data. Conclusion Our targeted review highlights a lack of clarity in capturing, interpreting and reporting data from proxies in adult populations. We provide a checklist of considerations to assist researchers and clinicians with including proxies in research studies and clinical care. Lastly, our review identified areas where further guidance and future research are necessary.