Stated-Preference Survey Design and Testing in Health Applications.

Following the conceptualization of a well-formulated and relevant research question, selection of an appropriate stated-preference method, and related methodological issues, researchers are tasked with developing a survey instrument. A major goal of designing a stated-preference survey for health applications is to elicit high-quality data that reflect thoughtful responses from well-informed respondents. Achieving this goal requires researchers to design engaging surveys that maximize response rates, minimize hypothetical bias, and collect all the necessary information needed to answer the research question. Designing such a survey requires researchers to make numerous interrelated decisions that build upon the decision context, selection of attributes, and experimental design. Such decisions include considering the setting(s) and study population in which the survey will be administered, the format and mode of administration, and types of contextual information to collect. Development of a survey is an interactive process in which feedback from respondents should be collected and documented through qualitative pre-test interviews and pilot testing. This paper describes important issues to consider across all major steps required to design and test a stated-choice survey to elicit patient preferences for health preference research.

Patient Preferences in Pulmonary Arterial Hypertension, a Latent Class Analysis to Identify Preference Heterogeneity.

OBJECTIVES: Pulmonary arterial hypertension (PAH) is a chronic, progressive disease of the pulmonary circulation characterized by vascular remodeling that, if untreated, can lead to right heart dysfunction and death. This analysis measured heterogeneity in patient preferences for PAH-specific treatment regimens. METHOD: Adult patients with PAH with slight to marked limitations during physical activity were recruited through a patient organization in Germany. Participants completed an online best-worst scaling case 3 survey. Patients chose among 3 hypothetical treatment profiles defined by 6 benefits and risks at varying levels. Participants completed 12 choice tasks. Preference heterogeneity was assessed using latent class analysis. RESULTS: A total of 83 participants (76% female) completed the survey. Best-fit model revealed 4 classes. Class 1 (19% of participants) assigned importance to multiple attributes particularly side effects, class 2 (34%) to physical activity limitations, class 3 (30%) to survival and physical activity limitations, and class 4 (17%) to survival. No differences in sociodemographic characteristics were observed across classes. Compared with other classes, class 4 was most likely to report having marked physical activity limitations (79%) and needing daily help (100%), while considering higher daily activity levels to be ordinary (walking >1 km [71%] or climbing several flights of stairs [50%]). CONCLUSION: This first patient preference study in a PAH population suggests that physical activity limitations in addition to survival matter most to patients; however, preference heterogeneity between groups of patients was observed. Patient preferences should be considered in treatment decision making to better balance patient's expectations regarding the known risk-benefit ratio of treatment.

Research Priorities to Increase Confidence in and Acceptance of Health Preference Research: What Questions Should be Prioritized Now?

BACKGROUND AND OBJECTIVE: There has been an increase in the study and use of stated-preference methods to inform medicine development decisions. The objective of this study was to identify prioritized topics and questions relating to health preferences based on the perspective of members of the preference research community. METHODS: Preference research stakeholders from industry, academia, consultancy, health technology assessment/regulatory, and patient organizations were recruited using professional networks and preference-targeted e-mail listservs and surveyed about their perspectives on 19 topics and questions for future studies that would increase acceptance of preference methods and their results by decision makers. The online survey consisted of an initial importance prioritization task, a best-worst scaling case 1 instrument, and open-ended questions. Rating counts were used for analysis. The best-worst scaling used a balanced incomplete block design. RESULTS: One hundred and one participants responded to the survey invitation with 66 completing the best-worst scaling. The most important research topics related to the synthesis of preferences across studies, transferability across populations or related diseases, and method topics including comparison of methods and non-discrete choice experiment methods. Prioritization differences were found between respondents whose primary affiliation was academia versus other stakeholders. Academic researchers prioritized methodological/less studied topics; other stakeholders prioritized applied research topics relating to consistency of practice. CONCLUSIONS: As the field of health preference research grows, there is a need to revisit and communicate previous work on preference selection and study design to ensure that new stakeholders are aware of this work and to update these works where necessary. These findings might encourage discussion and alignment among different stakeholders who might hold different research priorities. Research on the application of previous preference research to new contexts will also help increase the acceptance of health preference information by decision makers.

Patient Preferences for Lung Cancer Interception Therapy.

Importance: Interception therapy requires individuals to undergo treatment to prevent a future medical event, but little is known about preferences of individuals at high risk for lung cancer and whether they would be interested in this type of treatment. Objective: To explore preferences of individuals at high risk for lung cancer for potential interception therapies to reduce this risk. Design, Setting, and Participants: This survey study used a discrete-choice experiment and included hypothetical lung cancer interception treatments with 4 attributes: reduction in lung cancer risk over 3 years, injection site reaction severity, nonfatal serious infection, and death from serious infection. Respondents were assigned to a baseline lung cancer risk of 6%, 10%, or 16% over 3 years. The discrete-choice experiment was administered online (July 13 to September 6, 2022) to US respondents eligible for lung cancer screening according to US Preventive Services Task Force guidelines. Participants included adults aged 50 to 80 years with at least a 20 pack-year smoking history. Statistical analysis was performed from September to December 2022. Main Outcomes and Measures: Attribute-level preference weights were estimated, and conditional relative attribute importance, maximum acceptable risks, and minimum acceptable benefits were calculated. Characteristics of respondents who always selected no treatment were also explored. Results: Of the 803 survey respondents, 495 (61.6%) were female, 138 (17.2%) were African American or Black, 55 (6.8%) were Alaska Native, American Indian, or Native American, 44 (5.5%) were Asian or Native Hawaiian or Other Pacific Islander, 104 (13.0%) were Hispanic, Latin American, or Latinx, and 462 (57.5%) were White, Middle Eastern or North African, or a race or ethnicity not listed; and mean (SD) age was 63.0 (7.5) years. Most respondents were willing to accept interception therapy and viewed reduction in lung cancer risk as the most important attribute. Respondents would accept a greater than or equal to a 12.0 percentage point increase in risk of nonfatal serious infection if lung cancer risk was reduced by at least 20.0 percentage points; and a greater than or equal to 1.2 percentage point increase in risk of fatal serious infection if lung cancer risk was reduced by at least 30.0 percentage points. Respondents would require at least a 15.4 (95% CI, 10.6-20.2) percentage point decrease in lung cancer risk to accept a 12.0 percentage point increase in risk of nonfatal serious infection; and at least a 23.1 (95% CI, 16.4-29.8) percentage point decrease in lung cancer risk to accept a 1.2 percentage point increase in risk of death from serious infection. Respondents who were unwilling to accept interception therapy in any question (129 [16.1%]) were more likely to be older and to currently smoke with no prior cessation attempt, and less likely to have been vaccinated against COVID-19 or examined for skin cancer. Conclusions and Relevance: In this survey study of individuals at high risk of lung cancer, most respondents were willing to consider interception therapy. These results suggest the importance of benefit-risk assessments for future lung cancer interception treatments.

Maximum Acceptable Risk Estimation Based on a Discrete Choice Experiment and a Probabilistic Threshold Technique.

OBJECTIVE: We aimed to empirically compare maximum acceptable risk results estimated using both a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT). METHODS: Members of the UK general public (n = 982) completed an online survey including a DCE and a PTT (in random order) measuring their preferences for preventative treatment for rheumatoid arthritis. For the DCE, a Bayesian D-efficient design consisting of four blocks of 15 choice tasks was constructed including six attributes with varying levels. The PTT used identical risk and benefit attributes. For the DCE, a panel mixed-logit model was conducted, both mean and individual estimates were used to calculate maximum acceptable risk. For the PTT, interval regression was used to calculate maximum acceptable risk. Perceived complexity of the choice tasks and preference heterogeneity were investigated for both methods. RESULTS: Maximum acceptable risk confidence intervals of both methods overlapped for serious infection and serious side effects but not for mild side effects (maximum acceptable risk was 32.7 percent-points lower in the PTT). Although, both DCE and PTT tasks overall were considered easy or very easy to understand and answer, significantly more respondents rated the DCE choice tasks as easier to understand compared with those who rated the PTT as easier (7-percentage point difference; p < 0.05). CONCLUSIONS: Maximum acceptable risk estimate confidence intervals based on a DCE and a PTT overlapped for two out of the three included risk attributes. More respondents rated the DCE as easier to understand. This may suggest that the DCE is better suited in studies estimating maximum acceptable risk for multiple risk attributes of differing severity, while the PTT may be better suited when measuring heterogeneity in maximum acceptable risk estimates or when investigating one or more serious adverse events.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making. We provide the ISPOR Roadmap for Patient Preferences in Decision Making that invites patient-preference researchers to work with decision makers, patients and patient groups, and other stakeholders to ensure that studies are useful and impactful. The ISPOR Roadmap consists of 5 key elements: (1) context, (2) purpose, (3) population, (4) method, and (5) impact. In this report, we define these 5 elements and provide good practices on how patient-preference researchers and others can actively contribute to increasing the usefulness and impact of patient-preference studies in decision making. We also present a set of key questions that can support researchers and other stakeholders (eg, funders, reviewers, readers) to assess efforts that promote the ongoing impact (both intended and unintended) of a particular preference study and additional studies in the future.

Patient preferences for mitral valve regurgitation treatment: a discrete choice experiment.

INTRODUCTION: This study aimed to quantify patients' preferences for benefits and risks associated with treating degenerative mitral regurgitation (DMR) via open heart surgical repair versus a beating heart surgical approach. METHODS: A D-efficient main effects discrete choice experiment (DCE) survey with 10 choice tasks that involved trade-offs across six attributes varying between two and four levels each (procedure invasiveness, recovery intensity, risk of disabling stroke, risk of new onset atrial fibrillation, risk of symptom reappearance and risk of reintervention) was administered online to either clinically confirmed (n = 30) or self-reported DMR (n = 88) patients recruited from either cardiovascular clinics or online clinical patient databases. The error component logit (ECL) analysis combined both patient cohorts after performing a Swait-Louviere scale test. Patient trade-offs across attributes were estimated in relation to either an open-heart surgery (OHS) treatment profile or a beating heart approach. RESULTS: Patients demonstrated clear preferences across all attributes for the beating heart treatment. 76.0% (95% CI: 68.1,83.9) of patients would prefer a 'beating heart' intervention relative to the 'open heart' approach despite the higher likelihood of symptom recurrence and reintervention. In exchange for the combined net benefits associated with a 'beating heart' treatment, on average, participants were willing to accept a maximum acceptable risk (MAR) of 34.6 percentage points (95% CI: 23.8,45.4) for increased risk of symptom reappearance or 22.6 percentage points (95% CI: 14.7,30.4) increased risk of reintervention. CONCLUSION: This study of US adults with DMR provides quantitative measures of risk tolerance for tradeoffs related to repair by a beating heart approach relative to conventional open-heart surgery (standard of care). These results may inform DMR treatment choices from regulatory agencies, payers, clinicians, and patients considering a beating heart repair or treatments with similar attributes as potential new alternatives to conventional surgery.

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance.

OBJECTIVES: To quantify tolerance to risks of preventive treatments among first-degree relatives (FDRs) of patients with rheumatoid arthritis (RA). METHODS: Preventive treatments for RA are under investigation. In a preference survey, adult FDRs assumed a 60% chance of developing RA within 2 years and made choices between no treatment and hypothetical preventive treatment options with a fixed level of benefit (reduction in chance of developing RA from 60% to 20%) and varying levels of risks. Using a probabilistic threshold technique, each risk was increased or decreased until participants switched their choice. Perceived risk of RA, health literacy, numeracy, Brief Illness Perception Questionnaire and Beliefs about Medicines Questionnaire-General were also assessed. Maximum acceptable risk (MAR) was summarised using descriptive statistics. Associations between MARs and participants' characteristics were assessed using interval regression with effects coding. RESULTS: 289 FDRs (80 male) responded. The mean MAR for a 40% reduction in chance of developing RA was 29.08% risk of mild side effects, 9.09% risk of serious infection and 0.85% risk of a serious side effect. Participants aged over 60 years were less tolerant of serious infection risk (mean MAR ±2.06%) than younger participants. Risk of mild side effects was less acceptable to participants who perceived higher likelihood of developing RA (mean MAR ±3.34%) and more acceptable to those believing that if they developed RA it would last for a long time (mean MAR ±4.44%). CONCLUSIONS: Age, perceived chance of developing RA and perceived duration of RA were associated with tolerance to some risks of preventive RA therapy.

Development of a severity scale to assess chronic lung disease after extremely preterm birth.

OBJECTIVE: Chronic lung disease of prematurity (CLDP) is a frequent complication of prematurity. We aimed to identify what clinicians believe are the most important factors determining the severity of CLDP in extremely preterm infants (<28 weeks gestational age) after discharge from the neonatal intensive care unit (NICU) through 12 months corrected age (CA), and to evaluate how these factors should be weighted for scoring, to develop a CLDP severity scale. STUDY DESIGN: Clinicians completed a three-round online survey utilizing Delphi methodology. Clinicians rated the importance of various factors used to evaluate the severity of CLDP, from 0 (not at all important) to 10 (very important) for the period between discharge home from the NICU and 12 months CA. Fourteen factors were considered in Round 1; 13 in Rounds 2 and 3. The relative importance of factors was explored via a set of 16 single-profile tasks (i.e., hypothetical patient profiles with varying CLDP severity levels). RESULTS: Overall, 91 clinicians from 11 countries who were experienced in treating prematurity-related lung diseases completed Round 1; 88 completed Rounds 2 and 3. Based on Round 3, the most important factors in determining CLDP severity were mechanical ventilation (mean absolute importance rating, 8.89), supplemental oxygen ≥2 L/min (8.49), rehospitalizations (7.65), and supplemental oxygen <2 L/min (7.56). Single-profile tasks showed that supplemental oxygen had the greatest impact on profile classification. CONCLUSION: The most important factors for clinicians assigning CLDP severity during infancy were mechanical ventilation, supplemental oxygen ≥2 L/min, and respiratory-related rehospitalizations.

Do people have differing motivations for participating in a stated-preference study? Results from a latent-class analysis.

BACKGROUND: Researchers and policy makers have long suspected that people have differing, and potentially nefarious, motivations for participating in stated-preference studies such as discrete-choice experiments (DCE). While anecdotes and theories exist on why people participate in surveys, there is a paucity of evidence exploring variation in preferences for participating in stated-preference studies. METHODS: We used a DCE to estimate preferences for participating in preference research among an online survey panel sample. Preferences for the characteristics of a study to be conducted at a local hospital were assessed across five attributes (validity, relevance, bias, burden, time and payment) and described across three levels using a starring system. A D-efficient experimental design was used to construct three blocks of 12 choice tasks with two profiles each. Respondents were also asked about factors that motivated their choices. Mixed logistic regression was used to analyze the aggregate sample and latent class analysis identified segments of respondents. RESULTS: 629 respondents completed the experiment. In aggregate "study validity" was most important. Latent class results identified two segments based on underlying motivations: a quality-focused segment (76%) who focused most on validity, relevance, and bias and a convenience-focused segment (24%) who focused most on reimbursement and time. Quality-focused respondents spent more time completing the survey (p < 0.001) and were more likely to identify data quality (p < 0.01) and societal well-being (p < 0.01) as motivations to participate. CONCLUSIONS: This information can be used to better understand variability in motivations to participate in stated-preference surveys and the impact of motivations on response quality.

Clinical characteristics and burden of illness in patients with hereditary angioedema: findings from a multinational patient survey.

BACKGROUND: Hereditary angioedema (HAE) is a rare, debilitating, genetic disease characterized by unpredictable, recurrent, and potentially fatal swelling of the skin and mucous membranes. We conducted a noninterventional, cross-sectional, web-based survey of patients with a self-reported diagnosis of HAE type 1/2 in Australia, Austria, Canada, France, Germany, Spain, Switzerland, and the United Kingdom to gain a comprehensive real-world understanding of the characteristics of HAE and its burden from the perspective of the patient. The survey included questions on clinical and demographic characteristics, burden of disease, and treatment. Instruments used to measure patient-reported outcomes included the Angioedema Quality of Life questionnaire (AE-QoL), 12-Item Short-Form Health Survey (SF-12v2), Angioedema Control Test (AECT), Hospital Anxiety and Depression Scale (HADS), and Work Productivity and Impairment questionnaire (WPAI). Data were analyzed with descriptive statistics. RESULTS: A total of 242 patients (67.4% female; mean [range] age 43.8 [18-92] years) completed the survey. The mean (SD) age at first symptoms was 11.5 (8.9) years, while diagnosis occurred at 20.8 (13.2) years. Patients reported a mean (SD) of 12.5 (14.1) attacks in the past 6 months. The most recent attack occurred within the past month in 79.7% of patients; most were of moderate severity, 6.6% affected the larynx, 21.9% lasted ≥ 3 days, and 76.4% were treated with on-demand medication. Hospitalizations and emergency/urgent care visits were highest for patients with more attacks. At the time of the survey, 62.4% of patients were using long-term prophylaxis, including 34.4% using androgens. Moderate to severe anxiety and depression were reported in 38.0% and 17.4% of patients, respectively, as measured using the HADS. The severity of anxiety and depression was associated with poorer quality of life and productivity, measured using the AECT (mean overall score 8.00 [moderate perceived disease control]), AE-QoL, WPAI, and SF-12v2. Scores for AECT, AE-QoL, and WPAI were also worse with a higher number of attacks. CONCLUSIONS: This survey study of a broad international sample of patients with HAE showed that despite the availability of on-demand treatment and long-term prophylaxis for the prevention of attacks, patients across a wide geographical area continue to have high disease activity, likely due to restrictions in the availability of medications or incorrect use. Subsequently, significant disease burden, including impaired quality of life and mental health and decreased productivity, was evident. Increased patient education and access to newer, more effective therapies are needed.

Line of therapy and patient preferences regarding lung cancer treatment: a discrete-choice experiment.

OBJECTIVE: A growing literature on patient preferences informs decisions in research, regulatory science, and value assessment, but few studies have explored how preferences vary across patients with differing treatment experience. We sought to quantify patient preferences for the benefits and risks of lung cancer treatment and test how preferences differed by line of therapy (LOT). METHODS: Preferences were elicited using a discrete choice experiment (DCE) following rigorous patient and stakeholder engagement. The DCE spanned five attributes (each with three levels): progression-free survival (PFS), short-term side effects, long-term side effects, risk of developing late-onset side effects, and mode of administration (MOA) - each defined across 3 relevant levels. A D-efficient design was used to generate 3 survey blocks of 9 paired-profile choice tasks each and respondents were asked which profile they preferred and then if they preferred to have no treatment (opt-out). A mixed logit model, controlling for opt-out, was used to estimate preferences. Preferences and trade-offs between PFS and other attributes were compared across two groups: those receiving ≤1 LOT and those receiving ≥2 LOT. RESULTS: Of the 466 participants, 42% received ≤1 LOT and 58% received ≥2 LOT. Stated preferences differed between the groups overall (p<.001) and specifically for 18 months of PFS (p<.001), moderate short-term side effects (p<.001), no long-term side effects (p=.03), and 30% chance of late-onset side effects (p=.02). Those receiving differing amounts of LOT were willing to trade different amounts of PFS to change from moderate to mild short-term side effects (p<.001), moderate to no (p<.001) and mild to no (p<.001) long-term side effects. There were also differing amounts of tradeoff acceptable between the groups for a 10% decrease in risk of late-onset side effects (p=.016), a decrease in MOA from infusion every 3 weeks to pills taken daily at any time (p=.005) and from pills taken daily without food to pills taken daily at any time (p<.001). CONCLUSION: We demonstrate differences in preferences based on experience with LOT, suggesting that patient treatment experience may have an impact on their preferences. As patient preference data become an important component of treatment decision making, preference differences should be considered when recommending therapies at different stages in the treatment journey. Understanding patient preferences regarding treatment decisions is essential to informing shared decision-making and ensuring treatment plans are consistent with patients' goals.

Comparing the Preferences of Patients and the General Public for Treatment Outcomes in Type 2 Diabetes Mellitus.

BACKGROUND: Healthcare treatments and interventions are traditionally evaluated from the societal perspective, but a more patient-centric perspective has been proposed in recent years. We sought to compare preferences of patients and the general public for treatment outcomes of type 2 diabetes using both best-worst scaling (BWS) and rating approaches. METHODS: A survey evaluating the treatment priorities for type 2 diabetes was conducted in the United States. Members of the general public and patients with type 2 diabetes were recruited from a nationally sampled panel. Participants indicated the importance of seven potential treatment outcomes (hypoglycemic events, glycated hemoglobin [A1c], weight loss, mental health, functioning, glycemic stability, and cardiovascular health) using (1) BWS case 1 and (2) a rating task. Preference differences from BWS prioritizations were explored using mixed logistic regression (BWS preference weights were probability re-scaled so that the weightings of the seven items collectively summed to 100). The consistency of scale between samples was explored using heteroskedastic conditional logistic regression of BWS data. Spearman rank correlation was used to compare standardized BWS preference weights and rating scores for each group. Both groups evaluated the BWS and rating activities using debriefing questions. RESULTS: The public and patient samples included 314 and 313 respondents, respectively. The public was on average 16 years younger than patients (48 vs 64 years, P < 0.001). In BWS, patients and the public both ranked A1c, glycemic stability, and cardiovascular health within their top three outcomes. Patients valued the outcome A1c most highly and found it twice as important as did the public (41.0 vs 20.2, P < 0.001). The public valued cardiovascular health most highly, and found it to be twice as important than did patients (31.3 vs 17.4, P < 0.001). Patients were more consistent in their preferences than the public (λ = 1.66, P = 0.01). Preferences elicited using BWS and rating approaches were highly correlated for both patients (ρ = 0.96) and the public (ρ = 0.92). Patients were more likely than the public to endorse the BWS as easy to answer (P < 0.001), easy to understand (P < 0.001), consistent with preferences (P < 0.001), and relevant (P < 0.001). Both patients and the public found the rating activity easier to answer and understand, and more consistent with their preferences, than the BWS (P < 0.001). CONCLUSIONS: We provide some of the first evidence demonstrating a difference in patient and public treatment priorities for diabetes. That patients were more consistent in their preferences than the public and found the BWS and Likert rating instruments more relevant suggests that patient priorities may be more appropriate than those of the general public in some medical decision-making contexts.

Increasing the Patient-Centeredness of Health Economics and Outcomes Research Through Patient Engagement in Core Outcome Set Development.

Core outcome sets (COS) are becoming increasingly popular in clinical research and can provide important inputs for further health economics and outcomes research (HEOR) studies. Use of standard, consistently reported outcomes can demonstrate and allow differentiation of the effectiveness and value of different treatments. Incorporating patient values during COS development increases the patient centeredness of evidence available across decision-making contexts. However, the approach to meaningful patient engagement in the COS process is evolving and poses both unique challenges and opportunities. We describe an approach to patient-centered COS development and discuss challenges and adaptations to improve engagement across COS projects. We provide examples from our experience in patient engagement for COS development using three completed COS projects. This approach includes patient engagement in terms of partnering with patient organizations, orientation and training, and the consensus process. Including COS in clinical development programs and HEOR will ensure that relevant, consistent outcomes are available for healthcare decision making and should result in faster access to high-value and novel therapies for patients. Patient-centered COS development increases the likelihood that further HEOR studies and decisions made using the COS are relevant to patients.

Patient preferences for reducing bowel adverse events following prostate radiotherapy.

BACKGROUND: The Extended Prostate Cancer Index Composite (EPIC) instrument is a commonly used patient reported outcome (PRO) tool in prostate cancer clinical trials. Summary scores for EPIC subscales are calculated by averaging patient scores for attributes (e.g., side effects), implying equal weighting of the attributes in the absence of evidence showing otherwise. METHODS: We estimated patient preferences for each of the attributes included in the bowel subscale of the EPIC instrument using best-worst (B-W) scaling among a cohort of men with prostate cancer. Patients were presented with multiple tasks in which they were asked to indicate which attribute they would find most and least bothersome at different levels of severity. Analysis utilized both (simple) B-W counts and scores to estimate patient preferences for each attribute as well as attribute levels. RESULTS: A total of 174 respondents from two institutions participated in the survey. Preference estimates for each of the five attributes included in the EPIC-26 bowel subscale showed wide variation preferences: 'losing control of bowel movements' was found to be the most bothersome attribute, with a B-W score of -0.48, followed by bowel urgency which also had negative B-W score (-0.04). Increased frequency of bowel movements was the least bothersome attribute, with a B-W score of +0.33, followed by bloody stools (+0.12), and pelvic/rectal pain (+0.06). Analysis of preference weights for attribute bother levels showed preference estimates be linear. CONCLUSIONS: We provide novel evidence on patient preferences for side effect reduction following prostate radiotherapy. Within the bowel sub-scale of the EPIC-26 short form, we found that bowel incontinence was perceived to be the most bothersome treatment effect, while increased bowel frequency was least bothersome to patients.

Analysis of Patient Preferences in Lung Cancer - Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects.

OBJECTIVE: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects. PATIENTS AND METHODS: Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration's patient preference guidance. RESULTS: A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3-4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9-2.8) and cognitive long-term effects (CI: 1.8-2.8), 0.9 months (CI: 0.4-1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4-2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS. CONCLUSION: Participants would trade PFS for changes in short-term side effects and long-term side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.

Evaluating Patient Preferences of Maintenance Therapy for the Treatment of Chronic Obstructive Pulmonary Disease: A Discrete Choice Experiment in the UK, USA and Germany.

Introduction: With increasing availability of different treatments for chronic obstructive pulmonary disease (COPD), we sought to understand patient preferences for COPD treatment in the UK, USA, and Germany using a discrete choice experiment (DCE). Methods: Qualitative research identified six attributes associated with COPD maintenance treatments: ease of inhaler use, exacerbation frequency, frequency of inhaler use, number of different inhalers used, side effect frequency, and out-of-pocket costs. A DCE using these attributes, with three levels each, was designed and tested through cognitive interviews and piloting. It comprised 18 choice sets, selected using a D-efficient experimental design. Demographics and disease history were collected and the final DCE survey was completed online by participants recruited from panels in the UK, USA and Germany. Responses were analyzed using mixed logit models, with results expressed as odds ratios (ORs). Results: Overall, 450 participants (150 per country) completed the DCE; most (UK and Germany, 97.3%; USA, 98.0%) were included in the final analysis. Based on relative attribute importance, avoidance of side effects was found to be most important (UK: OR 11.65; USA: OR 7.17; Germany: OR 11.45; all p<0.0001), followed by the likelihood of fewer exacerbations (UK: OR 2.22; USA: OR 1.63; Germany: OR 2.54; all p<0.0001) and increased ease of use (UK: OR 1.84; USA: OR 1.84; Germany: OR 1.60; all p<0.0001). Number of inhalers, out-of-pocket costs, and frequency of inhaler use were found to be less important. Preferences were relatively consistent across the three countries. All participants required a reduction in exacerbations to accept more frequent inhaler use or use of more inhalers. Conclusion: When selecting COPD treatment, individuals assigned the highest value to the avoidance of side effects, experiencing fewer exacerbations, and ease of inhaler use. Ensuring that patients' preferences are considered may encourage treatment compliance.

Cost of behavioral weight loss programs implemented in clinical practice: The POWER trial at Johns Hopkins.

Obesity presents an important public health problem that affects more than a third of the U.S. adult population and that is associated with increased morbidity, mortality, and costs. Previously, we documented that two primary care-based weight loss interventions were clinically effective. To encourage the implementation of and reimbursement for these interventions, we evaluated their relative cost-effectiveness. We performed a cost analysis of the Practice-based Opportunities for Weight Reduction (POWER) trial, a three-arm trial that enrolled 415 patients with obesity from six primary care practices. Trial participants were randomized to a control arm, an in-person support intervention, or a remote support intervention; in the two intervention arms, behavioral interventions were delivered over 24 months, in two phases. Weight loss was measured at 6, 12, and 24 months. Using timesheets and empirical data, we evaluated the cost of the in-person and remote support interventions from the perspective of a health care system delivering the interventions. A univariate sensitivity analysis was conducted to evaluate uncertainty around model assumptions. All comparisons were tested using independent t-tests. Cost of the in-person intervention was higher at 6 months ($113 per participant per month and $117 per kg lost) than the remote support intervention ($101 per participant per month and $99 per kg lost; p < .001). Costs were also higher for the in-person support intervention at 24 months ($73 per participant per month and $342 per kg lost) than for the remote support intervention ($53 per participant per month and $275 per kg lost; p < .001). In the sensitivity analyses, cost ranged from $274/kg lost to $456/kg lost for the in-person support intervention and from $218/kg to $367/kg lost for the remote support intervention. A primary care weight loss intervention administered remotely was relatively more cost-effective than an in-person intervention. Expanding the scope of reimbursable programs to include other cost-effective interventions could help ensure that a broader range of patients receive the type of support needed.

Patient preferences for attributes of tyrosine kinase inhibitor treatments for EGFR mutation-positive non-small-cell lung cancer.

Aim: EGFR-tyrosine kinase inhibitors (TKIs) vary in efficacy, side effects (SEs) and dosing regimen. We explored EGFR-TKI treatment attribute preferences in EGFR mutation-positive metastatic non-small-cell lung cancer. Materials & methods: Patients completed a survey utilizing preference elicitation methods: direct elicitation of four EGFR-TKI profiles describing progression-free survival (PFS), severe SE risk, administration; discrete choice experiment involving 12 choice tasks. Results: 90 participated. The preferred profile (selected 89% of times) had the longest PFS (18 months) and the lowest severe SE risk (5%). Patients would need compensation with ≥three-times longer PFS for severe SEs. Patients would accept ≤7 months PFS reduction for oral treatments versus intravenous. Conclusion: Patients preferred longer PFS but were willing to accept reduced PFS for more favorable SEs and dosing convenience.