Patient preferences pertaining to treatment options for drug-resistant focal epilepsy.

OBJECTIVE: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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.

Who Would Pay Higher Taxes for Better Mental Health? Results of a Large-Sample National Choice Experiment.

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.

The preferences of women with ovarian cancer for oral versus intravenous recurrence regimens.

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.

Quantifying Value of Hope.

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.

Patient preferences for maintenance PARP inhibitor therapy in ovarian cancer treatment.

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.

Patient preferences for attributes of primary surgical debulking versus neoadjuvant chemotherapy for treatment of newly diagnosed ovarian cancer.

BACKGROUND: Randomized trials have reported conflicting findings on survival for advanced-stage ovarian cancer treated with primary debulking surgery (PDS) versus neoadjuvant chemotherapy with interval debulking; surgical complications and mortality are higher with PDS. We assessed women's preferences for tradeoffs related to this important clinical decision. METHODS: Ovarian cancer patients were recruited to complete a discrete-choice experiment (DCE) consisting of 8 choice tasks presenting experimentally designed treatment alternatives in terms of treatment order, extent of surgery including risk of ostomy, chance of death from surgical complications (1%-10%), readmission for surgical complications (5%-50%), progression-free survival (1-3 years), and overall survival (3-5 years). Random-parameters logit regression was applied to model participants' choices as a function of attribute levels. RESULTS: A total of 101 ovarian cancer survivors completed the DCE survey; of these participants, 30% were receiving chemotherapy at the time, and 33% had prior recurrence. Overall survival was of greatest importance to participants (36/100), followed by risk of readmission due to complications (23/100), progression-free survival (19/100), surgical mortality (16/100), extent of surgery (4/100), and order of surgery and chemotherapy (2/100). Overall, the participants would tolerate a 15-percentage point increase in risk of major complications (95% confidence interval [CI], 3%-29%) or a 4-percentage point increase in the risk of surgical mortality (95% CI, 2%-13%) in order to increase their expected overall survival from 3 to 3.5 years. CONCLUSIONS: Patients would accept a moderately higher risk of perioperative complications and surgical mortality in exchange for substantial gains in survival. These quantitative findings provide clinicians with a framework to discuss preferences with patients and to incorporate preferences into clinical trial design.

The Internal Validity of Discrete Choice Experiment Data: A Testing Tool for Quantitative Assessments.

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.

Something Is Better Than Nothing: The Value of Active Intervention in Stated Preferences for Treatments to Delay Onset of Alzheimer's Disease Symptoms.

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.

Do You Want to Hear the Bad News? The Value of Diagnostic Tests for Alzheimer's Disease.

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.

Patient preferences for the treatment of chronic cough: a discrete choice experiment.

BACKGROUND: Chronic cough is common, negatively affects quality of life and has limited treatment options. Inhibition of purinergic signalling is a promising therapeutic approach but is associated with taste-related adverse effects. Little is known about treatment preferences from the perspective of patients with chronic cough, such as trade-offs between efficacy and side effect. METHODS: Patients with chronic cough completed an online discrete choice experiment survey in which they answered a series of questions requiring a choice between two constructed treatment options characterised by varying attribute levels. Selection of cough and taste-related attributes was informed by qualitative interviews and clinical trial data. Logit-based models were used to analyse resulting choice data. RESULTS: The discrete choice experiment survey was completed by 472 participants with chronic cough. Among study attributes, frequency of intense cough attacks was the most important to participants, followed by taste change, frequency of night-time coughing and frequency of daytime coughing. To accept the least preferred taste disturbance of a bitter, metallic, chalky or oily taste change, participants required either: (1) elimination of night-time cough along with a slight reduction in daytime cough; (2) elimination of daytime cough along with a pronounced reduction in night-time or (3) reduction in intense cough attacks from 7 to 2 times per week. Two distinct preference patterns were identified, each placing different importance on efficacy versus side effect trade-offs. CONCLUSIONS: Participants with chronic cough were willing to accept some taste disturbances in exchange for improved efficacy of chronic cough treatments. Knowledge of patient preferences can facilitate shared decision-making.

Patient Risk-Benefit Preferences for Transcatheter Versus Surgical Mitral Valve Repair.

BACKGROUND: Transcatheter edge-to-edge repair (TEER) of mitral regurgitation is less invasive than surgery but has greater 5-year mortality and reintervention risks, and leads to smaller improvements in physical functioning. The study objective was to quantify patient preferences for risk-benefit trade-offs associated with TEER and surgery. METHODS AND RESULTS: A discrete choice experiment survey was administered to patients with mitral regurgitation. Attributes included procedure type; 30-day mortality risk; 5-year mortality risk and physical functioning for 5 years; number of hospitalizations in the next 5 years; and risk of additional surgery in the next 5 years. A mixed-logit regression model was fit to estimate preference weights. Two hundred one individuals completed the survey: 63% were female and mean age was 74 years. On average, respondents preferred TEER over surgery. To undergo a less invasive procedure (ie, TEER), respondents would accept up to a 13.3% (95% CI, 8.7%-18.5%) increase in reintervention risk above a baseline of 10%, 4.6 (95% CI, 3.1-6.2) more hospitalizations above a baseline of 1, a 10.7% (95% CI, 6.5%-14.5%) increase in 5-year mortality risk above a baseline of 20%, or more limited physical functioning representing nearly 1 New York Heart Association class (0.7 [95% CI, 0.4-1.1]) over 5 years. CONCLUSIONS: Patients in general preferred TEER over surgery. When holding constant all other factors, a functional improvement from New York Heart Association class III to class I maintained over 5 years would be needed, on average, for patients to prefer surgery over TEER.

Using Separate Single-Outcome Risk Presentations Instead of Integrated Multioutcome Formats Improves Comprehension in Discrete Choice Experiments.

INTRODUCTION: Despite decades of research on risk-communication approaches, questions remain about the optimal methods for conveying risks for different outcomes across multiple time points, which can be necessary in applications such as discrete choice experiments (DCEs). We sought to compare the effects of 3 design factors: 1) separated versus integrated presentations of the risks for different outcomes, 2) use or omission of icon arrays, and 3) vertical versus horizontal orientation of the time dimension. METHODS: We conducted a randomized study among a demographically diverse sample of 2,242 US adults recruited from an online panel (mean age 59.8 y, s = 10.4 y; 21.9% African American) that compared risk-communication approaches that varied in the 3 factors noted above. The primary outcome was the number of correct responses to 12 multiple-choice questions asking survey respondents to identify specific numbers, contrast options to recognize dominance (larger v. smaller risks), and compute differences. We used linear regression to test the effects of the 3 design factors, controlling for health literacy, graph literacy, and numeracy. We also measured choice consistency in a subsequent DCE choice module. RESULTS: Mean comprehension varied significantly across versions (P < 0.001), with higher comprehension in the 3 versions that provided separated risk information for each risk. In the multivariable regression, separated risk presentation was associated with 0.58 more correct responses (P < 0.001; 95% confidence interval: 0.39, 0.77) compared with integrated risk information. Neither providing icon arrays nor using vertical versus horizontal time formats affected comprehension rates, although participant understanding did correlate with DCE choice consistency. CONCLUSIONS: In presentations of multiple risks over multiple time points, presenting risk information separately for each health outcome appears to increase understanding. HIGHLIGHTS: When conveying information about risks of different outcomes at multiple time points, separate presentations of single-outcome risks resulted in higher comprehension than presentations that combined risk information for different outcomes.We also observed benefits of presenting single-outcome risks separately among respondents with lower numeracy and graph literacy.Study participants who scored higher on risk understanding were more internally consistent in their responses to a discrete choice experiment.

Assessing willingness to pay for improved sanitation in rural Vietnam.

OBJECTIVE: The willingness to pay (WTP) for the construction of bathrooms with a flush toilet was assessed in households in a rural community in northern Vietnam. We also examined the effects of socio-economic factors on the WTP. METHODS: The contingent valuation method, an economic survey technique, was used. We used the iterative bidding game technique to elicit household WTP that involved a sequence of dichotomous choice questions followed by a final open-ended question. A total of 370 households that did not have toilets were selected for this study. Respondents to the questionnaire were the primary income earners and decision-makers of their respective household. RESULTS: Of those responding to the questionnaire, 62.1 % reported being willing to pay for the construction of bathrooms with a flush toilet. The mean and median of maximum WTP amounts were Viet Nam Dong (VND) 15.6 million and VND 13.0 million, respectively (minimum VND 2.0 million; maximum VND 45.0 million). Significant correlates of the WTP rate were: (1) gender of the head of household, (2) age of the head of household, (3) economic status of household, (4) type of current toilet, (5) satisfaction with existing toilet, and (6) knowledge of health effects of poor sanitation. The significant determinants of WTP amount were (1) geographic location and (2) economic status of household. CONCLUSION: About two-third of the households in the study area were willing to pay for an improvement in their current sanitation arrangements. Both WTP rate and WP amount were strongly influenced by the economic status of the households and health knowledge of the study respondents.

Physician Preferences when Selecting Candidates for Lower-Quality Kidney Offers.

BACKGROUND: In the United States, more than 50% of kidneys in the lowest 15% quality range (those with Kidney Donor Profile Index >85) are discarded. Studies suggest that using more of these kidneys could benefit patients waiting for a transplant. This study assesses the trade-offs physicians make when selecting recipients for lower-quality kidneys. METHODS: A discrete choice experiment (DCE) was administered to surgeons and nephrologists in the United States who are involved in kidney acceptance decisions. The DCE presented kidneys that varied in terms of Kidney Donor Profile Index, expected cold ischemia time, donor age, pump parameters, serum creatinine levels, glomerulosclerosis, donor diabetes status, and whether donation was made after circulatory death. Candidate characteristics included recipients' age, diabetes history, time on dialysis, ejection fraction, HLA mismatch, calculated panel reactive antibody, and Karnofsky performance score. Regression analysis was used to estimate acceptability weights associated with kidney and recipient characteristics. RESULTS: A total of 108 physicians completed the DCE. The likelihood of acceptance was significantly lower with deterioration of kidney quality, expected cold ischemia time at transplantation, and missing biopsy and pump information. Acceptance was prioritized for patients who were higher on the waiting list, younger recipients, those who have spent less time on dialysis, and those without a history of diabetes. Performance status (Karnofsky score) and calculated panel reactive antibody also had a statistically significant but smaller association. Finally, ejection fraction had a marginally significant association, and HLA match had no significant association with the acceptance of marginal kidneys. A group of respondents were found to be primarily concerned about cold ischemia time. CONCLUSIONS: In this DCE, physicians considered the recipient characteristics that inform expected post-transplant survival score when they decided whether to accept a marginal kidney for a given recipient.

Discrete-Choice Experiment to Understand the Preferences of Patients with Hormone-Sensitive Prostate Cancer in the USA, Canada, and the UK.

BACKGROUND: Treatment options for patients with metastatic hormone-sensitive prostate cancer (mHSPC) have broadened, and treatment decisions can have a long-lasting impact on patients' quality of life. Data on patient preferences can improve therapeutic decision-making by helping physicians suggest treatments that align with patients' values and needs. OBJECTIVE: This study aims to quantify patient preferences for attributes of chemohormonal therapies among patients with mHSPC in the USA, Canada, and the UK. METHODS: A discrete-choice experiment survey instrument was developed and administered to patients with high- and very-high-risk localized prostate cancer and mHSPC. Patients chose between baseline androgen-deprivation therapy (ADT) alone and experimentally designed, hypothetical treatment alternatives representing chemohormonal therapies. Choices were analyzed using logit models to derive the relative importance of attributes for each country and to evaluate differences and similarities among patients across countries. RESULTS: A total of 550 respondents completed the survey (USA, 200; Canada, 200; UK, 150); the mean age of respondents was 64.3 years. Treatment choices revealed that patients were most concerned with treatment efficacy. However, treatment-related convenience factors, such as route of drug administration and frequency of monitoring visits, were as important as some treatment-related side effects, such as skin rash, nausea, and fatigue. Patient preferences across countries were similar, although patients in Canada appeared to be more affected by concomitant steroid use. CONCLUSION: Patients with mHSPC believe the use of ADT alone is insufficient when more effective treatments are available. Efficacy is the most significant driver of patient choices. Treatment-related convenience factors can be as important as safety concerns for patients.

Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.

Objective of this study is to quantify benefit-risk tradeoffs pertaining to potential gene therapies among adults and parents/caregivers of children with sickle cell disease (SCD). A discrete-choice experiment survey was developed in which respondents selected their preferred treatment alternatives in a series of experimentally controlled pairs of hypothetical gene therapies and a "no gene therapy" option. Gene therapy alternatives were defined based on the chance of eliminating SCD symptoms, expected increases in life expectancy they could offer, treatment-related risk of death, and potential increases in lifetime cancer risk. Respondents made selections based on their current disease severity and in the context of expectations of worsened disease. Three clinical sites and 1 patient organization recruited 174 adult patients and 109 parents of children with SCD to complete the survey. Adult and parent respondents were generally willing to choose gene therapies, but the adults required higher expected levels of efficacy (ie, higher chance of eliminating symptoms) than parents to choose gene therapies that conferred mortality risks of ≥10%. When adults and parents of children with less severe symptoms were asked to consider scenarios of higher levels of disease severity, the increased risk tolerance, and the lowest acceptable level of efficacy for gene therapies with mortality risks dropped by >50%. Baseline SCD symptoms are a major driver of gene therapy acceptability. Adults and parents of patients with milder symptoms may prefer other treatment options; however, an expectation of symptoms deterioration triggers strong reassessment of the acceptable benefit-risk balance of this novel technology.

Patients' preferences for treatment outcomes of add-on antiepileptic drugs: a conjoint analysis.

To understand the relative importance of the outcomes of add-on antiepileptic drugs (AEDs) and the willingness of patients with epilepsy to accept therapeutic trade-offs between seizure control and tolerability, we administered a Web-enabled, choice-format conjoint survey to patients with a self-reported physician diagnosis of epilepsy and symptoms of partial seizures. Patients answered nine choice questions to evaluate treatment outcomes of two different hypothetical add-on AEDs. Patients were first asked to choose the better of the two medicines and then asked a follow-up question about whether or not they would add the selected AED to their current treatment regimen. Our study demonstrated that patients with epilepsy consider seizure reduction to be the top priority when ranking it against the reduction or elimination of side effects. This study aids in better understanding of patients' AED treatment preferences and may aid in management of epilepsy.

Patient Preferences for Waiting Time and Kidney Quality.

BACKGROUND AND OBJECTIVES: Approximately 20% of deceased donor kidneys are discarded each year in the United States. Some of these kidneys could benefit patients who are waitlisted. Understanding patient preferences regarding accepting marginal-quality kidneys could help more of the currently discarded kidneys be transplanted. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: This study uses a discrete choice experiment that presents a deceased donor kidney to patients who are waiting for, or have received, a kidney transplant. The choices involve trade-offs between accepting a kidney today or a future kidney. The options were designed experimentally to quantify the relative importance of kidney quality (expected graft survival and level of kidney function) and waiting time. Choices were analyzed using a random-parameters logit model and latent-class analysis. RESULTS: In total, 605 participants completed the discrete choice experiment. Respondents made trade-offs between kidney quality and waiting time. The average respondent would accept a kidney today, with 6.5 years of expected graft survival (95% confidence interval, 5.9 to 7.0), to avoid waiting 2 additional years for a kidney, with 11 years of expected graft survival. Three patient-preference classes were identified. Class 1 was averse to additional waiting time, but still responsive to improvements in kidney quality. Class 2 was less willing to accept increases in waiting time for improvements in kidney quality. Class 3 was willing to accept increases in waiting time even for small improvements in kidney quality. Relative to class 1, respondents in class 3 were likely to be age ≤61 years and to be waitlisted before starting dialysis, and respondents in class 2 were more likely to be older, Black, not have a college degree, and have lower Karnofsky performance status. CONCLUSIONS: Participants preferred accepting a lower-quality kidney in return for shorter waiting time, particularly those who were older and had lower functional status.

Quantifying Benefit-Risk Preferences for Heart Failure Devices: A Stated-Preference Study.

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.

Is Easier Better Than Harder? An Experiment on Choice Experiments for Benefit-Risk Tradeoff Preferences.

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.