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Because immunizing large numbers of healthy people could be required to reduce a relatively small number of infections, disease incidence has a large impact on cost effectiveness, even if the infection is associated with very serious health outcomes. In addition to cost effectiveness, the US Advisory Committee on Immunization Practices requires evidence of stakeholders' values and preferences to help inform vaccine recommendations. This study quantified general-population preferences for vaccine trade-offs among disease severity, disease incidence, and other vaccine features.We developed a best-practice discrete choice experiment survey and administered it to 1185 parents of children aged 12-23 years and 1203 young adults aged 18-25 years from a national opt-in consumer panel. The data were analyzed using exploded-logit latent-class analysis.Latent-class analysis identified two classes with similar relative-importance weights in both samples. One of the two classes represented about half the samples and had preferences consistent with well-structured, logically ordered, and acceptably precise stated-preference utility. Preferences for the other half of the samples were poorly defined over the ranges of vaccine and disease attributes evaluated. Both parents and young adults in the first class evaluated protection from a disease with 1 in 100 incidence and full recovery at home as having statistically the same preference utility as a disease with 1 in 1 million incidence requiring hospitalization and resulting in permanent deafness.The results suggest that vaccines that protect against low-incidence, severe-outcome diseases, provide 'peace of mind' benefits not captured by standard health-outcome metrics. The fact that half the respondents had poorly defined vaccine preferences is a reminder of the challenges of implementing patient-centric vaccine decision making.

Background While clinical practice guidelines underscore the need to incorporate patient preferences in clinical decision making, incorporating meaningful assessment of patient preferences in clinical encounters is challenging. Structured approaches that combine quantitative patient preferences and clinical evidence could facilitate effective patient-provider communication and more patient-centric health care decisions. Adaptive conjoint or stated-preference approaches can identify individual preference parameters, but they can require a relatively large number of choice questions or simplifying assumptions about the error with which preferences are elicited. Method We propose an approach to efficiently diagnose preferences of patients for outcomes of treatment alternatives by leveraging prior information on patient preferences to generate adaptive choice questions to identify a patient’s proximity to known preference phenotypes. This information can be used for measuring sensitivity and specificity, much like any other diagnostic procedure. We simulated responses with varying levels of choice errors for hypothetical patients with specific preference profiles to measure sensitivity and specificity of a 2-question preference diagnostic. Results We identified 4 classes representing distinct preference profiles for patients who participated in a previous first-time anterior shoulder dislocation (FTASD) survey. Posterior probabilities of class membership at the end of a 2-question sequence ranged from 87% to 89%. We found that specificity and sensitivity of the 2-question sequences were robust to respondent errors. The questions appeared to have better specificity than sensitivity. Conclusions Our results suggest that this approach could help diagnose patient preferences for treatments for a condition such as FTASD with acceptable precision using as few as 2 choice questions. Such preference-diagnostic tools could be used to improve and document alignment of treatment choices and patient preferences. Highlights Approaches that combine patient preferences and clinical evidence can facilitate effective patient-provider communication and more patient-centric healthcare decisions. However, diagnosing individual-level preferences is challenging, and no formal diagnostic tools exist. We propose a structured approach to efficiently diagnose patient preferences based on prior information on the distribution of patient preferences in a population. We generated a 2-question test of preferences for the outcomes associated with the treatment of first-time anterior shoulder dislocation. The diagnosis of preferences can help physicians discuss relevant aspects of the treatment options and proactively address patient concerns during the clinical encounter.

Women with inflammatory bowel disease (IBD) face difficult decisions regarding treatment during pregnancy: while the majority of IBD medications are safe, there is substantial societal pressure to avoid exposures during pregnancy. However, discontinuation of IBD medications risks a disease flare occurring during pregnancy. This study quantified women’s knowledge about pregnancy and IBD and their willingness to accept the risks of adverse pregnancy outcomes to avoid disease activity or medication use during pregnancy. Women with IBD recruited from four centers completed an online discrete-choice experiment stated-preference study including eight choice tasks and the Crohn’s and Colitis Pregnancy Knowledge questionnaire. Random-parameters logit was used to estimate preferences for both the respondent personally and what the respondent thought most women would prefer. We also tested for systematically different preferences among individuals with different demographic and personal characteristics, including IBD knowledge. The primary outcome was the maximum acceptable risk of premature birth, birth defects, or miscarriage that women with IBD were willing to accept to avoid (1) taking an IBD medication or (2) having a disease flare during pregnancy. Among 230 respondents, women would accept, on average, up to a 4.9% chance of miscarriage to avoid a disease flare. On average, there were no statistically significant differences in women’s preferences for continuing versus avoiding medication in the absence of a flare. However, prior understanding of IBD and pregnancy significantly affected preferences for IBD medication use during pregnancy: women with “poor knowledge” would accept up to a 6.4% chance of miscarriage to avoid IBD medication use during pregnancy, whereas women with “adequate knowledge” would accept up to a 5.1% chance of miscarriage in order to remain on their medication. Respondents’ personal treatment preferences did not differ from their assessment of other women’s preferences. Women with IBD demonstrated a strong preference for avoiding disease activity during pregnancy. Knowledge regarding pregnancy and IBD was a strong modifier of preferences for continuation of IBD medications during pregnancy. These findings point to an important opportunity for intervention to improve disease control through education to increase medication adherence and alleviate unnecessary fears about IBD medication use during pregnancy.

Policy Points Public funding for mental health programs must compete with other funding priorities in limited state budgets. Valuing state-funded mental health programs in a policy-relevant context requires consideration of how much benefit from other programs the public is willing to forgo to increase mental health program benefits and how much the public is willing to be taxed for such program benefits. Taxpayer resistance to increased taxes to pay for publicly funded mental health programs and perceived benefits of such programs vary with state population size. In all states, taxpayers seem to support increased public funding for mental health programs such as state Medicaid services, suggesting such programs are underfunded from the perspective of the average taxpayer. Context The direct and indirect impacts of serious mental illness (SMI) on health care systems and communities represents a significant burden. However, the value that community members place on alleviating this burden is not known, and SMI treatment must compete with a long list of other publicly funded priorities. This study defines the value of public mental health interventions as what the public would accept, either in the form of higher taxes or in reductions in nonhealth programs, in return for increases in the number of mental health program beneficiaries. Methods We developed and fielded a best-practice discrete-choice experiment survey to quantify respondents' willingness to be taxed for increased spending among several competing programs, including a program for treating severe mental health conditions. A realistic decision frame was used to elicit respondents' willingness to support expanded state budgets for mental health programs if that expansion required either cuts in the competing publicly financed programs or tax increases. The survey was administered to a general population national sample of 10,000 respondents. Findings Nearly half the respondents in our sample either chose "no budget increase" for all budget scenarios or had preferences that were too disordered to estimate trade-off values. Including zero values for those respondents, we found that the mean (median) amount that all respondents were willing to be taxed annually for public mental health programs ranged between $156 ($99) per year for large-population states and $343 ($181) per year for small-population states. Respondents would accept reductions of between 1.6 and 3.4 beneficiaries in other programs in return for 1 additional mental health program beneficiary. Conclusions Our results are consistent with findings that a substantial portion of the US public is unwilling to pay higher taxes. Nevertheless, even including the substantial number of respondents who opposed any tax increase, the willingness of both the mean and median respondent to be taxed for mental health program expansions implies that programs providing mental health services such as state Medicaid are underfunded.

Objective To assess preferences of women with ovarian cancer regarding features of available anti-cancer regimens for platinum-resistant, biomarker-positive disease, with an emphasis on oral PARP inhibitor and standard intravenous (IV) chemotherapy regimens. Methods A discrete-choice-experiment preferences survey was designed, tested, and administered to women with ovarian cancer, with 11 pairs of treatment profiles defined using seven attributes (levels/ranges): regimen (oral daily, IV weekly, IV monthly); probability of progression-free (PFS) at 6 months (40%–60%); probability of PFS at 2 years (10%–20%); nausea (none, moderate); peripheral neuropathy (none, mild, moderate); memory problems (none, mild); and total out-of-pocket cost ($0 to $10,000). Results Of 123 participants, 38% had experienced recurrence, 25% were currently receiving chemotherapy, and 18% were currently taking a PARP inhibitor. Given attributes and levels, the relative importance weights (sum 100) were: 2-year PFS, 28; cost, 27; 6-month PFS, 19; neuropathy,14; memory problems, nausea, and regimen, all ≤5. To accept moderate neuropathy, participants required a 49% (versus 40%) chance of PFS at 6 months or 14% (versus 10%) chance at 2 years. Given a 3-way choice where PFS and cost were equal, 49% preferred a monthly IV regimen causing mild memory problems, 47% preferred an oral regimen causing moderate nausea, and 4% preferred a weekly IV regimen causing mild memory and mild neuropathy. Conclusions These findings challenge the assumption that oral anti-cancer therapies are universally preferred by patients and demonstrate that there is no “one size fits all” regimen that is preferable to women with ovarian cancer when considering recurrence treatment regimens.

Violations of the assumptions of complete information [CI] and independence of irrelevant alternatives (IIA) in discrete-choice experiment (DCE) data imply sensitivity of preference estimates to the decision context and the alternatives evaluated. There is a paucity of evidence on how these two assumptions affect health-preference results and whether the usual specifications of random-parameters logit models are sufficient to address these violations. We assessed the appropriateness of these assumptions in a DCE valuating interventions to prevent long-term health problems that could be identified through whole genome sequencing. A DCE survey was administered to members of a nationally representative consumer panel to elicit their preferences for options to reduce the risk of health problems. The treatment options presented (surgery, medication, and watchful waiting) and the context for the decisions elicited (severity and likelihood of the health problem) were varied experimentally to evaluate the sensitivity of preference results to such changes. We find evidence of IIA violations as the options presented to prevent health changed. Our results also are consistent with the expectation that additional substitutes decrease the monetized value of alternatives. We also find some evidence that the decision context can moderate such effects, which constitutes a new finding.

Objectives To test the convergent validity of simple and more complex study designs in a discrete-choice experiment (DCE) of multiple sclerosis (MS) treatment preferences. Methods Five hundred US adults with MS completed an online DCE survey. Respondents answered 8 choice questions with pairs of constructed MS treatment profiles defined by delays in problems with walking, delays in problems with cognition, thyroid disorders, and 10-y risks of kidney failure and serious brain infection (i.e., progressive multifocal leukoencephalopathy [PML]). Four hundred respondents completed choice questions using 4 levels for all attributes, except thyroid disorders with 3 levels. One hundred respondents completed choice questions using only the 2 extreme attribute levels of the 4-level version. Random-parameters logit models were used to estimate choice-model parameters. Results Respondents viewing the 4-level and 2-level versions agreed on the relative importance of the 3 most important attributes: cognition, walking, and PML. Respondents viewing the 4-level version indicated much stronger disutility for a 0% to 0.5% increase in kidney-failure risk than those viewing the 2-level version where the risk for kidney failure increased from 0% to 3%. Otherwise, utilities for other 4-level attributes were approximately linear but with significantly steeper slopes (except for cognition) than the 2-level estimates, indicating that attributes were perceived as more important as the number of levels increased. Conclusions Although the relative importance of some attributes was similar, the 2-level and 4-level versions generally failed to demonstrate convergent validity. If the study goal is attribute rankings, a 2-level version could be adequate. If goals include quantifying tradeoffs among attribute levels, more complex designs can help respondents discriminate among attribute levels. Reductions in measurement error using fewer attribute levels appear to have come at the expense of less discriminating evaluations.

Background Total knee arthroplasty (TKA) is a common treatment for end-stage knee osteoarthritis but is associated with increased complication rates compared with unicompartmental knee arthroplasty (UKA). UKA offers better functional outcomes but is associated with a higher risk of revision. The purpose of this study was to apply good-practice, stated-preference methods to quantify patient preferences for benefit-risk tradeoffs associated with arthroplasty treatments for end-stage knee osteoarthritis. Methods A discrete-choice experiment was developed with the following attributes: chance of complications, functional ability, awareness of the knee implant, and chance of needing another operation within 10 years. Patients included those aged 40 to 80 years with knee osteoarthritis. A pivot design filtered respondents into 1 of 2 surveys on the basis of self-reported functional ability (good compared with fair or poor) as measured by the Oxford Knee Score. Treatment-preference data were collected, and relative attribute-importance weights were estimated. Results Two hundred and fifty-eight completed survey instruments from 92 males and 164 females were analyzed, with 72 respondents in the good-function cohort and 186 in the fair/poor-function cohort. Patients placed the greatest value or relative importance on serious complications and rates of revision in both cohorts. Preference weights did not vary between cohorts for any attribute. In the good-function cohort, 42% of respondents chose TKA and 58% chose UKA. In the fair/poor-function cohort, 54% chose TKA and 46% chose UKA. Conclusions Patient preferences for various treatment attributes varied among patients in a knee osteoarthritis population. Complication and revision rates were the most important factors to patients, suggesting that physicians should focus on these areas when discussing treatments. The proportion of patients who chose UKA suggests that the current trend of increased UKA utilization is aligned with patient preferences. Clinical relevance Systematic elicitation of patient preferences for knee arthroplasty procedures, which lays out evidence-based risks and benefits of different treatments, indicates a larger subset of the knee osteoarthritis population may prefer UKA than would be suggested by the current rates of utilization of the procedure. Arthroplasty treatment should align with patient preferences and eligibility criteria to better deliver patient-centered care.

Background Novel ketamine-based pharmacotherapies can reduce depressive symptoms among patients with treatment-resistant depression (TRD), but associated short-term symptoms and potential adverse events raise complex benefit-risk questions. Methods A web-based discrete-choice experiment was administered to 161 esketamine-treated TRD subjects participating in the SUSTAIN-2 and SUSTAIN-3 clinical-trials; and to 301 online panel participants. Participants evaluated hypothetical depression treatments defined by varying levels of improvement in depression symptoms; time to response; transient post-dose issues (dissociation, dizziness, monitoring requirements, and driving restrictions); and potential long-term risks of ulcerative cystitis and cognitive impairment previously reported from ketamine abuse. Results The clinical-trial and panel respondents had similar preferences. On average, the 54 % of clinical-trial and 64 % of panel respondents who accepted benefit-risk tradeoffs placed the highest value on improving depression symptoms (relative importance = 10) and the least importance on avoiding transient post-dose issues (relative importance 5.0 %] or ulcerative cystitis higher than the survey’s maximum 5 % level to improve their depression symptoms from MADRS-40 (severe) equivalent to MADRS-20 (moderate) equivalent; panel respondents accepted somewhat lower risks (P>.05). Conclusions Most patients and panelists indicated a willingness to accept significant ulcerative cystitis or cognitive risks to realize improvements in depression, with few differences between samples. Avoiding transient post-dose issues with esketamine was of relatively little concern to most participants.

Objective To measure preferences of women with ovarian cancer regarding risks, side effects, costs and benefits afforded by maintenance therapy (MT) with a poly ADP ribose polymerase (PARP) inhibitor. Methods A discrete-choice experiment elicited preferences of women with ovarian cancer regarding 6 attributes (levels in parentheses) relevant to decisions for MT versus treatment break: (1) overall survival (OS; 36, 38, 42 months); (2) progression-free survival (PFS; 15, 17, 21 months); (3) nausea (none, mild, moderate); (4) fatigue (none, mild, moderate); (5) probability of death from myelodysplastic syndrome/acute myelogenous leukemia (MDS/AML; 0% to 10%); (6) monthly out-of-pocket cost ($0 to $1000). Participants chose between 2 variable MT scenarios and a static scenario representing treatment break, with multiple iterations. Random-parameters logit regression was applied to model choices as a function of attribute levels. Results 95 eligible participants completed the survey; mean age was 62, 48% had recurrence, and 17% were ever-PARP inhibitor users. Participants valued OS (average importance weight 24.5 out of 100) and monthly costs (24.6) most highly, followed by risk of death from MDS/AML (17.9), nausea (14.7), PFS (10.5) and fatigue (7.8). Participants would accept 5% risk of MDS/AML if treatment provided 2.2 months additional OS or 4.8 months PFS. Participants would require gains of 2.6 months PFS to accept mild treatment-related fatigue and 4.4 months to accept mild nausea. Conclusions When considering MT, women with ovarian cancer are most motivated by gains in OS. Women expect at least 3–4 months of PFS benefit to bear mild side effects of treatment.

Supplemental Digital Content is available in the text., Background: Regulatory and clinical decisions involving health technologies require judgements about relative importance of their expected benefits and risks. We sought to quantify heart-failure patients’ acceptance of therapeutic risks in exchange for improved effectiveness with implantable devices. Methods: Individuals with heart failure recruited from a national web panel or academic medical center completed a web-based discrete-choice experiment survey in which they were randomized to one of 40 blocks of 8 experimentally controlled choice questions comprised of 2 device scenarios and a no-device scenario. Device scenarios offered an additional year of physical functioning equivalent to New York Heart Association class III or a year with improved (ie, class II) symptoms, or both, with 30-day mortality risks ranging from 0% to 15%, in-hospital complication risks ranging from 0% to 40%, and a remote adjustment device feature. Logit-based regression models fit participants’ choices as a function of health outcomes, risks and remote adjustment. Results: Latent-class analysis of 613 participants (mean age, 65; 49% female) revealed that two-thirds were best represented by a pro-device, more risk-tolerant class, accepting up to 9% (95% CI, 7%–11%) absolute risk of device-associated mortality for a one-year gain in improved functioning (New York Heart Association class II). Approximately 20% were best represented by a less risk-tolerant class, accepting a maximum device-associated mortality risk of 3% (95% CI, 1%–4%) for the same benefit. The remaining class had strong antidevice preferences, thus maximum-acceptable risk was not calculated. Conclusions: Quantitative evidence on benefit-risk tradeoffs for implantable heart-failure device profiles may facilitate incorporating patients’ views during product development, regulatory decision-making, and clinical practice.

Women with inflammatory bowel disease (IBD) face difficult decisions regarding treatment during pregnancy: while the majority of IBD medications are safe, there is substantial societal pressure to avoid exposures during pregnancy. However, discontinuation of IBD medications risks a disease flare occurring during pregnancy.This study quantified women's knowledge about pregnancy and IBD and their willingness to accept the risks of adverse pregnancy outcomes to avoid disease activity or medication use during pregnancy.Women with IBD recruited from four centers completed an online discrete-choice experiment stated-preference study including eight choice tasks and the Crohn's and Colitis Pregnancy Knowledge questionnaire. Random-parameters logit was used to estimate preferences for both the respondent personally and what the respondent thought most women would prefer. We also tested for systematically different preferences among individuals with different demographic and personal characteristics, including IBD knowledge. The primary outcome was the maximum acceptable risk of premature birth, birth defects, or miscarriage that women with IBD were willing to accept to avoid (1) taking an IBD medication or (2) having a disease flare during pregnancy.Among 230 respondents, women would accept, on average, up to a 4.9% chance of miscarriage to avoid a disease flare. On average, there were no statistically significant differences in women's preferences for continuing versus avoiding medication in the absence of a flare. However, prior understanding of IBD and pregnancy significantly affected preferences for IBD medication use during pregnancy: women with "poor knowledge" would accept up to a 6.4% chance of miscarriage to avoid IBD medication use during pregnancy, whereas women with "adequate knowledge" would accept up to a 5.1% chance of miscarriage in order to remain on their medication. Respondents' personal treatment preferences did not differ from their assessment of other women's preferences.Women with IBD demonstrated a strong preference for avoiding disease activity during pregnancy. Knowledge regarding pregnancy and IBD was a strong modifier of preferences for continuation of IBD medications during pregnancy. These findings point to an important opportunity for intervention to improve disease control through education to increase medication adherence and alleviate unnecessary fears about IBD medication use during pregnancy.

To determine patient acceptability of benefit-risk trade-offs in selecting treatment options for drug-resistant mesial temporal lobe epilepsy, including open brain surgery, laser ablation (laser interstitial thermal therapy [LITT]), and continued medications.A discrete-choice experiment survey was developed, consisting of 20 versions that were randomly assigned to respondents. Each version had 8 sets of constructed treatment alternatives, representing open brain surgery, LITT, or continued medical management. For each set, respondents indicated the treatment alternative they would choose first. Treatment alternatives were characterized by varying levels of chance of seizure freedom for at least 2 years (20-70%), risk of 30-day mortality (0-10%), and risk of neurological deficits (0-40%). Respondents' choices were analyzed using random-parameters logit models to quantify acceptable benefit-risk trade-offs. Preference heterogeneity was evaluated using latent-class analysis.The survey was administered to 2 cohorts of adult patients with drug-resistant epilepsy: a Duke cohort identified using diagnostic codes (n = 106) and a web-recruited panel with a self-reported physician diagnosis of drug-resistant epilepsy (n = 300). Based on mean preference weights, respondents who indicated a willingness to consider surgical intervention would accept a reduction in chance of seizure freedom from 70% to a minimum-acceptable benefit (MAB) of 23% if they could undergo LITT rather than open brain surgery. For a reduction in 30-day mortality from 1% to 0%, MAB was 52%. For a reduction in risk of long-term deficits from 10% to 0%, MAB was 39%. Latent-class analysis revealed additional choice patterns identifying respondent groups that more strongly favored continuing medications or undergoing surgery.Patients who are receptive to surgery would accept significantly lower treatment effectiveness to undergo a minimally invasive procedure relative to open brain surgery. They also were willing to accept lower treatment benefit to reduce risks of mortality or neurological deficits.

Context Health systems should aim to deliver on what matters most to patients. With respect to end of life (EOL) care, knowledge on patient preferences for care is currently lacking. Objective To quantify preference weights for key EOL care indicators. Methods We developed a discrete choice experiment (DCE) survey with 13 key indicators related to patients' experience in the last 6 weeks of life. We fielded the survey to a web-panel of caregiver proxies for recently deceased care recipients. We obtained 250 responses in each of 5 countries: India, Singapore, Kenya, the UK and the US. Latent-class analysis was used to evaluate preference weights each indicator within and across countries. Results A 2-class latent-class model was the best fit. Class 1 (average class probability = 64.7%) preference weights were logically ordered and highly significant, while Class 2 estimates were generally disordered, suggesting poor data quality. Class 1 results indicated health care providers' ability to control patients' pain to desired levels was most important (11.5%, 95% CI: 10.3-12.6%), followed by clean, safe, and comfortable facilities (10.0%, 95% CI: 9.0-11.0%); and kind and sympathetic health care providers (9.8%, 95% CI: 8.8-10.9%). Providers' support for non-medical concerns had the lowest preference weight (4.4%, 95% CI: 3.6-5.3%). Differences in preference weights across countries were not statistically significant. Conclusions Results reveal that not all aspects of EOL care are equally valued. Not accounting for these differences would lead to inappropriate conclusions on how best to improve EOL care.

Background The popularity of quality-adjusted life years (QALYs) has been resistant to concerns about validity and reliability. Utility-theoretic outcome equivalents are widely used in other areas of applied economics. Equivalence values can be derived for time, money, risk, and other metrics. These equivalence measures preserve all available information about individual preferences and are valid measures of individual welfare changes. Objective The objective of this study was to derive alternative generalized equivalence measures from first principles and illustrate their application in an empirical comparative-effectiveness example. Methods We specify a general-equilibrium model incorporating neoclassical utility functions, health production function, severity-duration preferences, and labor-market tradeoff function. The empirical implementation takes advantage of discrete-choice experiment methods that are widely accepted in other areas of applied economics and increasingly in health economics. We illustrate the practical significance of restrictive QALY assumptions using comparative-effectiveness results based on both QALYs and estimates of welfare-theoretic time-equivalent values for anti-tumor necrosis factor and prolonged corticosteroid treatments for Crohn’s disease in three distinct preference classes. Results The QALY difference between the two treatments is 0.2 months, while time-equivalent values range between 0.5 and 1.3 months for aggregate and class-specific differences. Thus, the QALY-based analysis understates welfare-theoretic values by 60%-85%. Conclusion These results suggest that using disease-specific equivalence values offer a meaningful alternative to QALYs to compare global outcomes across treatments. The equivalence values approach is consistent with principles of welfare economics and offers several features not represented in QALYs, including accounting for preference nonlinearities in disease severity and duration, inclusion of preference-relevant nonclinical healthcare factors, representing preferences of clinically-relevant patient subpopulations, and including utility losses related to risk aversion.

Supplemental material, sj-pdf-1-mdm-10.1177_0272989X20979833 for Is Easier Better Than Harder? An Experiment on Choice Experiments for Benefit-Risk Tradeoff Preferences by Jui-Chen Yang, Shelby D. Reed, Steve Hass, Mark B. Skeen and F. Reed Johnson in Medical Decision Making

Supplemental material, sj-doc-2-mdm-10.1177_0272989X20979833 for Is Easier Better Than Harder? An Experiment on Choice Experiments for Benefit-Risk Tradeoff Preferences by Jui-Chen Yang, Shelby D. Reed, Steve Hass, Mark B. Skeen and F. Reed Johnson in Medical Decision Making

Background Recently developed peanut desensitization treatment reduces the incidence of allergic reactions, the anxiety associated with the risk of accidental exposure, and the burden of precautionary behavior. Eliciting parent preferences for tradeoffs involving treatment effectiveness, tolerability, costs, and convenience quantifies the burden of juvenile peanut allergy and the perceived value of peanut desensitization therapies. Objective To understand heterogeneity in parents' treatment preferences and the role of personal characteristics in explaining differences. Methods An Internet-based, discrete-choice experiment survey was administered to a national sample of 500 parents of children aged 4 to 17 years with peanut allergy to quantify parents' preferences for peanut desensitization therapies for their children. Latent-class, mixed-logit analysis estimated relative importance coefficients for groups of participants with distinctly different preferences. Results Parents' choice patterns fell into 1 of 4 preference subgroups: (1) Cost-sensitive, (2) Protreatment (but Side-Effect–Averse), (3) Trader, and (4) Inconsistent. Mode of administration had little relative importance across all subgroups. Characteristics associated with belonging to a given preference subgroup included parent age, child age, income, parent perception of child risk and ability to manage allergic reactions, past allergic reactions, and changes in precautionary behaviors posttreatment. Conclusions We found distinct differences in parent preferences for tradeoffs involving effectiveness, tolerability, and costs of peanut desensitization treatments. Parents' treatment preferences help quantify the burden of juvenile peanut allergy and the perceived value of new therapies. Such information can inform patient-centric clinical and regulatory decision making.

Background ‘Hope’ is a construct in patient-centered value frameworks, but few studies have attempted to measure the value of hope separately from treatment-related gains in quality of life and survival to support its application in economic evaluation. Objective To generate quantitative information on the “value of hope”. Methods We designed a discrete-choice experiment in which treatment alternatives varied the probability of achieving 10-year survival, expected survival as the weighted sum of short-term and long-term survival, health status, and out-of-pocket cost. Two-hundred patients with cancer or history of cancer recruited by Cancer Support Community each completed 10 choice questions. We used mixed-logit and latent-class models to analyze the choice data. Results Relative to fixed survival periods of two, three or five years with 0% chance of 10-year survival, participants positively valued treatments with 5% and 10% chances of 10-year survival. However, participants negatively valued a 20% chance of 10-year survival that required an offsetting 80% chance of shorter survival. This finding was particularly strong when expected survival was two years. Compared to a 0% chance, dollar-equivalent values of 5% and 10% chances of long-term survival were $5,975 and $12,421, respectively, independent of health status or expected survival. The corresponding value for 20% versus 0% chance of long-term survival was negative. Latent-class analysis revealed 4 groups with distinct preference patterns. Conclusions Our findings affirm positive value for hope independent of expected survival and health status. However, this finding does not universally hold in all situations nor across all groups.

Objective To conduct a discrete-choice experiment to quantify Americans’ acceptance of severe acute respiratory syndrome coronavirus 2 infection risks for earlier lifting of social-distancing restrictions and diminishing the pandemic’s economic impact. Methods We designed a discrete-choice experiment to administer 10 choice questions to each respondent representing experimentally controlled pairs of scenarios defined by when nonessential businesses could reopen (May, July, or October 2020), cumulative percentage of Americans contracting coronavirus disease 2019 (COVID-19) through 2020 (2% to 20%), time for economic recovery (2 to 5 years), and the percentage of US households falling below the poverty threshold (16% to 25%). Respondents were recruited by SurveyHealthcareGlobus. Results A total of 5953 adults across all 50 states completed the survey between May 8 and 20, 2020. Latent-class analysis supported a 4-class model. The largest class (36%) represented COVID-19 risk-minimizers, reluctant to accept any increases in COVID-19 risks. About 26% were waiters, strongly preferring to delay reopening nonessential businesses, independent of COVID-19 risk levels. Another 25% represented recovery-supporters, primarily concerned about time required for economic recovery. This group would accept COVID-19 risks as high as 16% (95% CI: 13%-19%) to shorten economic recovery from 3 to 2 years. The final openers class prioritized lifting social distancing restrictions, accepting of COVID-19 risks greater than 20% to open in May rather than July or October. Political affiliation, race, household income, and employment status were all associated with class membership (P Conclusion Americans have diverse preferences pertaining to social-distancing restrictions, infection risks, and economic outcomes. These findings can assist elected and public-health officials in making difficult policy decisions related to the pandemic.

The International Society for Pharmacoeconomics and Outcomes Research (ISPOR)'s "Good Practices Task Force" reports are highly cited, multistakeholder perspective expert guidance reports that reflect international standards for health economics and outcomes research (HEOR) and their use in healthcare decision making. In this report, we discuss the criteria, development, and evaluation/consensus review and approval process for initiating a task force. The rationale for a task force must include a justification, including why this good practice guidance is important and its potential impact on the scientific community. The criteria include: (1) necessity (why is this task force required?); (2) a methodology-oriented focus (focus on research methods, approaches, analysis, interpretation, and dissemination); (3) relevance (to ISPOR's mission and its members); (4) durability over time; (5) broad applicability; and 6) an evidence-based approach. In addition, the proposal must be a priority specifically for ISPOR. These reports are valuable to researchers, academics, students, health technology assessors, medical technology developers and service providers, those working in other commercial entities, regulators, and payers. These stakeholder perspectives are represented in task force membership to ensure the report's overall usefulness and relevance to the global ISPOR membership. We hope that this discussion will bring transparency to the process of initiating, approving, and producing these task force reports and encourage participation from a diverse range of experts within and outside ISPOR.

The Goldman dilemma presented athletes with a Faustian bargain that guaranteed winning an Olympic gold medal in their sport but resulted in certain death 5 years later. Athletes’ responses to Goldman’s bargain were reported from 1982 to 1995. Several studies subsequently evaluated people’s willingness to accept the bargain proposed in the Goldman question. Our study updates Goldman’s question using contingent-behavior questions, a preference-elicitation method widely applied in economics, marketing and psychology to understand people’s choice behavior. Contingent-behavior questions ask people to evaluate hypothetical tradeoffs between outcomes when real-world decisions are unobservable, nonexistent, or unreliable. A web-enabled survey was conducted with athletes in 50 sports between June, 2012 and April, 2013. Athletes were invited by their sport governing bodies in the United States to complete the online survey. Responses from 2888 athletes were collected. Our reformulation elicited athletes’ willingness to accept a performance-enhancing drug (PED) associated with the risk of a realistic fatal event, not certain death. A double-bounded dichotomous-choice question format was used to elicit athletes’ maximum acceptable mortality risk (MAMR) for winning an Olympic gold medal. Data were analyzed using an interval regression model to estimate the implicit probability of accepting a continuous risk level. MAMR was defined as the mortality risk level with a 0.50 probability of acceptance. Estimated mean MAMRs varied between 7 and 14% across athletes in different ranks and sports. Elite athletes were generally the most willing to accept a fatal cardiovascular risk to win a gold medal in the Olympics. This range was similar to the levels of risk that patients accept for life-changing interventions. Results suggest that very few athletes would be expected to accept a PED in the bargain postulated by the Goldman dilemma. Risk tolerance among elite athletes suggest they may be more aware of the potential financial and nonfinancial benefits of such a win, and/or less optimistic about their potential to move up in the level of competition without the use of PEDs.

Background: As more studies report on patient preferences for diabetes treatment, identifying diabetes outcomes other than glycated hemoglobin (HbA1c) to describe effectiveness is warranted to understand patient-relevant, benefit-risk tradeoffs. Objective: The aim of the study was to evaluate how preferences differ when effectiveness (glycemic control) is presented as long-term sequela (LTS) risk mitigation rather than an asymptomatic technical marker (HbA1c). Methods: People with type 2 diabetes and using insulin (n = 3160) were randomly assigned to four self-administered, discrete-choice experiments that differed by their presentation of effectiveness. Epidemiologic reviews were conducted to ensure a close approximation of LTS risk relative to HbA1c levels. The relative importance of treatment benefit-risk characteristics and maximum acceptable risk tradeoffs was estimated using an error-component logit model. Log-likelihood ratio tests were used to compare parameter vectors. Results: In total, 1031 people responded to the survey. Significantly more severe hypoglycemic events were accepted for a health improvement in terms of LTS mitigation versus HbA1c improvement (0.7 events per year; 95% confidence interval [CI]: 0.4-1.0 vs. 0.2 events per year 95% CI: -0.02 to 0.5) and avoidance of treatment-related heart attack risk (1.4 severe hypoglycemic events per year; 95% CI: 0.8-1.9 vs. 1 event per year; 95% CI: 0.6-1.3). This finding is supported by a log-likelihood test that rejected at the 0.05 level that respondent preference structures are similar across the different experimental arms of the discrete-choice experiment. Conclusion: We found evidence that benefit descriptions influence elicited preferences for the benefit-risk characteristics of injectable diabetes treatment. These findings argue for using carefully defined effectiveness measures to accurately take account of the patient perspective in benefit-risk assessments.

Background: The Food and Drug Administration’s Center for Devices and Radiological Health issued Guidance in 2016 on generating patient preference information to aid evaluation of medical devices. Consistent with this guidance, we aimed to provide quantitative patient preference evidence on benefit-risk tradeoffs relevant to transcatheter mitral valve repair versus medical therapy for patients with heart failure and symptomatic secondary mitral regurgitation. Methods: A discrete-choice experiment survey was designed to quantify patients’ tolerance for 30-day mortality or serious bleeding risks to achieve improvements in physical functioning or reductions in heart failure hospitalizations. Two samples were recruited: an online US panel of individuals reporting a diagnosis of heart failure (n=244) and patients with heart failure treated at Duke University Health System (n=175). Random-effects logit regression was used to model treatment choices as a function of benefit and risk levels. Results: Across both samples, approximately one-quarter (23.5%) consistently chose device profiles offering the higher level of physical functioning despite mortality and bleeding risks as high as 10%. Among respondents who at least once chose a device profile offering a lower level of functioning, improvement in physical functioning equivalent to a change from New York Heart Association class IV to III was ≈6 times more preferred than a change from New York Heart Association class III to II. Estimated discrete-choice experiment utility gains and losses revealed that respondents would accept up to a 9.7 percentage-point (95% CI, 8.2%–13.3%) increase in risk of 30-day mortality with devices that could improve functioning from New York Heart Association class IV to III, or up to 2.0% (95% CI, 1.4%–2.7%) for an improvement from New York Heart Association class III to II. Conclusions: Severity of heart failure symptoms influences patients’ willingness to accept risks associated with mitral valve medical devices. These findings can inform shared decision-making discussions with patients who are being evaluated for transcatheter mitral valve repair.

Background The objective of the study was to understand respondents' willingness to accept hypothetical treatment-related risks in return for the benefit of additional time with normal memory from potential Alzheimer's disease interception therapies. Methods A US web-based discrete-choice survey was administered to respondents ages 60 to 85 years with no Alzheimer's disease diagnosis and no cognitive symptoms. Choice questions required respondents to indicate whether they preferred a constant, no-treatment condition described as 4 years of normal memory followed by 3 years of cognitive impairment and 5 years of dementia or an interception treatment with chosen risks of disabling stroke and death, but with increased duration of normal memory. The study design included internal validity tests to verify data quality. Results On average, respondents were willing to accept a 5% to 13% risk of stroke or death in the first year for treatments that could provide 1 or more additional years with normal memory. Nevertheless, 30% of respondents failed a simple internal-validity test question where the treatment alternative offered no improvement in disease progression but had significant side effects. These respondents also were more likely to choose active treatment in the subsequent series of choice questions. This unexpected finding is consistent with hopeful attitudes of patients with debilitating and potentially fatal conditions. Conclusion Pro-treatment attitudes are clinically relevant and can affect the analysis and interpretation of stated-preference data. Internal-validity tests generally are underutilized in preference research. This study demonstrated how analysis of apparent validity failures can yield important insights about patient preferences.

This study demonstrates how experimental survey methods can be used to assess preferences for budget-constrained combinations of public and publicly provided goods and services. The study shows how to calculate welfare changes based on preferences for increases and decreases in programs within particular budget portfolios, and quantifies the effect of ideology on valuations of public programs. The findings show that respondents, including conservatives, were reluctant to approve of cuts when they were faced with the exact definition of what they needed to give up or how a decrease would affect the provision of services.

Demands for greater transparency in US regulatory assessments of benefits and risks, together with growing interest in engaging patients in Food and Drug Administration regulatory decision making, have resulted in several recent regulatory developments. Although Food and Drug Administration's Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH) have established patient-engagement initiatives, CDRH has proposed guidelines for considering quantitative data on patients' benefit-risk perspectives, while CDER has focused on a more qualitative approach. We summarize two significant studies that were developed in collaboration and consultation with CDER and CDRH. CDER encouraged a patient advocacy group to propose draft guidance on engaging patient and caregiver stakeholders in regulatory decision making for Duchenne muscular dystrophy. CDRH sponsored a discrete-choice experiment case study to quantify obese respondents' perspectives on "meaningful benefits." CDRH and CDER issued draft guidance in May and June 2015, respectively, on including patient-preference data in regulatory submissions. Both organizations face challenges. CDER is working on integrating qualitative data into existing evidence-based review processes and is exploring options for therapeutic areas not included on a priority list. CDRH has adopted an approach that requires patient-preference data to satisfy standards of valid scientific evidence. Although that strategy could facilitate integrating patient perspectives directly with clinical data on benefits and harms, generating such data requires building capacity.

Objective The diagnosis of Alzheimer's disease (AD) remains difficult. Lack of diagnostic certainty or possible distress related to a positive result from diagnostic testing could limit the application of new testing technologies. The objective of this paper is to quantify respondents' preferences for obtaining AD diagnostic tests and to estimate the perceived value of AD test information. Methods Discrete-choice experiment and contingent-valuation questions were administered to respondents in Germany and the United Kingdom. Choice data were analyzed by using random-parameters logit. A probit model characterized respondents who were not willing to take a test. Results Most respondents indicated a positive value for AD diagnostic test information. Respondents who indicated an interest in testing preferred brain imaging without the use of radioactive markers. German respondents had relatively lower money-equivalent values for test features compared with respondents in the United Kingdom. Conclusions Respondents preferred less invasive diagnostic procedures and tests with higher accuracy and expressed a willingness to pay up to €700 to receive a less invasive test with the highest accuracy.

We investigated the perceived value of government programs on early-childhood development as a means of reducing childhood poverty. We incorporated preferences for the process as well as the outcome by developing two stated-preference survey instruments. One survey directly elicited respondents’ willingness to pay specifically for high-quality, intensive, early-childhood development programs at federal and state levels. A second survey elicited respondents’ preferences for increasing or decreasing taxes and reallocating expenditures between other government programs and early-childhood programs. We found that respondents cared greatly about how childhood poverty was reduced, not just reducing poverty per se. The perceived effectiveness of a program and ideological perspective were found to be important determinants of preferences for a poverty-reduction program. Respondents across all groups, including conservatives and respondents who perceived the effectiveness of early-childhood programs to be low, were not in favor of reducing the early-childhood program.

Fragile X syndrome (FXS) is the most common inherited form of intellectual disability. The objective of this study was to determine the relative importance that caregivers place on improving different phenotypic traits observed in males with FXS to better understand the greatest medical needs for developing and evaluating FXS treatments.Fragile X syndrome caregivers (n = 614) compared hypothetical treatments in a discrete-choice experiment. The treatments varied in their effects on 6 outcomes associated with FXS: learning and applying new skills, explaining needs, controlling behavior, taking part in new social activities, caring for oneself, and paying attention. The relative importance was calculated for improving severe or moderate levels of disability and transformed to a 10-point scale. Relative importance was also quantified by patient age group (child, adolescent, and adult).Most important to caregivers were controlling behavior (10.0) and caring for oneself (9.9). Least important was taking part in new social activities (4.2). A partial improvement in controlling behavior or self-care was more important than full resolution of the least important disabilities. This was consistent across age groups. Improvements from severe to moderate disability were more important than from moderate to no disability.Caregivers expressed strong preferences for improvement in self-care and behavioral control, independent of the age of the individual with FXS. These data may be helpful when designing studies to test the efficacy of FXS treatments because small treatment effects on very important outcomes may be valued more than large treatment effects on less valued outcomes.

Background & Aims Patients with Crohn’s disease (CD) must make decisions about their treatment. We aimed to quantify patients’ preferences for different treatment outcomes and adverse events. We also evaluated the effects of latent class heterogeneity on these preferences. Methods An online stated-preference survey was completed by 812 individuals with CD in the Crohn’s and Colitis Foundation Partners cohort (IBD Partners). Patients were given information on symptoms and severity of active disease; duration of therapy with corticosteroids; and risks of serious infection, cancer and surgery. Patients were asked to assume that their treatment was not working and to choose an alternative therapy. The primary outcome was remission-time equivalents (RTE) of a given duration of symptom severity or treatment-related risk. Latent class choice models identified groups of patients with dominant treatment-outcome preferences and associated patient characteristics with these groups. Results Latent class analysis demonstrated 3 distinct groups of survey responders whose choices were strongly influenced by avoidance of active symptoms (61%), avoidance of corticosteroid use (25%), or avoidance of risks of cancer, infection or surgery (14%) when choosing a therapy. Class membership was correlated with age, sex, mean short CD activity index score and corticosteroid avoidance. RTEs in each latent class differed significantly from the mean RTEs for the overall sample, although the symptom-avoidant class most closely approximated the overall sample. Conclusions In an online survey of patients with CD, we found substantial heterogeneity in preference for medication efficacy and risk of harm. Physicians and regulators should therefore not assume that all patients have mean-value preferences—this could result in significant differences in health-technology assessment models.

Benefit-risk assessment is the foundation for decision making throughout the life cycle of medical products. Because patients are the beneficiaries of the efficacy of medical treatments and also bear their possible risks, their perspectives and judgments about value and the relative importance of benefits and risks should be at the heart of the medical decision-making process. Patient engagement is now at a tipping point; there have been a growing number of patient engagement initiatives over the past several years, but there remains the need for a common language, alignment on engagement approaches and best practices, and a shared vision regarding a desired future state. This article discusses insights gleaned from the DIA conference, "Patient Engagement in Benefit-Risk Assessment throughout the Life Cycle of Medical Products" (September 2015). It highlights the changes that will need to occur within the patient, medical-product sponsor, and regulatory cultures in order for patient engagement to become integrated into the medical-product development process and life cycle maintenance. Furthermore, it emphasizes that reaching the desired future state will require a conscious commitment from all stakeholders to work collaboratively to develop shared solutions and to map a common path forward.

Objectives To develop a tool for testing internal validity of discrete choice experiment (DCE) data, deploy the program, and collect summary test results from a sample of active health researchers to demonstrate the practical utility of the tool in a wide range of health applications. Methods A previously developed Gauss program had been in use for testing internal validity. The program was translated to MATLAB and adapted, compiled, and deployed. Sixty-seven authors who had coauthored one or more published DCE studies between 2013 and 2016 were contacted by email; provided access to the tool, instructions, and an example data file; and invited to submit test summaries for tabulation. Results Twenty-one researchers from 10 countries contributed test results from a total of 55 DCE data sets. Fifty-one studies included at least two out of a possible six tests. Attribute dominance was the most common test, and stability had the highest failure incidence. Only three summaries included a transitivity test, and no failures were detected. Conclusions It was possible to evaluate multiple internal validity checks for most data sets even when the experimental design did not explicitly include tests. Nevertheless, internal validity is rarely reported. Free availability of the tool for testing data quality could improve both reporting and more careful design of DCE studies to help validate and interpret stated preference data.

•A meta-simulation strategy analyzes what affects utility-difference precision.•Meta-data are obtained from 34 individual DCE patient-preference data sets.•Utility-difference precision is regressed on sample size and study characteristics.•Study-design features affect precision more than experimental-design efficiency.•An empirical sample-size power equation is derived for DCE health studies.

The objective of this study was to quantify patients' preferences related to benefits and risks of antipsychotic treatments for schizophrenia and to assess the relative importance of treatment attributes and adherence.Treatment-related preferences among U.S. residents with a self-reported physician diagnosis of schizophrenia were assessed via a discrete-choice experiment. Patients chose between competing hypothetical scenarios characterized by improvements in positive symptoms, negative symptoms, and social functioning; incidence of weight gain, extrapyramidal symptoms (EPS), hyperprolactinemia, and hyperglycemia; and medication formulation. Preferences were estimated by using a random-parameters logit model, and the impact of adherence was estimated with conditional logit models.The final sample consisted of 271 patients. Complete improvement in positive symptoms was the most preferred outcome (relative importance score of 10.0), followed by elimination of hyperglycemia (3.6, 95% confidence interval [CI]=2.6-4.6), improvement in negative symptoms (3.0, CI=1.6-4.3), reduced weight gain (2.6, CI=1.2-4.0), avoidance of hyperprolactinemia (1.7, CI=.9-2.6), improved social functioning (1.5, CI=.4-2.5), and avoidance of EPS (1.0, CI=.3-1.8). Patients judged a daily pill superior to monthly injections (p.01) and monthly injections superior to injections every three months (p.01) for adherent patients and monthly injections superior to a daily pill for nonadherent patients (p=.01).Persons who self-identified as having schizophrenia judged improvement in positive symptoms as the most important treatment benefit. Hyperglycemia was identified as the most important adverse event. Patients judged oral formulations to be better than monthly injections for adherent patients and monthly injections to be a better choice for nonadherent patients.

Background Best-practice guidelines for stated-preference methods suggest there is a limit to the number of attributes respondents can reliably evaluate. This study explores a cost-effective solution to combining elicitation formats from a single study to obtain more preference information from a given sample while limiting respondents’ cognitive burden. Methods A stated-preference survey combined both discrete-choice experiment (DCE) and best–worst scaling (BWS) elicitation formats to Alzheimer's disease caregivers. DCE questions elicited attribute-level preferences for one subset of attributes, and object-case BWS elicited overall relative attribute importance for another subset of attributes, with two overlapping attributes in both designs. Two alternative joint models combined preferences from the BWS and DCE data. One model controlled for confounding between response-error variance and preference parameters in the DCE model, and the other did not. Results About 400 caregivers completed the survey. We estimated attribute-level preference parameters for 17 attributes, 9 of which were directly estimated using the DCE data, and 8 of which were extrapolated based on the overall relative importance estimated using the object-case BWS data. Results from both joint models and individual models indicate that relative preferences from the two question formats were the same up to a scale factor. Conclusion Our results suggest that combining DCE and object-case BWS is a cost-effective solution to the need for more information when study resources are limited. Moreover, for these data at least, researchers' concerns about serious confounding between DCE model estimates and response-error variance appear unwarranted.