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.

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.

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.

Examining Generalizability of Older Adults' Preferences for Discussing Cessation of Screening Colonoscopies in Older Adults with Low Health Literacy.

BACKGROUND/OBJECTIVES: Many older adults receive unnecessary screening colonoscopies. We previously conducted a survey using a national online panel to assess older adults' preferences for how clinicians can discuss stopping screening colonoscopies. We sought to assess the generalizability of those results by comparing them to a sample of older adults with low health literacy. DESIGN: Cross-sectional survey. SETTING: Baltimore metropolitan area (low health literacy sample) and a national, probability-based online panel-KnowledgePanel (national sample). PARTICIPANTS: Adults 65+ with low health literacy measured using a single-question screen (low health literacy sample, n = 113) and KnowledgePanel members 65+ who completed survey about colorectal cancer screening (national sample, n = 441). MEASUREMENTS: The same survey was administered to both groups. Using the best-worst scaling method, we assessed relative preferences for 13 different ways to explain stopping screening colonoscopies. We used conditional logistic regression to quantify the relative preference for each explanation, where a higher preference weight indicates stronger preference. We analyzed each sample separately, then compared the two samples using Spearman's correlation coefficient, the likelihood ratio test to assess for overall differences between the two sets of preference weights, and the Wald test to assess differences in preference weights for each individual phrases. RESULTS: The responses from the two samples were highly correlated (Spearman's coefficient 0.92, p < 0.0001). The most preferred phrase to explain stopping screening colonoscopy was "Your other health issues should take priority" in both groups. The three least preferred options were also the same for both groups, with the least preferred being "The doctor does not give an explanation." The explanation that referred to "quality of life" was more preferred by the low health literacy group whereas explanations that mentioned "unlikely to benefit" and "high risk for harms" were more preferred by the national survey group (all p < 0.001). CONCLUSION: Among two different populations of older adults with different health literacy levels, the preferred strategies for clinicians to discuss stopping screening colonoscopies were highly correlated. Our results can inform effective communication about stopping screening colonoscopies in older adults across different health literacy levels.

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.

AcroVoice: eliciting the patients' perspective on acromegaly disease activity.

PURPOSE: To determine how patients define acromegaly disease activity and treatment success and to quantify from the patients' perspective the relative importance of each disease parameter included in the ACRODAT®. METHODS: One hundred acromegaly patients on medical therapy (mean age = 47.1 years; SD = 11.96) completed an online preference study evaluating hypothetical patient profiles described in terms of insulin-like growth factor-I (IGF-I) levels, tumor size, comorbid conditions, signs/symptoms, and quality of life (QoL). Participants first completed a single-profile task experiment by rating 20 single patient profiles as exhibiting stable, mild, or significant disease activity based on treatment success. Next, participants completed a double-profile discrete choice experiment (DCE) by selecting the patient that was doing "better" from 15 profile pairs. Results were analyzed using logistic and conditional logistic models. RESULTS: When choosing between stable vs. mild or significant disease activity, signs/symptoms, tumor size, and IGF-I levels were weighted equally; IGF-I and signs and symptoms were valued equally when selecting mild vs. significant disease activity. The DCE showed that, statistically, all disease parameters, except comorbid conditions, predicted health status equally. Tumor size and IGF-I levels each accounted for 23% of the decision-making process; QoL, signs/symptoms, and comorbid conditions accounted for 21%, 19%, and 14%, respectively. CONCLUSION: All five ACRODAT® parameters had some influence on disease activity from the patients' perspective. To account for patients' preferences and optimize treatment and outcomes, a holistic disease management approach should be employed.

Conducting a Discrete-Choice Experiment Study Following Recommendations for Good Research Practices: An Application for Eliciting Patient Preferences for Diabetes Treatments.

OBJECTIVES: To consolidate and illustrate good research practices in health care to the application and reporting of a study measuring patient preferences for type 2 diabetes mellitus medications, given recent methodological advances in stated-preference methods. METHODS: The International Society for Pharmacoeconomics and Outcomes Research good research practices and other recommendations were used to conduct a discrete-choice experiment. Members of a US online panel with type 2 diabetes mellitus completed a Web-enabled, self-administered survey that elicited choices between treatment pairs with six attributes at three possible levels each. A D-efficient experimental design blocked 48 choice tasks into three 16-task surveys. Preference estimates were obtained using mixed logit estimation and were used to calculate choice probabilities. RESULTS: A total of 552 participants (51% males) completed the survey. Avoiding 90 minutes of nausea was valued the highest (mean -10.00; 95% confidence interval [CI] -10.53 to -9.47). Participants wanted to avoid low blood glucose during the day and/or night (mean -3.87; 95% CI -4.32 to -3.42) or one pill and one injection per day (mean -7.04; 95% CI -7.63 to -6.45). Participants preferred stable blood glucose 6 d/wk (mean 4.63; 95% CI 4.15 to 5.12) and a 1% decrease in glycated hemoglobin (mean 5.74; 95% CI 5.22 to 6.25). If cost increased by $1, the probability that a treatment profile would be chosen decreased by 1%. CONCLUSIONS: These results are consistent with the idea that people have strong preferences for immediate consequences of medication. Despite efforts to produce recommendations, ambiguity surrounding good practices remains and various judgments need to be made when conducting stated-preference studies. To ensure transparency, these judgments should be described and justified.

Assessing Rationality in Discrete Choice Experiments in Health: An Investigation into the Use of Dominance Tests.

BACKGROUND: Dominance tests are often applied to test for the rationality in the choice behavior of participants in discrete choice experiments (DCEs). OBJECTIVES: To examine how dominance tests have been implemented in recent DCE applications in health and discuss their theoretical and empirical interpretation. METHODS: Health-related DCEs published in 2015 were reviewed for the inclusion of tests on choice behavior. For studies that implemented a dominance test, information on application and interpretation of the test was extracted. Authors were contacted for test choice sets and observed proportions of subjects who chose the dominated option. Coefficients corresponding to the choice set were extracted to estimate the expected probability of choosing the dominated option with a logistic model and compared with the observed proportion. The theoretical range of expected probabilities of possible dominance tests was calculated. RESULTS: Of 112 health-related DCEs, 49% included at least one test for choice behavior; 28 studies (25%) included a dominance test. The proportion of subjects in each study who chose the dominated option ranged from 0% to 21%. In 46% of the studies, the dominance test led to the exclusion of participants. In the 15 choice sets that were analyzed, 2 had larger proportions of participants choosing the dominated option than expected (P < 0.05). CONCLUSIONS: Although dominance tests are frequently applied in DCEs, there is no consensus on how to account for them in data analysis and interpretation. Comparison of expected and observed proportions of participants failing the test might be indicative of DCE quality.

Older Adults' Preferences for Discussing Long-Term Life Expectancy: Results From a National Survey.

PURPOSE: Clinical practice guidelines recommend incorporating long-term life expectancy to inform a number of decisions in primary care. We aimed to examine older adults' preferences for discussing life expectancy in a national sample. METHODS: We invited 1,272 older adults (aged 65 or older) from a national, probability-based online panel to participate in 2016. We presented a hypothetical patient with limited life expectancy who was not imminently dying. We asked participants if they were that patient, whether they would like to talk with the doctor about how long they may live, whether it was acceptable for the doctor to offer this discussion, whether they want the doctor to discuss life expectancy with family or friends, and when it should be discussed. RESULTS: The 878 participants (69.0% participation rate) had a mean age of 73.4 years. The majority, 59.4%, did not want to discuss how long they might live in the presented scenario. Within this group, 59.9% also did not think that the doctor should offer the discussion, and 87.7% also did not want the doctor to discuss life expectancy with family or friends. Fully 55.8% wanted to discuss life expectancy only if it were less than 2 years. Factors positively associated with wanting to have the discussion included higher educational level, believing that doctors can accurately predict life expectancy, and past experience with either a life-threatening illness or having discussed life expectancy of a loved one. Reporting that religion is important was negatively associated. CONCLUSIONS: The majority of older adults did not wish to discuss life expectancy when we depicted a hypothetical patient with limited life expectancy. Many also did not want to be offered discussion, raising a dilemma for how clinicians may identify patients' preferences regarding this sensitive topic.

Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy.

PURPOSE: Patient preference information (PPI) have an increasing role in regulatory decision-making, especially in benefit-risk assessment. PPI can also facilitate prioritization of symptoms to treat and inform meaningful selection of clinical trial endpoints. We engaged patients and caregivers to prioritize symptoms of Duchenne and Becker muscular dystrophy (DBMD) and explored preference heterogeneity. METHODS: Best-worst scaling (object case) was used to assess priorities across 11 symptoms of DBMD that impact quality of life and for which there is unmet need. Respondents selected the most and least important symptoms to treat among a subset of five. Relative importance scores were estimated for each symptom, and preference heterogeneity was identified using mixed logit and latent class analysis. RESULTS: Respondents included patients (n = 59) and caregivers (n = 96) affected by DBMD. Results indicated that respondents prioritized "weaker heart pumping" [score = 5.13; 95% CI (4.67, 5.59)] and pulmonary symptoms: "lung infections" [3.15; (2.80, 3.50)] and "weaker ability to cough" [2.65; (2.33, 2.97)] as the most important symptoms to treat and "poor attention span" as the least important symptom to treat [- 5.23; (- 5.93, - 4.54)]. Statistically significant preference heterogeneity existed (p value < 0.001). At least two classes existed with different priorities. Priorities of the majority latent class (80%) reflected the aggregate results, whereas the minority latent class (20%) did not distinguish among pulmonary and other symptoms. CONCLUSIONS: Estimates of the relative importance for symptoms of Duchenne muscular dystrophy indicated that symptoms with direct links to morbidity and mortality were prioritized above other non-skeletal muscle symptoms. Findings suggested the existence of preference heterogeneity for symptoms, which may be related to symptom experience.

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.

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.

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.

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.

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.

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.

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.

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.