Transforming lived experience into a sense of purpose through patient advocacy in oncology research and policy development is increasingly demanded. However, the long-term sustainability of patient advocates — who formally operate as unpaid volunteers — remains under-researched. This study explores the motivational drivers and structural hurdles characterising the field of Dutch oncology patient advocacy to identify the prerequisites for a sustainable patient voice in oncology research and policy development. We conducted a qualitative study among 16 Dutch oncology patient advocates using semi-structured interviews based on the self-determination theory and the Job-Demands-Resources model. Data were analysed through thematic analysis, to understand participant’s experiences, motivations and the structural dynamics of their roles. Participants fulfil three primary advocacy roles: strategic policy representation, knowledge translation and organisational support. Engagement is driven by an intrinsic motivation to transform lived experience into experiential expertise, supported by the fulfilment of the needs for autonomy, competence and relatedness. However, this drive is challenged by significant job demands, including emotional burden, responsibility burden, high workload and organisational demands, and health constraints. While advocates provide mandatory input for research funding, they navigate a landscape of inconsistent financial compensation and varying levels of professional appreciation. The findings of this study reveal a “professional-volunteer paradox”: a systematic tension that challenges the sustainability of the patient voice in oncology research and policy development. To move from symbolic participation toward empowered partnership, a shift in perspective is required. This necessitates institutionalising professional support, fostering role clarity, and ensuring financial and structural equity. By acknowledging advocates as professional experts whose experiential authority is essential, healthcare systems can secure a robust and sustainable role for the patient advocate voice in the future of oncology research and policy development.
Mantle cell lymphoma (MCL) is a rare subtype of non-Hodgkin lymphoma with a high relapse rate and poor treatment outcome. Treatment options for patients with relapsed/refractory (R/R) MCL remain limited and are associated with heterogeneous efficacy and safety profiles, especially in those previously treated with covalent Bruton tyrosine kinase inhibitors (cBTKi). This study aimed to elicit patients’ and physicians’ preferences for treatments for R/R MCL after cBTKi failure. From January to March 2025, a discrete choice experiment was conducted through face-to-face paper-based surveys in China. Respondents completed choice tasks comparing hypothetical treatments defined by seven attributes: duration of response (DOR), overall survival (OS), risk of serious infections, risk of cytokine-release syndrome (CRS), impact of chronic adverse events (AEs) on daily life, risk of discontinuation due to AEs, and treatment procedure. Mixed logit models were employed, and relative attribute importance (RAI) was calculated. Maximum acceptable risk analysis and scenario analysis were conducted to reveal the trade-offs respondents were willing to make. Preference heterogeneity was also explored through subgroup analysis. A total of 150 patients and 150 physicians were included in the analysis. Most patients (76.0
Patient engagement in value assessment and health economics and outcomes research (HEOR) must be meaningful and authentic, not simply patient participation as study subjects. This article provides researchers with guidelines for incorporating patient engagement in value assessment and HEOR grant proposals. In the USA, the Patient-Centered Outcomes Research Institute (PCORI) offers six “foundational expectations” for partnerships in research: representative involvement, early and ongoing engagement, building capacity to work as a team, dedicated funds for engagement and partner compensation, meaningful inclusion in decision-making, and ongoing review and assessment. To these, we added two more expectations—informational exchange and mutual learning, and benefit to patients and involved groups—which emphasize the outcomes of active engagement. For each of the eight expectations, we provide recommendations that inform how research proposals can demonstrate them in the most appropriate manner. While including all eight expectations may not be feasible in every proposal, the framework encourages researchers to advance patient-centered research practices progressively. These guidelines aim to motivate researchers toward including more meaningful and authentic forms of patient engagement in their proposals, ultimately strengthening the quality and patient-centeredness of value assessment and HEOR projects, while fostering collaboration between researchers and patient communities. Patients should be real partners in healthcare research—not just people who take part in studies. Researchers need to work with patients from the very start and throughout the entire project, which means listening to patients’ ideas, experiences, and priorities, and integrating them into the research. This article outlines eight ways in which researchers can enhance their grant proposals with meaningful patient engagement. These include ensuring research team have patients from different backgrounds, incorporating patient input while the study is being planned and designed, providing training and support so patients can confidently participate, paying patients fairly for their expertise and contributions, and establishing collaborative decision-making processes that position patients as equal partners. When patients are real partners, research is more likely to focus on the questions that matter most to patients and their communities, resulting in studies that better inform healthcare decisions and help patients live their best lives.
Digital mental health programs can expand access to psychological support for children and adolescents, especially where stigma, cost, and workforce shortages limit traditional care. Understanding which features young people and parents value most is essential for improving engagement and real-world uptake. We conducted a cross-sectional discrete choice experiment to quantify preferences for online youth mental health program features among adolescents and parents. Data were collected through an online survey of BRAVE Program families with prior experience using the BRAVE self-help anxiety program. Participants completed ten choice tasks comparing two program options varying by content format, effectiveness or professional contact, personalization, cost, and session frequency or additional supports. Preferences were analyzed using mixed multinomial logit models. Among 238 participants (80 parents; 158 adolescents), both groups preferred programs that were engaging, interactive, and personalized, and both were sensitive to program cost. Parents placed strong emphasis on effectiveness, with cost (36
As therapeutic options for inflammatory bowel disease (IBD) expand, understanding patient preferences—and how these vary across subgroups—is critical for patient-centered drug development and evaluation. This patient preference study aimed to (1) determine the relative preference weights of IBD treatment- and disease-related attributes (i.e., which features most influence patients’ treatment choices) and (2) explain preference heterogeneity. A survey incorporating a discrete choice experiment (DCE) was disseminated worldwide. Patients repeatedly chose between hypothetical treatment profiles varying across 14 attributes using a partial profile design. The survey design was informed by literature review, qualitative research, and advisory boards. Mixed logit models were applied. Data from 1452 patients (51.0
Parkinson’s disease (PD) is a progressive neurodegenerative disorder characterized by motor and non-motor symptoms that significantly impact quality of life. As the disease advances, treatment options expand from oral medications to include device-aided therapies, which differ widely in their mechanisms, invasiveness, and impact on daily life. These therapies have distinct attributes that may align differently with individual patient preferences; therefore, understanding such preferences is crucial for informed, shared clinical decision making. This scoping review synthesizes quantitative evidence on treatment preferences among people with Parkinson’s disease (PwP), highlighting research gaps on key attributes. A systematic search of PubMed and Embase (January 2026) identified studies using quantitative stated-preference methods—discrete choice experiments, best-worst scaling, conjoint analysis, or threshold techniques—with PwP, care partners, or healthcare professionals. Twelve studies met inclusion criteria. Treatment efficacy—particularly increased ‘ON time’ (periods without troublesome dyskinesia)—and treatment modality were the strongest drivers of preferences. PwP favored non-invasive treatments, such as oral or sublingual formulations, over surgical options like deep brain stimulation. Convenience factors, including pill burden and device management frequency, also influenced preferences but were generally secondary to efficacy and modality. Safety attributes, such as risk of depression or cognitive decline, were important but often outweighed by perceived benefits in symptom control. No studies assessed care partners’ preferences for modalities such as subcutaneous infusions. PwP prioritize treatment efficacy and non-invasive modalities, with convenience and safety considered secondary. Further research should explore preferences for new treatment modalities and include care partners’ perspectives to optimize treatment planning and outcomes in PD care.
Best–worst scaling (BWS) is a theory-driven method to measure priorities with growing applications in health and other literatures. Priorities in BWS are elicited through a series of choice tasks. While several study design factors have been linked to the overall quality of published BWS studies, there is a paucity of evidence linking the choice of task format on quality. We sought to identify the range of task formats used in published BWS studies, assess how frequently each format was applied, and examine whether task format influences the quality of published BWS studies. We identified the task format of BWS studies published prior to January 1, 2025, via example tasks, descriptions, appendices, and/or supplementary materials. We then developed a simple taxonomy of the most common format types (e.g., best anchor on the left), except for when task format was missing. Quality scores were calculated using the PREFS checklist and then used to test for differences across task formats in a two-sample t-test. Z-scores were used to summarize differences relative to the best anchor on the left, and differences across task format types were examined using one-way ANOVA with pairwise comparisons. Among the 714 published BWS studies identified, over a third were missing information on task format (n = 253). The remaining studies were classified into four types: best anchor on the left, which was both the first format used in a published study and accounted for 62
People with type 2 diabetes (PwD) and healthcare professionals (HCPs) often perceive daily basal insulin regimens as burdensome, and many PwD requiring basal insulin fail to achieve glycemic targets. Once-weekly insulin efsitora alfa (efsitora) demonstrated non-inferior efficacy and similar safety compared to daily basal insulin. To understand PwD perceptions of efsitora treatment and HCP perceptions of prescribing once-weekly efsitora, qualitative interview sub-studies with PwD and HCPs were performed as part of the QWINT trials. Four qualitative interview sub-studies explored the experiences of PwD and HCPs with efsitora during QWINT-1, QWINT-2, and QWINT-3 trials in the United States (US), Canada, or Mexico. Telephone interviews were conducted in English or Spanish and followed a semi-structured interview guide. De-identified interview transcripts were coded and analyzed deductively following a content analysis approach. The interview sample was described using sociodemographic and key clinical trial data. The interviews included 72 PwD treated with efsitora and 22 HCPs. Across trials, PwD highlighted the convenience, flexibility, and ease of use of efsitora. Insulin-experienced PwD (QWINT-3) particularly valued the reduced injection frequency, ease of adherence, and improvements in glycemic control and overall well-being. Insulin-naïve PwD (QWINT-1 and QWINT-2) especially appreciated ease of use/convenience. Most PwD reported that efsitora improved their quality of life, expressed high satisfaction and confidence with its use, and were willing to continue treatment beyond the trial period. HCPs highlighted improved adherence, fewer injections, better glycemic control, and enhanced convenience for both PwD and clinical staff as the main advantages of once-weekly dosing. HCPs felt that PwD already on basal insulin and/or with adherence challenges or busy or irregular schedules might particularly benefit from once-weekly dosing. The most frequently cited disadvantage of efsitora was reduced opportunity for dose adjustment compared to daily regimens. This qualitative study provides in-depth insights of the experiences of PwD and HCPs with efsitora treatment. Both groups emphasized its convenience, flexibility, and ease of use, while remaining confident in its glycemic control. These findings augment both clinical and patient-reported data and indicate that efsitora may mitigate challenges in diabetes management by reducing the burden and demands associated with conventional daily basal insulin regimens.
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. 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. 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
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.
INTRODUCTION:Spinal muscular atrophy (SMA) is a rare autosomal recessive hereditary neuromuscular disease that leads to progressive muscle weakness and atrophy. Available treatments differ significantly in terms of effectiveness, administration, costs, and side-effect profiles. Currently, evidence regarding patient treatment preferences remains scarce, especially in developing countries. This study aimed to provide evidence on patient preferences toward SMA treatments. METHODS:We conducted a discrete choice experiment (DCE) among patients with SMA in China. Attributes were selected on the basis of a systematic literature review, in-depth patient and expert interviews, and validation through a focus group discussion. Five attributes were included: mode of administration, annual out-of-pocket costs, motor function, respiratory function, and incidence of side effects. A mixed logit model was applied for preference estimation. On the basis of the model results, we derived attribute relative importance (RI), conducted scenario analyses to predict the uptake of hypothetical treatment profiles, and performed willingness-to-pay (WTP) analyses to quantify the monetary value of attribute improvements. RESULTS:A total of 202 patients completed the questionnaire, with 190 included in the final analysis. Respondents had a mean age of 29.2 years, were predominantly diagnosed with type 3 SMA (67%), and mainly received parental care (70%). Annual out-of-pocket expenses (RI = 56%) emerged as the most influential attribute, followed by motor function improvements (RI = 20%), incidence of side effects (RI = 13%), respiratory function (RI = 8%), and mode of administration (RI = 3%). Patients expressed a substantial willingness to pay of renminbi (RMB) 32,217 annually for improvements in motor function. Scenario analyses indicated that 95% of patients would prefer a new treatment profile providing enhanced motor function, even at higher costs. CONCLUSIONS:Adult patients with type 2 or type 3 SMA in China strongly prefer affordable treatments with oral administration, improved motor and respiratory functions, and minimal side effects. Understanding these preferences can guide clinical decision-making, supporting personalized treatment strategies that may ultimately enhance treatment adherence and clinical outcomes.
IntroductionWhile medicine regulation in many countries requires prescription drug labels to classify side effects based on their incidence, when eliciting preferences of patients, most discrete choice experiments (DCEs) group side effects by severity. This mismatch between the presentation of side-effect attribute labels in DCEs and real-world choice contexts may impact patients' choices, bias risk preferences, and threaten external validity. We investigate whether using incidence compared with severity labels impacts preferences and DCE reliability.Methods1105 Dutch respondents aged 60 years or older completed a survey including a DCE on influenza vaccination uptake, receiving either severity (i.e., mild or severe) or incidence (i.e., very rare or very common) attribute labels summarizing the included side effects. We randomized only the attribute labels across respondents, all else was held constant. We evaluated the effect of severity versus incidence labels on maximum acceptable risks (MAR), heterogeneity in side-effect risk preferences, reliability, choice consistency, and predicted uptake. Choice data were analyzed using mixed and heteroscedastic logit models. Other survey questions pertained to demographics, experiences, and attitudes towards the included mild and severe side effects.ResultsRespondents' perceptions of side effect severity did not always correspond to the clinical severity labels. Using severity compared with incidence labels increased the MAR and preference heterogeneity for severe/very rare side effects. No significant differences with regards to MAR and preference heterogeneity for mild/very common side effects, reliability or choice consistency were detected.DiscussionUsing severity compared with incidence-based attribute labels impacted MAR estimates and preference heterogeneity, suggesting potential priming effects. However, predicted uptake was not significantly impacted. Still, respondents' severity perception may not align with conventional severity labels, and in many countries, categorizing side effects using incidence-based attribute labels may represent patients' real-world choice contexts more accurately.
Background and ObjectiveHidradenitis suppurativa is a burdensome chronic inflammatory skin disorder. While surgery can be essential for many patients, preferences and values regarding surgery are insufficiently characterized. This study aimed to investigate preferences of adult patients with hidradenitis suppurativa in the USA when choosing an hidradenitis suppurativa surgery method.MethodsA discrete choice experiment was conducted. Patients were asked to choose between two hypothetical procedures 15 times. Literature review and patients with hidradenitis suppurativa and expert interviews informed treatment attributes selection. A mixed logit model was applied to estimate preference coefficients. Subgroup analyses and a latent class model were conducted.ResultsRelevant attributes for the 205 participants (90% female, aged 41 years) ranked as follows: effectiveness on lesion improvement (36.75%), recurrence/persistence risk (15.02%), out-of-pocket costs (13.27%), post-surgical wound care (13.27%), post-surgical pain duration (9.64%), recovery time (7.12%) and complications risk (4.94%). A latent class model revealed participants in class 1 including more people of White/Caucasian, Asian and Latino ethnicity and full-time and part-time employed and non-working people put a higher value on risk of return and less priority on wound care post-surgery and out-of-pocket costs. For participants of class 2 consisting of more people of Black or other ethnicity and self-employed, students or retired people, the opposite was observed.ConclusionsOf the evaluated attributes, effectiveness for improvement, recurrence risk, out-of-pocket costs and post-surgical wound care contributed most to patient preference. Surgeons should account for these aspects of procedures when offering surgical interventions. Studies of surgical outcomes should place high value on these aspects of procedural outcomes.
Video consultation in family health stations offers potential for improving healthcare access for cultural and religious minority populations who face unique challenges in healthcare utilization. Ultra-Orthodox Jewish women provide a case study for understanding preferences in hard-to-reach communities. We aimed to elicit preferences for video consultations versus in-clinic consultations at family health stations among Ultra-Orthodox women, and to demonstrate the application of discrete choice experiment methodology in religious and culturally sensitive research. A face-to-face, paper-based, discrete choice experiment was conducted. Following culturally adapted qualitative interviews and focus groups, four attributes were examined: consultation timing, waiting time, child presence requirement, and nurse familiarity. Participants evaluated 12 choice scenarios comparing video consultations, using rabbinically approved devices, with traditional in-clinic consultations. A mixed logit model analyzed preferences and heterogeneity across demographic subgroups. A total of 174 women participated, with a mean age of 30.0 ± 6.1 years and a mean of 3.45 ± 2.1 children. Waiting time was the strongest predictor of consultation preference (β = −1.50, p < 0.001), followed by consultation hours (β = 1.10, p < 0.001), provider familiarity (β = 0.55, p < 0.001), and child presence requirement (β = −0.51, p < 0.001). Consultation type (video vs in person) was not independently significant. Subgroup analyses showed that older women had weaker preferences for video consultation. Policy simulation estimated that optimized video consultation configurations could capture 58–78
