Background: Randomized controlled trials (RCTs) are needed to evaluate multicancer detection (MCD) tests. A novel “intended-effect” RCT design in which control group participants receive MCD testing but results remain undisclosed can increase statistical efficiency. Prospective participants may find this design unacceptable due to ambiguity aversion introduced by the existence of undisclosed but theoretically knowable test results. We evaluated this effect and tested if reducing ambiguity by ensuring the unknowability of test results mitigates this bias. Methods: US adults aged 45 to 70 y ( N = 1,576) were randomized to read about 1 of 4 RCT designs evaluating MCD testing: 1) RCT with a conventional control group (no testing), 2) RCT with a standard intended-effect control group (ie, control gets testing but not the results), 3) modified intended-effect RCT emphasizing disclosure safeguards to ensure the unknowability of control group test results to participants and researchers, and 4) modified intended-effect RCT with time-delayed testing on control group blood samples after trial completion. Outcomes were study interest (5-point scale, with higher values indicating more interest) and intention to participate (yes/no). Analyses of variance, chi-square tests, and regression-based analyses examined the main effects and potential mediating roles of the perceived net benefit of participating in the study and trust in study researchers. Results: Intention to participate was similar across all conditions (range: 14%–21%). Study interest was higher in the conventional control ( M = 3.73) vs the 3 intended-effect designs ( P < 0.05), which did not differ ( M s = 3.36–3.45). The negative influence of intended-effect designs on study interest was partially mediated by perceived net benefit. Conclusion: Intended-effect RCT designs lower interest in MCD trial participation, in part by reducing prospective participants’ perceptions of benefiting from the trial. Strategies to reduce ambiguity do not mitigate this effect.
Background: Patients often report that clinical guidance does not reflect personal values, such as balancing survival with quality of life. Values-clarification tools can improve treatment alignment but have been understudied. Objective: To evaluate the impact of the Values and Outcomes to Improve Cancer Experiences (VOICE) tool on values clarification and shared decision making (SDM) in simulated encounters. Methods: Adults (age ≥60 y) with advanced cancer were randomized to receive VOICE or an American Cancer Society (ACS) communication guide. Participants completed 2 simulated decision-making encounters in randomized order, values based (VB; clinician-initiated discussion of patient values) and non–values based (NVB; values addressed only if patient initiates), with medical students. Two independent raters assessed values‑clarification and SDM behaviors (interrater reliability = 0.82). Patient-reported SDM was assessed with validated measures. Postsimulation interviews were thematically analyzed. Results: Forty-four adults (aged 60–88 y) participated in 88 simulated encounters focused on discussing a secondary cancer diagnosis and treatment options. VOICE recipients demonstrated higher rater-assessed values clarification than those receiving the ACS guide in VB (91.5 vs 80.1, P = 0.01) and NVB (25.2 vs 16.2, P = 0.01) encounters. During NVB encounters, more participants receiving VOICE initiated a values discussion (48% vs 13%). Values-clarification ratings were higher when clinicians initiated VB discussions than patient-initiated discussions (92.9 vs 35.7; P < 0.001). Patient-reported SDM was better in VB versus NVB encounters (relative risk [RR] = 1.71, P = 0.04). VOICE better prepared participants for SDM (70.0 vs 0.0; P < 0.001). Qualitative findings suggest VOICE led to greater self-reflection and values clarification. Conclusions: In this randomized simulation study, VOICE improved older adults’ preparation for values-based discussions and supported reflection on how treatment options align with what matters most to them. Findings highlight the importance of pairing values-clarification tools with clinician-initiated values-based discussions and for further evaluation in real-world settings.
Background: Shared decision making is an effective strategy for collaborative goal setting and action planning with older patients. However, many older patients feel unable to actively participate. Increasing patient participation requires preparing patients for this process. The 4-Circles tool, a validated visual tool designed to support the goal setting phase of shared decision making, might serve as a preparation tool. This study describes the development of the 4-Circles tool 2.0 and examines its feasibility. Methods: We applied a human-centered design consisting of 3 phases: developing a prototype, exploring feasibility, and finalizing the 4-Circles tool 2.0. Data were collected through co-creation sessions with older patients ( n = 9), primary care professionals ( n = 10), and experts ( n = 3) as well as through (focus group) interviews with primary care professionals ( n = 8) and interviews with older patients ( n = 8). We applied conventional content analysis. Results: The 4-Circles tool 2.0 is a self-reflection booklet accompanied by training for professionals. The feasibility analysis showed that professionals gained deeper insights into older patients’ personal goals. Older patients reported finding it easier to articulate their goals. However, professionals perceived that some older patients might lack the cognitive abilities required to complete the tool, adopted a passive role, or feared giving “wrong” answers. For some older patients, goal setting was not considered valuable because they perceived their potential for improvement as limited. Conclusion and Implications: Feasibility testing of the 4-Circles tool 2.0 showed that professionals gained deeper insights into older patients’ goals. However, using self-reflection tools such as the 4-Circles tool 2.0 may be challenging because of the power asymmetry experienced between professionals and older patients. This issue should be addressed more actively during the implementation of such tools.
Objective: The population-level quality-of-life impact of nonfatal health outcomes, including prolonged hospitalizations and long COVID, remains poorly understood. In this study, we estimate the cumulative quality-adjusted life-year (QALY) loss attributable to fatal and nonfatal COVID-19 outcomes in the United States and compare that burden with that of other diseases. Methods: We developed a probabilistic model that accounts for the loss in population health due to COVID-19 symptoms, hospitalizations, long COVID, and deaths. The model was applied to weekly county-level data from July 2020 to December 2022. We used appropriate probability distributions to describe uncertainty in parameter values and used 1,000 repeated samples from these distributions to estimate the expected QALY loss and the 95% uncertainty interval (UI). Results: We estimate the cumulative QALY loss as 9,242,000 (95% UI: 7,808,000–11,311,000) life-years. Following deaths, long COVID was the second most significant contributor to QALY loss, with an estimated annual QALY loss of approximately 865,000 (95% UI: 355,000–1,591,000). This burden is comparable to the total annual disability-adjusted life-years (DALYs) associated with liver cancer: 581,000 DALYs (95% UI: 549,000–607,000). Although symptomatic infections contributed less to overall QALY loss, their burden was comparable to that of HIV/AIDS (377,000 DALYs, 95% UI: 309,000–465,000). Conclusion: The quality-of-life burden of nonfatal COVID-19 outcomes, particularly long COVID, is on the same scale as that of major chronic and infectious diseases. As COVID-19 mortality declines and the pandemic transitions to an endemic phase, it is essential to recognize and address the long-term effects of nonfatal outcomes as public health priorities.
Background:Preventive tests and procedures (PTPs) help to reduce disease burden. However, patient expectations often differ from current guidelines, resulting in overuse or underuse of PTPs. Shared decision making can bridge this gap but requires clear, evidence-based information. This study aimed to develop and conduct an initial evaluation of prototype fact sheets designed to support informed decisions about PTPs. Methods:We selected 11 PTPs based on their potential for over- or underuse. For each, we developed a fact sheet following guideline recommendations. These summarised key benefits, risks and relevance using verbal, numerical and graphical formats. User experience and comprehensibility were assessed through a survey among a university population. Participants rated comprehensibility, content and design on a 5-point scale. Approval was defined as the proportion of 'very good' or 'rather good' ratings. Participants giving poor ratings were invited to provide free-text comments. Subgroup analyses were performed by age and educational level. Results:A total of 2,082 individuals answered all relevant questions to fact sheets. The median fact sheet approval score was 77% (interquartile range: 55%, 91%). Six of the 11 fact sheets received an approval of greater than 80% for comprehensibility in general. Lower approval was reported for risk descriptions, usefulness in doctor-patient conversations and visuals. Cancer screening sheets scored the lowest. Subgroup analyses showed lower approval in younger age groups and among individuals with higher levels of education. Conclusions:Initial user testing of the fact sheets showed generally positive ratings regarding comprehensibility and user experience. However, the presentation of specific risks and the use of visuals needed to be adapted, especially for cancer-related topics. The findings highlighted the importance of tailoring informational materials to effectively support shared decision making.
Background: The transition to adulthood is a worrying time for female youth with a physical disability and their mothers. We sought to develop an instrument to assess which transition-related worries are most (and least) worrisome to youth and mothers to inform transition preparation interventions.Design: A community-centered approach was used to identify and refine the most common transition-related worries of youth and mothers. A novel best-worst scaling (BWS) instrument was created in partnership with an advisory board of female youth with a physical disability and their mothers. BWS is a theory-driven survey method to identify priorities. The instrument was then pretested, refined, and piloted with youth and mothers. Findings were assessed by consultation with stakeholders.Results: After input from youth and mothers, 11 transition-related worries were included in the pilot instrument using an object case experimental design. Pilot testing was conducted with 22 youth and 16 mothers, including 3 mother-daughter dyads. The completion rate was 86% for youth and 100% for mothers. The instrument was sensitive enough to detect differences between youth and mothers at both the aggregate and dyad level. On aggregate, both youth and mothers worried more about health-related compared to social-related worries but differed on the prioritization of independence-related worries. Discussion of results with participants and an advocacy group confirmed the face validity of the findings.Limitations: Male youth and fathers were not included.Conclusions: The novel BWS instrument is feasible for female youth and mothers to complete independently and was valid in prioritizing worries on the aggregate and dyad levels.Implications: A subsequent large-scale study using the instrument may inform transition initiatives, while use among in a clinical setting may guide decision making.
Background: Adolescents and young adults (AYA) comprise 50% of sexually transmitted infections (STIs) diagnosed annually. AYA frequently access emergency departments (EDs) for health care. Thus, the ED could be a strategic venue for the diagnosis and treatment of STIs. Objective: Cost-effectiveness analysis examining screening strategies for Chlamydia trachomatis and Neisseria gonorrhea (CT/GC). Design: Decision analytic cost-effectiveness model. Setting: Six pediatric EDs. Participants: AYA 15 to 21 y of age seeking acute care at pediatric EDs. Interventions: 1) Usual care, 2) targeted screening (using a computerized sexual health survey), and 3) universally offered screening. Main Outcomes and Measures: Cost in 2024 US dollars and effectiveness measured as STIs detected and successfully treated. Secondary effectiveness outcome metric: quality-adjusted life-years (QALYs). The cost perspective is the direct health care sector, and the time horizon is lifelong. Results: Targeted screening was the most effective and most costly (incremental cost-effectiveness ratio [ICER] of $517 per case detected and successfully treated). In a secondary analysis using QALYs lost for long-term complications of untreated CT/GC, targeted screening had an ICER of $23,320/QALY. In this analysis, usual care was dominated, being more costly and less effective than universally offered screening. In subgroup analyses of female versus male, only cohorts using $/QALY, targeted screening remained highly cost-effective for females ($6,389/QALY) compared with universally offered screening but was not cost-effective in males. Conclusions and Relevance: Targeted screening is a highly cost-effective strategy for detecting and treating STIs in adolescents seeking pediatric ED care compared with universally offered screening, with an ICER of $517 per case detected and treated. When considering quality of life for female- versus male-only subgroups, screening for males becomes less clear.
Background: Improving shared decision making (SDM) training curricula may support wider training uptake and the use of SDM across health care settings. Limited uptake of a freely available SDM training curriculum suggested it could benefit from a systematic review and revision process. This report describes the process and revisions of the SHARE Approach curriculum, subsequently used in an implementation trial demonstrating that the revised curriculum remained effective. Methods: A qualitative study using a systematic review on shared decision making, training, and physicians published between 2015 and 2020; clinician and patient reviews and feedback on curriculum training materials; and synthesis of recommended improvements and a team-revised curriculum for testing in clinical practice. Results: Recommendations from the literature and reviewers (9 clinicians and 6 patients) focused on substantially reducing the length of the training curriculum, ideally under 4 h. Reviewers found original content well done and important but suggested removing content that was not explicitly focused on training clinicians how to implement shared decision making in clinical practice by removing discussions of applicable laws, medical interpreters, and patient-centered outcomes research while retaining important strategies for addressing patient health literacy/numeracy and use of decision aids. Limitations: Clinician and patient feedback might not represent the full scope of clinical specialties or patient types who may have different recommendations for revising the original curriculum for other clinical settings. Conclusion: Literature review and user feedback agreed that training curricula on shared decision making needed to be shorter and focus on only essential techniques clinicians need to effectively implement shared decision making in routine practice. Implications: A substantially shortened SHARE Approach curriculum is effective and may facilitate broader shared decision making training of clinicians.
Background:Implementation delays reduce population-level effects of evidence-based interventions. We conducted a modeling study to estimate the effect of priority policy decisions on a human papillomavirus (HPV) vaccination program in Kenya. Methods:We used a static cohort model to estimate the health effects and costs of introduction and 1-dose switch delays, switching to nonavalent vaccine (9vHPV), supply disruptions, and various scale-up scenarios. Costs were evaluated from the health system perspective. We estimated cervical cancer cases, deaths, disability-adjusted life-years (DALYs), and program and health care costs. Results:Compared with no vaccination, maintaining current program performance would avert approximately 173,000 (95% UI: 149,000-191,000) cases and 2.7 (2.3-3.0) million DALYs across 2019 to 2100. A gradual scale-up of the current 2-dose quadrivalent vaccine (4vHPV) program would avert an additional 16,000 (14-18,000) cases (9%), increasing to 33,000 (28-37,000) cases (19%) if a multiage catchup is implemented in 2030. Accelerated scale-up of 1-dose bivalent vaccine (2vHPV) would avert 184,000 (164-199,000) cases and 3.0 (2.6-3.2) million DALYs, compared with no vaccination; translating to an additional 11,000 (2-17,000) cases averted (6%) compared with maintaining a 2-dose strategy, but at lower program ($81 million vs $76 million) and treatment costs ($2.09 billion vs $2.07 billion). HPV vaccination introduction in 2015 rather than 2019 would have averted an additional 74,000 (43%) cases. Annual vaccine supply disruptions translates to less than 9,000 (200-16,000) cases (5%) and 212,000 (74-305,000) DALYs averted (7%) compared with a 1-dose strategy. A 1-dose 9vHPV strategy would have more health benefits and save additional treatment costs compared with a 1-dose 2vHPV vaccine. Conclusions:Prompt 1-dose switch and rapid scale-up and adoption of a 9vHPV program should be priority policy decisions for Kenya.
Purpose. The diagnosis, management, and therapy of cancer are rapidly advancing and becoming more costly. Data linking the diagnosis, management, and prescription of systemic anticancer therapy (SACT) are not publicly available, are time-consuming to obtain, and are onerous to analyze. We aimed to illustrate how a simple expert elicitation method can be used rapidly to calculate the cost of diagnosing and treating a cancer by stage of disease. We illustrate the method using the example of melanoma. Methods. We designed a simple structured elicitation exercise for melanoma experts from the United Kingdom with the aim of describing the diagnosis, management, and SACT prescription for people with melanoma and the associated proportions offered each option. We modeled experts’ beliefs using scaled beta distributions. We used random-effects meta-analysis to combine the estimates. Published unit costs (£; 2024–2025) were multiplied by estimated proportions to calculate the mean costs for the diagnosis, management, and SACT by disease stage. Results. Seven dermatologists, 5 oncologists, and 4 surgeons participated (2022–2023). There was variation in the estimates of the proportions receiving possible diagnosis, management, or SACT options. Diagnosing suspicious lesions cost £424 to £699 depending on the investigations required. Mean (95% confidence intervals) management costs were £782 (£611, £981) for stage 0, £946 (£776, £1,145) for stage 1a, £4,729 (£4,532, £4,944) for stage 1b/2, £3,719 (£3,213, £4,150) for macroscopic 3, and £2,827 (£2,757, £2,904) for microscopic 3. Mean SACT costs were £65,289 (£50,581, £74,521) for stage 3 and £134,065 (£115,787, £152,938) for stage 4. Conclusions. Our new approach to eliciting and combining estimates balances practical and theoretical considerations. We illustrate how this method produces estimates of the costs, and associated uncertainty, of the diagnosis, management, and SACT for melanoma by stage of disease in the absence of readily accessible diagnosis, management, and prescribing data. These cost estimates were not validated against registry data. Highlights Linked diagnosis, primary surgical management (hereafter “management”), and prescribing of systemic anticancer therapies (SACT) data describing the number and proportion of people with cancer offered these options are not readily available. The time to obtain data together with the time to conduct onerous analyses are a key barrier to obtaining values for use by decision analysts building economic models for evaluating new options for the early detection or prevention of cancer. We designed a simple expert elicitation exercise, completed in Excel. The exercise was designed to provide estimates of the total cost of cancer by stage of disease and crucially also to understand the extent of uncertainty around these estimates. We used melanoma as an exemplar cancer. Clinical experts (consultant dermatologists, surgeons, and oncologists) estimated the proportions of people offered possible diagnostic, management, or SACT options for melanoma (by stage of disease). Our study suggested that they found the expert elicitation exercise easy to understand and managed to complete the task. Random-effects meta-analysis used to pool the individual estimates indicated substantial heterogeneity across experts’ estimates of most elicited proportions. This finding suggested wide variation in possible approaches to the diagnosis, management, and use of SACT for melanoma.
Background . Nonpharmaceutical interventions (NPIs) were critical for mitigating COVID-19 transmission, yet their social and economic costs generated substantial public resistance. We aimed to quantify US adults’ acceptance of key NPIs. Methods . We conducted a national discrete-choice experiment between May and December 2024 among a quota-based panel of US adults, considering 6 NPIs: mask mandates, reduced public transit capacity, school closures with/without remote learning, closure of crowded indoor venues, and closure of nonessential businesses (e.g., restaurants). Scenarios varied by COVID-19 infection risk and health care restrictions. Results . A total of 5,047 adults participated, with age, gender, race, and income distributions reflecting those of the US population. Participants considered mask mandates, reduced transit capacity, and school closures with remote learning acceptable even if they do not reduce cases. School closures without remote learning and closure of nonessential businesses were acceptable only if they reduced cases by at least 75 per 100 persons within 1 mo. Restricted access to primary and optional care was strongly objected to. Male participants, Republicans, Independents, adults ≥65 y, and those without chronic conditions showed the lowest acceptance of NPIs regardless of projected benefit. Female participants, younger adults, those living with children, and those with chronic conditions were more receptive. Still, they required a reduction of >50 cases per 100 persons to accept school closures without remote learning and closure of nonessential businesses. Limitations . Temporal distancing may have affected participants’ recollection of their experiences with NPIs. Conclusions . US adults displayed heterogeneous but generally high thresholds for accepting school closures without remote learning, closure of nonessential businesses, and health care restrictions. These findings can guide pandemic preparedness plans that balance public acceptance with disease-control goals. Highlights What levels of COVID-19 risk reduction were required for US adults to accept nonpharmaceutical interventions (NPIs) such as school or business closures? In a discrete-choice experiment of 5,047 US adults, mask mandates and other moderate NPIs were broadly acceptable even if they do not reduce cases. School closures without remote learning and closure of nonessential businesses were acceptable only if they reduced infections by ≥75 per 100 persons per month. Our survey participants did not find pandemic response plans that rely on disruptive NPIs acceptable but found indoor mask mandates and school closures with remote learning acceptable, independent of their impact on infection risk.
Background. Policies on telehealth coverage and reimbursement have been evolving. To support national decisions on telehealth use and delivery, this study assessed patient preferences on care delivery modalities and value of time for primary care in outpatient settings. Design. Discrete choice experiments were designed to calibrate care visits by 1 price attribute and 3 nonprice attributes: delivery mode (phone, video, in-person), time costs, and waiting time. Mixed logistic regression models were used to estimate patient preferences and willingness to pay (WTP). Sample. A total of 110 respondents were recruited from a state representative cohort in the Midwestern United States with oversampling of Black and Hispanic minorities. Results. Patients were willing to pay $23.2 (95% confidence interval [CI] 13.3, 37.9) for a primary care visit and $30.3 extra (95% CI 21.1, 44.0) for in-person care but discounted the value of care by $8.7 for a video visit and $21.6 for a phone visit. Reducing time costs from 45 to 30 min increased patients’ WTP by $4.0 (1.8, 7.3). Patients’ WTP was $4.1 higher (or $2.2) if care visits were scheduled on the same day (or within 3 d) rather than in 7 d. Patients who were non-White minorities, older than 65 y, did not own a property, or were Medicaid beneficiaries valued care visits lower than the average ($11–$18 v. $23) but valued same-day visits more ($6.2–$6.3 v. $4.1). Conclusions. Patients discounted care delivered by phone or video visits but preferred to have such options for flexibility and timely access. The benefits of telehealth such as reducing time costs and providing timely care access increased its desirability and the potential to avoid missed or delayed care for preventive and care maintenance purposes. Highlights Patients are willing to pay about $23 for a typical primary care visit on prevention and maintenance. Patients discount video or phone visits by $8 to $20 but prefer to have such options for flexibility and timely access. Patients are willing to pay $4 to $6 for shorter time cost obtaining care or same-day timely access to care. Non-White minorities and older and low-income patients have lower valuation for a care visit but a higher price premium for same-day visits than the general population, suggesting high potential of telehealth to help avoid missed or delayed care for these subpopulations.
Introduction. Cost-effectiveness analyses are vital in guiding decisions on treatment reimbursement. Natural history models are central to these, enabling the estimation of long-term costs and quality-adjusted life-years (QALYs) in the absence of lifetime trial data. Rare disease data are often scarce, resulting in disease progression being estimated through clinical assumptions. This study aims to evaluate how different modeling approaches influence cost-effectiveness estimates in rare disease health technology assessments (HTAs), using Duchenne muscular dystrophy (DMD) as a case study. Methods. A published economic model was used to compare 2 approaches for estimating disease progression: an assumption-based method relying on clinical plausibility and data-driven methods using data from 1,005 patients with DMD across 8 studies. Transition probabilities were estimated assuming increasing flexibility of study heterogeneity and compared with a simulated treatment cohort. Models were evaluated by comparing incremental cost-effectiveness ratios (ICERs) across approaches. No gold standard exists, so the plausibility of predictions was evaluated by comparing survival and disease progression estimates to published milestones. Results. Results showed that although the assumption-based model was clinically plausible, it predicted higher QALY gains (0.77) and lower ICERs (£1.96M per QALY) than data-driven methods did, which estimated QALY gains of 0.25, 0.26, 0.27, and 0.28 and ICERs of £6.2M, £6.2M, £5.8M, and £5.7M per QALY for the least to most flexible models, respectively. Limitations. No covariate effects or updated cost and utility data were incorporated, as the study purpose was a methodological comparison between approaches. Analyses were deterministic not probabilistic. Conclusions and Implications. This study emphasizes the critical role of model selection for HTA in rare diseases, showing that cost-effectiveness estimates from robust data-driven approaches can differ from clinically plausible assumption-based models. Highlights The choice of a natural history modeling method can drastically alter the cost-effectiveness results in rare disease evaluations. A case study in Duchenne muscular dystrophy demonstrates how different modeling approaches yield divergent cost-effectiveness outcomes. Assumption-based models, even when clinically plausible, may underestimate measures of cost-effectiveness and result in less reliable guidance for decision makers. Data-driven models using real-world patient data provide more reliable estimates for health technology assessment (HTA). This study offers practical guidance for analysts and HTA bodies on selecting robust modeling approaches in rare disease contexts.
Background. Arthritis is a common chronic condition among older adults in Canada. Shared decision making (SDM) regarding medical cannabis (MC) use has been anticipated to result in improved health outcomes. However, the practice of SDM for MC remains limited. Few studies addressed what clinicians need to decide with their patients about MC. We explored clinicians’ perspectives on what determines their decisional needs to participate in MC SDM with older adults experiencing arthritis. Methods. In-depth, semi-structured interviews using purposive sampling were conducted online. An interview guide based on the Ottawa Decision Support Framework (ODSF) was used. Recordings were transcribed verbatim, and the data were analyzed deductively and inductively using reflexive thematic analysis. Results. Semi-structured interviews with 12 participants (33% pharmacists, 25% family physicians, 25% rheumatologists, and 17% registered nurses) were conducted. Three main themes were constructed to summarize determinants of clinicians’ needs to be able to participate in MC-related SDM: 1) perception of the decision, 2) perception of others, and 3) decisional conflict. Conclusion. While gaps in evidence remain a critical concern affecting inadequate knowledge and contributing to decision-making needs, findings suggest that a tailored decision support intervention (DSI) would strengthen clinicians’ self-efficacy, clarify role expectations, and attend to the interpersonal dynamics that shape MC SDM. To enhance usability, a DSI should be developed to address clinicians’ decisional needs while remaining responsive to workflow constraints. Highlights Using the Ottawa Decision Support Framework, this study explored clinicians’ decisional needs regarding shared decision making about medical cannabis (MC) use for arthritis. Findings highlight gaps that hinder clinicians from engaging in open discussions, leaving many older adults to make MC decisions without professional guidance. Addressing clinicians’ decisional needs is essential for developing decision support interventions and policies that ensure safe, informed care.
Introduction. Telemedicine may improve access to care for children, but whether telemedicine alters decision-making processes is not known. We aimed to describe differences in shared decision making (SDM) in telemedicine and in-person pediatric primary care visits with children with chronic conditions. Methods. We conducted qualitative analyses of primary care visits video recorded across 6 pediatric primary care practices. Telemedicine visits were matched to in-person visits on age, diagnosis, timing of visit, and clinical site. Each recording was summarized by a trained reviewer. The summaries were then coded and analyzed using a framework approach. Codes were organized into a matrix to facilitate thematic analyses both within and across visits and visit types. Results. We analyzed 16 telemedicine and 18 in-person visits with 34 unique patients and 12 unique clinicians. The samples of telemedicine and in-person visits were similar in distribution across patient and parent characteristics. Most of the visits discussed attention-deficit/hyperactivity disorder. Thematic analysis identified 2 major themes. First, employment of SDM processes do not differ by visit modality. Across constructs of team talk, options talk, and decision talk, optimal SDM processes were lacking in both visit modalities. Second, visit modality affects the patient’s decision engagement. Patients were physically present during all in-person visits but only briefly present during most telemedicine visits, limiting their opportunities to engage in decision making. Conclusion. Our study did not identify qualitative differences in SDM across the mode of pediatric primary care delivery: telemedicine versus in person. These data may alleviate concerns about potential negative effects of telemedicine on communication in pediatric primary care visits for chronic conditions. However, there is a need to improve shared decision making across visit types in pediatric primary care. Highlights Our study demonstrates that the mode of pediatric primary care delivery does not affect the use of SDM in primary care visits for pediatric chronic conditions. There are opportunities to improve SDM across both care modalities. Future work should consider how approaches to increasing SDM and family decision engagement may differ by visit modality.
Background. Parents of children diagnosed with attention-deficit/hyperactivity disorder (ADHD) often encounter complex treatment decisions, especially the initiation of pharmacologic therapy. While evidence-based decision aids facilitate informed, values-congruent choices, tools remain limited outside English-speaking settings. Objective. This study aimed to develop and evaluate the feasibility and acceptability of a Hebrew-language decision aid intended to support parents in making informed decisions regarding the initiation of ADHD medication for their children. Methods. The development process adhered to the International Patient Decision Aid Standards and was guided by the Ottawa Decision Support Framework. Acceptability testing involved 20 stakeholders using a mixed-methods questionnaire. Field testing used a pre/post design with 38 parents of children recently diagnosed with ADHD. Outcomes, measured using validated instruments, were knowledge, decisional conflict, decision self-efficacy, decision preparedness, and preference about SDM. Results. Most participants found the decision aid to be clear, well-structured, and easy to understand. All (100%) agreed it would be helpful for others facing similar decisions. Compared with baseline, post–decision aid parental knowledge significantly increased (67.4 to 84.7 out of 100; P < 0.001) and decisional conflict decreased (29.8 to 20.8 out of 100; P = 0.01). Decision self-efficacy increased from 83.6 to 90.0 out of 100 ( P = 0.09). Participants reported high decision preparedness (mean = 89.5 of 100), and 45% preferred shared decision making with the physician. Conclusions. The systematically developed decision aid about ADHD treatments demonstrated feasibility and acceptability. It showed potential to enhance informed, value-based parental decision making; reduce decisional conflict; and facilitate shared decision making in pediatric clinical care. Highlights A decision aid was developed to support parents in making ADHD medication decisions. The decision aid significantly increased parents’ knowledge about ADHD and treatment options and reduced parents’ decisional conflict. Stakeholders, including parents and health care professionals, reported that the decision aid was acceptable. The decision aid prepared parents for shared decision making within pediatric ADHD care.
Background. Growing evidence indicates that fecal hemoglobin (f-Hb) concentrations from prior negative fecal immunochemical tests (FITs) can predict the future detection of colorectal cancer (CRC) and advanced neoplasia. As a result, there are opportunities for risk-based screening tailored to prior FIT outcomes. However, current CRC screening decision models do not explicitly simulate quantitative f-Hb concentrations. They simulate only a binary positive or negative test result based on the sensitivity and specificity of the underlying lesions, which makes it impossible to evaluate risk-based screening by f-Hb with these models. Methods. We extended our well-established MISCAN-Colon microsimulation model with a module that simulates quantitative f-Hb concentrations based on age, sex, and the presence of colorectal lesions. This module was based on a zero-inflated negative binomial mixed-effect model and calibrated and validated using observational data from the Dutch national CRC screening program between 2014 and 2020. The new MISCAN-Colon was then applied to a case study, in which we compared the effectiveness of uniform biennial screening intervals versus risk-based intervals (3 y after a FIT result of 0 µg/g, 2 y after >0–15 µg/g, and 1 y after >15–46.9 µg/g). Results. The updated model closely reflected real-world results, including positivity rate (observed: 4.7%, model: 4.3%), CRC detection rates (observed: 0.3%, model: 0.4%), f-Hb distributions, and longitudinal patterns across 3 screening rounds. Compared with biennial screening, the risk-based strategy resulted in slightly more CRC cases (+5%) and deaths (+11%) but required substantially fewer FITs (−28%) and colonoscopies (−13%). Conclusions. Integrating the f-Hb module into MISCAN-Colon enables explicit modeling of f-Hb concentrations and facilitates the evaluation of risk-based screening by prior f-Hb concentrations. Highlights In colorectal cancer screening (CRC), risk-based screening using fecal hemoglobin (f-Hb) from prior screening rounds has been suggested to improve the balance between the benefits and harms of screening. However, the added value of this approach has never been quantified because none of the existing decision models for CRC screening simulate f-Hb concentrations. We used data from the Dutch national CRC screening program to extend the widely used MISCAN-Colon microsimulation model to explicitly simulate an individual’s f-Hb concentration based on sex, age, and the presence of colorectal lesions. This model accurately reproduced cross-sectional and longitudinal associations between f-Hb concentration and screen-detected colorectal lesions, and model estimates showed that risk-based screening using f-Hb concentrations could be more efficient than current uniform screening.
Introduction. The recent expansion of treatment options for older adults with blood cancers has increased the necessity for understanding patient preferences and values to inform treatment decision making. Currently, no values elicitation measures have been validated for clinical use in this population. Values-HM (Values elicitation measure for Hematologic Malignancies) is a novel values elicitation measure based on best–worst scaling. It was developed specifically for older adults with blood cancers involving multiple stakeholders including patients, caregivers, and clinicians. Objective. The objective of this study was to test the preliminary validity, reliability, and acceptability of this measure for clinical use based on US Food and Drug Administration and International Society for Pharmacoeconomics and Outcomes Research guidance. Results. Twenty-nine adults aged ≥60 y with newly diagnosed lymphoma, leukemia, myelodysplastic syndrome, and multiple myeloma were enrolled. Most patients were White (97%). Most patients (88%) felt that the measure was relevant to them, showed their real preferences (73%), and was acceptable to clarify their preferences (68%). The measure demonstrated discriminant validity of importance scores and convergent validity for most patients (77%) with a ranking exercise of the treatment values. Cognitive interviews with 10 participants suggested question comprehension, outcome understanding, and appropriate judgment. Data from 18 participants who completed the measure more than once provide some evidence for the reliability of the measure to capture repeated values over time. Conclusions. These initial data suggest that Values-HM may be acceptable to patients and valid and reliable to capture patient values. Additional data are needed to confirm these findings in a larger and more diverse sample. This trial was registered at www.clinicaltrials.gov as #NCT05061095. Highlights Values-HM is a novel values elicitation measure designed based on best–worst scaling for adults with blood cancers. Values-HM appears to be acceptable to patients and may be valid and reliable to capture patient values in clinical care.
Background. The Medicare Part D program provides stand-alone prescription drug insurance to US residents aged ≥65 y and those with disabilities. Annually, they can choose to stay with their plan or to switch to one of the many alternative plans. Behavioral decision-making research has proposed various strategies for tackling such complex choices. Here, we interviewed Medicare Part D beneficiaries to understand their real-world experiences with making their plan choices and examined quotes from those who stayed with their first plan and those who had switched. Method. In July 2023, we conducted phone interviews with 20 Medicare Part D beneficiaries aged 57 to 86 y who were recruited through the US-wide Understanding America Study. Results. Eleven interviewees had stayed with their first plan, and 9 had switched. Stayers and switchers applied similar strategies, with mixed success. Using decision-support tools such as the Medicare plan-finder Web site, booklet, and mailers could be time-consuming and confusing. Recommendations were taken from insurance brokers, agents, and pharmacists, often without questioning their advice. Interviewees varied widely in the amount of information they considered, with some using heuristics to reduce their choice set and others considering only 1 recommended option. Few beneficiaries made annual plan comparisons, and while some deferred choices until necessary, others did not act even after being confronted with plan changes or negative experiences – due to knowledge and time constraints. Discussion. Medicare beneficiaries use various strategies known from the behavioral decision-making research literature for tackling complex choices between Medicare Part D stand-alone plans. Each strategy posed its own challenges. We discuss our findings in light of the behavioral decision-making literature and recommend policies for helping beneficiaries with their plan choices. Highlights We interviewed Medicare Part D beneficiaries about how they tackled drug insurance plan choices with many options and complicated features. Interviewees reported using decision-support tools, seeking help, applying heuristics, and relying on choice deferral, but with mixed success. Qualitatively, similar experiences were described by interviewees who stayed with their initial plan and those who had switched. We recommend policies for addressing the difficulties beneficiaries had in applying these strategies.
Background. Deciding whether to participate in a radiotherapy trial is complex for patients with head and neck cancer. Standard patient information leaflets meet ethical requirements but do not provide balanced comparisons, value clarification, or structured decision support. Few patient decision aids (PtDAs) have been developed for clinical trial participation. Objective. To develop a PtDA to support decisions about participation in a randomized trial comparing proton versus photon radiotherapy (DAHANCA 35). Methods. Following the Medical Research Council framework for complex interventions, an iterative development process incorporating elements of user-centered design was undertaken, informed by the Ottawa Decision Support Framework (ODSF), International Patient Decision Aid Standards (IPDAS), and Measure of User- and Human-Centred Design for Personal Health Tools (UCD-11). Five steps were conducted: 1) scoping informed by 3 preceding studies and a workshop with clinicians, 2) drafting of an initial prototype, 3) iterative refinement in a multidisciplinary development group, 4) alpha testing using think-aloud interviews with patients ( n = 5) and clinicians ( n = 8), and 5) finalization. Data from workshops, written feedback, group discussions, and interviews informed revisions. Results. The final iteration of the PtDA provides balanced, plain-language information; value clarification; and decision support regarding participation in DAHANCA 35, including an explicit presentation of the decision between standard photon radiotherapy and the trial option. Visual elements, a comparison table, a decision tree, and tools from the Ottawa Personal Decision Guide were integrated to support comprehension and deliberation. Alpha testing confirmed usability and informed refinements that improved clarity and navigation. Conclusions. This iterative development process, incorporating elements of user-centered design, resulted in a PtDA that addressed decision-making needs within the trial context. Future research will assess the feasibility and implementation of this approach. The development approach may serve as a foundation for adapting PtDAs to other radiotherapy clinical trials. Highlights A patient decision aid (PtDA) was developed to support individuals with head and neck cancer in deciding whether to participate in a proton radiotherapy trial. The PtDA was developed through an iterative, theory-informed, and user-centered process with input from clinicians and trial-eligible patients. The PtDA provides structured, balanced information and explicit comparisons between standard photon radiotherapy and the trial option. This development approach may be adapted to support decision making in other radiotherapy trials and clinical trial contexts.
