Patient-Patient Centered Outcomes Research最新文献

Objectives: There is growing interest in how probability information is framed and presented in health-related discrete choice experiments (DCEs), given evidence that presentation formats can influence respondents' choices. Recent regulatory and methodological initiatives call for clear and standardized probability presentation but the extent to which these practices have been adopted in DCEs is unclear. This scoping review sought to characterize changes in DCE practices over time compared with prior similar review studies, and to examine the extent to which current practices align with the latest evidence-based guidance.

Methods: We conducted a scoping review of 98 health-related DCE studies published between October 2022 and August 2024 that included numeric probabilistic attributes, extracting data on probability framing, use of visual aids, and alignment with best-practice guidance. Articles were identified from Medline, Embase, Web of Science, EconLit, and PsychINFO.

Results: A total of 583 attributes were presented across the 98 included studies, of which 249 were probabilistic attributes. Probabilistic risk attributes were more common than benefit attributes (present in 85% versus 65% of studies). All risk attributes were framed in absolute terms, and 16% of benefit attributes were framed in relative terms, indicating some deviation from best practices. Graphical aids were frequently used to convey probabilities, more often for risk attributes (59% of studies) versus benefit attributes (47%). Icon arrays were the predominant visual aid (86% of risk visuals, 72% of benefit visuals), but design choices varied widely. More than half of these icon arrays depicted only male figures, and color schemes were inconsistent.

Conclusions: Compared with a decade ago, recent studies increasingly incorporated recommended practices (e.g., pretesting and visual aids). Nevertheless, variability persists in probability framing (especially for benefits) and visual design choices, highlighting the need for further methodological guidance to standardize probabilistic attribute presentation in DCEs.

Background: Expanding access to oral contraceptive pills (OCPs) through over-the-counter (OTC) reclassification may reduce barriers to contraception, but Australian consumer preferences for OTC OCP access remain poorly understood.

Methods: We conducted a discrete choice experiment (DCE) to quantify women's preferences for OTC OCP access in Australia. The DCE incorporated five attributes: method of accessing OCPs, provider training, ability to discuss other health issues, consultation time, and out-of-pocket cost. A D-efficient design generated 12 choice sets. Data were collected through quota sampling from 878 women during November to December 2023, including 359 current OCP users and 519 non-OCP users. Preferences were analysed using mixed logit models.

Results: Women using less effective contraceptive methods or no contraception favoured OTC access, whereas current OCP users did not show the same preference. Across groups, online general practitioner consultations and pharmacist consultations without a private setting were strongly disliked. Predicted uptake of, and switching to, OTC options was highest when OCPs received a high level of government funding, pharmacists had additional training in contraceptive care, women could discuss other health issues, and consultations were brief. Current OCP users were more likely to switch to OTC OCPs if they faced GP co-payments. Support for OTC access was greater among younger women, high-income women, and those with greater household decision-making autonomy.

Conclusions: OTC access to OCPs is likely to be acceptable to many Australian women, particularly if pharmacists have received additional training in contraceptive care and consultations are affordable, private, and sufficiently comprehensive. Careful policy design will be needed to ensure acceptability, support informed contraceptive choices, and maximise the potential public health benefits of OCP reclassification.

Background: Grade 2 isocitrate dehydrogenase (IDH)-mutant (mIDH) glioma imposes significant health-related quality of life (HRQoL) and economic burden.

Objective: The aim of this study was to explore patient preferences and socioeconomic burden in grade 2 mIDH glioma and its treatment.

Methods: A discrete choice experiment (DCE) was administered to patients across the US, UK, Canada and Germany. Attributes included life expectancy, time until tumour progression, side effects, future risk of challenges performing work/usual activities and treatment modality. DCE data were analysed using a mixed-effects logit model. Relative attribute importance scores and trade-offs between attributes were estimated.

Results: A total of 118 participants (56% male; mean age 41.9 years; 67.8% employed) were included in the final analysis. Four of the five attributes were independent drivers of patients' treatment preferences. Participants preferred treatments with additional years of life expectancy and tumour-free progression and were averse to treatments with higher risks of side effects and future challenges performing work/usual activities. Treatment modality did not significantly influence preferences. Based on 10% risk increments, relative attribute importance scores showed life expectancy was the most important driver of choice (26.5%), followed by side effects (21.6%). Side effects (31.9-37.9%) and future issues with work/usual activities (20.6-24.4%) became relatively more important when expressed as 20-30% increments. Patients were willing to trade life expectancy to avoid increased risk of side effects.

Conclusions: Patients placed the greatest value on gains in survival. Avoidance of risks of side effects or impacts to daily activities were also significant predictors of choice, with patients willing to trade life expectancy to avoid risk attributes. Incorporating patient preferences is essential to support shared treatment decision making and ensure treatment strategies align with patients' values and expectations.

Background: Valid and reliable health-related quality of life (HRQoL) instruments are needed in clinical practice to quantify the burden of juvenile idiopathic arthritis (JIA) and capture changes in health over time. We examined the responsiveness of the parent-proxy EuroQol 5-Dimension Youth 5-Level (EQ-5D-Y-5L) instrument in children with JIA.

Methods: This multicenter international cohort study included consecutive children with JIA who were enrolled in the Understanding Childhood Arthritis Network Canadian-Dutch (UCAN CAN-DU) eHealth platform. Demographics, Clinical Juvenile Arthritis Disease Activity Score-10 (cJADAS10) disease activity, Childhood Health Assessment Questionnaire (CHAQ) disability index, and health status were assessed at baseline and at follow-up visits 3-12 months later. Patients were categorized as improved, stable, or deteriorated using cJADAS10 and CHAQ. Responsiveness was evaluated at both the dimension level (distributional change) and continuous score level (EQ-5D-Y-5L level summary score [LSS] and EuroQol Visual Analogue Scale [EQ VAS]) using effect size and standardized response mean (SRM).

Results: A total of 246 patients were included (median age 12 years [interquartile range: 8-15]; 54% female). At the dimension level, children classified as improved showed clear shifts toward lower problem levels across all five EQ-5D-Y-5L dimensions, particularly in physical dimensions, while stable patients showed minimal distributional change. At the continuous score level, improvements were mostly associated with large effect size and SRM for both LSS and EQ VAS (0.8-1.6), whereas changes in stable groups were negligible to small.

Conclusions: The parent-proxy EQ-5D-Y-5L demonstrates responsiveness to clinically defined changes in children with JIA. Its ability to detect improvement while remaining stable in the absence of change supports its use in longitudinal clinical studies and economic evaluations.

Patient engagement is expected to improve the safety and efficacy of anticoagulant medication. By identifying the factors and patient characteristics influencing patients' decisions regarding anticoagulant medication, we aim to support healthcare professionals regarding patient engagement. This review of discrete choice experiments (DCEs) explores the considerations and preferences of patients with various underlying conditions when choosing their anticoagulant medication. Two international databases were searched in March 2024: PubMed and Web of Science Core Collection. The search was updated in July 2025. Eligible studies included original DCE studies that explored the considerations and preferences of patients and covered long-term anticoagulant use beyond the hospital setting (direct oral anticoagulants, vitamin K antagonists, antiplatelet agents, and low molecular weight heparins). The initial search identified a total of 174 records, after which two authors independently assessed the articles for both the title and abstract and the full-text inclusion rounds. After the update, this resulted in the inclusion of 13 articles. The PRISMA 2020 statement was followed and the quality of the included studies was assessed. The results show that overall patients prioritize safety and effectiveness of anticoagulants over convenience factors. If only convenience factors were taken into account, frequency of administration emerges as the most important. Subgroup analysis showed that patient preferences vary based on geographic, demographic, and socioeconomic factors, health status, as well as previous experiences with anticoagulation medication or related health issues. By acknowledging the impact of these diverse factors on patient preferences, healthcare professionals can better support safe and effective anticoagulant care tailored to the needs of individual patients.

In phase I clinical trials, the recommended phase II dose (RP2D) is usually set at or near the maximum tolerated dose (MTD), which is determined based on the observation of dose-limiting toxicities (DLTs). Clinicians typically evaluate toxicities using the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE), with grade three or higher toxicities classified as DLTs. However, it has been repeatedly demonstrated that physicians tend to underestimate patient's symptoms. Therefore, patient-reported outcomes (PROs), especially the NCI PRO-CTCAE questionnaire, can complement clinician assessments by providing direct patient input on adverse events. This integration could lead to a more accurate definition of DLT and better informed RP2D decisions. Moreover, PROs could optimize sample size strategies in later-stage trials and enable comparison of health-related quality of life (HRQoL) data with synthetic control arms to confirm the benefit of a drug, especially in rare oncogene-driven subsets. Whilst stakeholders and regulatory authorities acknowledge the value of integrating PROs early in drug development, they emphasize the lack of methodological guidelines to support broader adoption. The integration of PROs represents an opportunity to improve the patient-centeredness of phase I trials, ultimately strengthening the drug development process.

Objective: This study presents findings from a pilot study that aimed to examine the feasibility of routine measurement of quality of life in residential aged care, including the examination of barriers to and facilitators of collecting and using that data to improve quality of care.

Methods: This study was conducted at two not-for-profit residential aged care facilities in Melbourne, VIC, Australia. All residents were eligible to participate if consent was provided. Self-reported quality-of-life data were collected from residents, alongside proxy-reported data from aged care staff and relatives, primarily using the EQ-5D-5L in addition to a randomly assigned second measure (i.e. The Adult Social Care Outcomes Toolkit [ASCOT], Quality of Life-Aged Care Consumers [QOL-ACC], EQ Health and Wellbeing Instrument [EQ-HWB]). Feasibility was assessed in terms of missing data, residents' level of engagement and understanding, and difficulty experienced by staff and relatives in providing proxy reports. Perceived facilitators and barriers were identified via qualitative interviewers with staff who collected the data.

Results: From 103 consenting participants, we gathered quality-of-life data through self-report (n = 90), staff proxy-report (n = 101) and family proxy-report (n = 49). Most residents (94%) were able to respond to the EQ-5D-5L questions and residents' level of engagement was rated by staff as good. Only a few missing values (0-10%) were recorded for the EQ-5D-5L. Qualitative findings indicate that while quality-of-life data collection has benefits, barriers include time pressures, residents being too unwell to self-report, staff uncertainty about responding on their behalf and issues with the measure itself.

Conclusions: While it is feasible to routinely collect quality-of-life data in residential aged care, addressing the barriers identified will optimise the efficiency of the process and maximise the use of data to guide quality improvement strategies.