Jessica Roydhouse

Purpose: Informal carers (‘proxies’) are often asked to report on the health of individuals who cannot self-report, either from their own (‘proxy-proxy’) or the patient’s (‘proxy-person’) perspectives. It remains unclear which perspective proxies find most useful. Understanding this is important for designing measures that proxies can complete. We conducted an exploratory study on which perspective proxies find most useful and why, when reporting pain and physical function in dementia and symptoms in multiple sclerosis (MS). Methods: This study used data from two cross-sectional online surveys: one for proxies of people with MS and another for people with dementia. Proxies were asked to indicate which perspective they found most useful and why for communicating symptoms in MS, and pain or physical function in dementia, to healthcare professionals (HCPs). Quantitative responses were summarised using percentages; qualitative responses were analysed using content analysis. Results: 55 proxies reported on MS symptoms, 29 on pain in dementia, and 33 on physical function in dementia. The most selected perspectives were: 35% (19/55) proxy-proxy for MS symptoms, 38% (11/29) proxy-person for pain and 39% (13/33) either of the perspectives for physical function. Proxy-person was valued for empathy and person-centredness, especially in reporting pain and MS symptoms. Proxy-proxy was appreciated for objectivity and reducing conflict, particularly in physical function. Using either perspective was considered most useful for balancing views and reducing misinterpretation in both conditions. Conclusion: Proxies’ most useful perspectives vary slightly by domain and condition. Incorporating an understanding of how proxies approach the challenge of reporting may improve measure design.

Background: Patient‐reported outcome measures can highlight the impact of kidney failure and kidney replacement therapy on a person's health‐related quality of life and symptom burden. But their implementation is limited and heterogeneous within Australian and New Zealand kidney care. Objective: Identify one generic health‐related quality of life and one kidney‐specific patient‐reported outcome measure suitable for inclusion within the Australia and New Zealand Dialysis and Transplant Registry, including the mode and location of administration, timing and frequency of completion, and data presentation format. Design: A three‐phase modified Delphi study comprising two online surveys and a virtual consensus workshop. Participants: Patients, carers, clinicians, researchers, and kidney registry staff from Australia, New Zealand and the United Kingdom. Measurements: Participants voted on: their preferred patient‐reported outcome measures, the mode and location of administration, timing and frequency of completion, and how they would like the data presented at the individual and aggregated levels. Results: Fifty‐three people consented to the study, 41 completed Survey #1, 43 completed Survey #2, and 30 attended the consensus workshop. The EuroQOL‐5 Dimensions‐5 Levels for generic health‐related quality of life and the Integrated Palliative care Outcome Scale‐Renal for kidney‐specific outcomes reached majority vote. Participants agreed on electronic completion at home or in the dialysis unit to allow for staff support if needed. Consensus was not reached on the frequency of administration; most voted for completion at the commencement of dialysis or transplant and 6‐monthly thereafter as a minimum. Conclusion: The EuroQOL‐5 Dimensions‐5 Levels and the Integrated Palliative care Outcome Scale‐Renal were recommended for collection by the Australia and New Zealand Dialysis and Transplant Registry. These will enable standardised, meaningful assessments of patients' health outcomes and support continuous quality improvement across kidney care services bi‐nationally.

Purpose: Patient-reported outcome measures are often administered with specific recall periods to standardise reporting. However, research on recall periods in proxy reporting is limited. This study sought to explore (1) how informal carers interpret the “last 7 days” recall period of the EQ health and well-being instrument (EQ-HWB)-9 and (2) the perspective they adopt when completing the measure to report on the health and well-being of persons with dementia. Methods: A qualitative, descriptive study was conducted. Convenience sampling was used for recruitment. We interviewed informal carers of persons with dementia in Australia using semi-structured cognitive interviews. Participants were asked to think aloud when completing the EQ-HWB-9, and three additional positively-framed questions from the EQ-HWB. Thematic analysis was used, where transcripts were inductively coded to capture new insights and deductively coded as guided by the EQ-HWB-9 questions. Results: Nineteen carers completed interviews. Carers used two main retrieval strategies: supplementing recent observations with key events or self-report occurring slightly outside the recall period, and drawing on the past to contextualise recent behaviours. All participants used their own observations and opinions of the person with dementia (“proxy–proxy perspective”). When questions about the person’s feelings were difficult to answer, carers relied on various observable behaviours as indirect indicators. Nearly half also imagined how the person might answer (“proxy–patient perspective”), even if they sometimes disagreed with the person’s self-assessment. Conclusion: The 7-day recall period for the EQ-HWB-9 was more consistently adhered to for observable aspects of health. Wording changes may be needed to support consistent use, particularly for less observable items. Challenges in perspective-taking and adherence were also identified, suggesting areas to address in instructions.

Background and Objective Patient preference studies have been used to identify aspects of medical products that matter to patients. This paper sought to summarise patient preferences for treatment processes (mode and frequency). Methods Search strategies based on existing validated search filters were applied to databases including MEDLINE, Embase, Scopus, EconLit and Health Preference Research. Additional references were obtained by searching the reference lists of systematic reviews. English-language peer-reviewed studies involving patients that quantitatively elicited preferences and compared different modes or frequencies were eligible. Data extraction was performed by two reviewers using Covidence. As a heuristic, we considered if process attributes were ranked in the top two attributes. The Purpose, Respondents, Explanation, Findings, Significance (PREFS) checklist (0–5, higher = better) was used to evaluate study quality. Results Data were extracted from 164 articles of an identified 16,357 (12,872 screened by title/abstract and 795 full text screened). Mean study quality was 3.48 on PREFS (standard deviation = 0.70), but patient engagement was rarely reported in studies (n = 20, 12.2%). Most studies included both mode and frequency attributes, either presented as a combined attribute (n = 65, 39.6%) or as individual separate attributes (n = 43, 26.2%). Regardless of how attributes were presented, mode/frequency attributes were infrequently in the top two attributes. In terms of level preference, when an oral mode was included as a level, the oral level was the most preferred level in nearly all studies. Patients with less severe disease or who were less experienced with treatment tended to prefer oral modes or have preferences against injection. Conclusions Mode/frequency attributes are not necessarily the most important attributes, but patients, particularly those with less treatment experience or more mild disease, may have strong preferences. Although PREFS scores indicated high study quality, future studies would benefit from deeper patient engagement. In line with ISPOR’s definition, patient engagement should extend beyond participation to meaningful involvement across the research lifecycle, including opportunities for co-authorship and partnership in study design, conduct and dissemination.

Introduction: There is widespread interest among patients, clinicians, regulators and other constituents in post-treatment patient-reported cancer data. Side effect bother is a patient-reported outcome (PRO) that can capture an important aspect of tolerability. In this study, we examined side effect bother at cancer treatment discontinuation and post-discontinuation in commercial cancer trials. We sought to understand completion rates, the extent of bother and its association with other PROs. Materials and Methods: Data were evaluated from three trials in patients with solid tumours (renal cell carcinoma and breast cancer). Side effect bother was measured with the Functional Assessment of Chronic Illness Therapy (FACIT) GP5 item. Symptom items were drawn from FACIT and function items were drawn from the EQ-5D-3L. FACIT items, including the GP5, are on a 0–4 scale (higher = worse symptoms/bother), and were dichotomised as 0–1 (“low”) vs 2–4 (“moderate”). EQ-5D-3L items were characterised as no problems (1) and some problems (2–3). Descriptive and correlation analyses were conducted separately for each trial. Results: Among patients who received treatment, completion rates at discontinuation for most items were at least 70%, and 52% to 78% at follow up. More than 20% of patients had high side effect bother at and after discontinuation, and similar percentages were seen for symptom items and functioning problems. GP5 and most items were at least somewhat correlated (≥ 0.2 in nearly all evaluations). Discussion and Conclusions: Persistent side effect bother and symptomatic and functional detriments at and after discontinuation suggest capturing this information post-treatment can inform understanding of tolerability, particularly with improved PRO completion.

Background: There is growing recognition of the importance of patient-reported tolerability in complementing traditional clinician-reported safety evaluation of cancer therapies. Recent regulatory guidance listed the evaluation of overall side effect impact as a core patient-reported outcome in oncology clinical trials. A single item (‘GP5’) that asks about side effect bother is included in the Functional Assessment of Chronic Illness Therapy and has been used to capture overall side effect impact. This paper sought to expand the evidence base for GP5 by examining its association with clinician-reported treatment-emergent adverse events and patient-reported global health. Methods: We examined six commercial cancer clinical trials that collected GP5. The patient population was drawn from the safety population and the analysis focused on the first on-treatment assessment. Clinician-reported adverse events were classified as symptomatic if such adverse events were considered amenable to patient self-reporting (e.g. nausea). Chi-square tests and Pearson’s correlation were used to examine associations. We considered adverse event grade and frequency, both for symptomatic adverse events and any type of adverse events. Global health was measured using the visual analogue scale of the EuroQol-5 Dimensions-3 Levels measure. ‘Moderate-severe’ bother was characterised as scores of 2–4 on a 0–4 point scale for GP5, and ‘severe’ bother was characterised as scores of 3–4. Analyses were conducted separately for each trial. Results: Data from 3,557 patients were included. Across the trials, most (71.7%–94.2%) patients had an adverse event of some kind, but fewer (17.1%–44.4%) had an adverse event of grade 3 or higher. In general, fewer than 50% of patients (20.6%–44.2%) reported moderate-severe bother and 5.8%–17.% reported severe bother. There were consistent, albeit not always statistically significant, associations between GP5 and adverse events, and GP5/global health correlations ranged from −0.17 to −0.41. Discussion: GP5 is associated with both clinician- and patient-reported symptoms, suggesting its validity and usefulness as part of comprehensive tolerability assessment of cancer trials.

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.