Recommendations for Emerging Good Practice and Future Research in Relation to Family and Caregiver Health Spillovers in Health Economic Evaluations: A Report of the SHEER Task Force.

BACKGROUND: Omission of family and caregiver health spillovers from the economic evaluation of healthcare interventions remains common practice. When reported, a high degree of methodological inconsistency in incorporating spillovers has been observed. AIM: To promote emerging good practice, this paper from the Spillovers in Health Economic Evaluation and Research (SHEER) task force aims to provide guidance on the incorporation of family and caregiver health spillovers in cost-effectiveness and cost-utility analysis. SHEER also seeks to inform the basis for a spillover research agenda and future practice. METHODS: A modified nominal group technique was used to reach consensus on a set of recommendations, representative of the views of participating subject-matter experts. Through the structured discussions of the group, as well as on the basis of evidence identified during a review process, recommendations were proposed and voted upon, with voting being held over two rounds. RESULTS: This report describes 11 consensus recommendations for emerging good practice. SHEER advocates for the incorporation of health spillovers into analyses conducted from a healthcare/health payer perspective, and more generally inclusive perspectives such as a societal perspective. Where possible, spillovers related to displaced/foregone activities should be considered, as should the distributional consequences of inclusion. Time horizons ought to be sufficient to capture all relevant impacts. Currently, the collection of primary spillover data is preferred and clear justification should be provided when using secondary data. Transparency and consistency when reporting on the incorporation of health spillovers are crucial. In addition, given that the evidence base relating to health spillovers remains limited and requires much development, 12 avenues for future research are proposed. CONCLUSIONS: Consideration of health spillovers in economic evaluations has been called for by researchers and policymakers alike. Accordingly, it is hoped that the consensus recommendations of SHEER will motivate more widespread incorporation of health spillovers into analyses. The developing nature of spillover research necessitates that this guidance be viewed as an initial roadmap, rather than a strict checklist. Moreover, there is a need for balance between consistency in approach, where valuable in a decision making context, and variation in application, to reflect differing decision maker perspectives and to support innovation.

A Discrete Choice Analysis Comparing COVID-19 Vaccination Decisions for Children and Adults.

Importance: COVID-19 vaccination rates in the US remain below optimal levels. Patient preferences for different attributes of vaccine products and the vaccination experience can be important in determining vaccine uptake decisions. Objective: To assess preferences for attributes of adult and pediatric COVID-19 vaccination among US adults. Design, Setting, and Participants: An online survey of a national panel of 1040 US adults was conducted in May and June 2021. A discrete choice analysis was used to measure the relative value of each attribute in the decision to choose a COVID-19 vaccination option for adults or children. Six attributes were used to described hypothetical vaccination options: vaccine effectiveness, mild side effects, rare adverse events, number of doses, time required for vaccination, and regulatory approval. Respondents chose between hypothetical vaccination profiles or no vaccination. Additional survey questions asked about vaccination beliefs, COVID-19 illness experience, COVID-19 risk factors, vaccination status, and opinions about the risk of COVID-19. Exposures: Respondents chose which vaccine profile they would prefer to receive for themselves (or no vaccination). Respondents then considered an identical set of profiles for a hypothetical child aged 0 to 17 years. Main Outcomes and Measures: Relative value of vaccination-related attributes were estimated using Bayesian logit regression. Preference profiles for subgroups were estimated using latent class analyses. Results: A total of 1040 adults (610 [59%] female; 379 participants [36%] with an age of 55 years and older years) responded to the survey. When asked about vaccination choices for themselves, participants indicated that vaccine effectiveness (95% vs 60%) was a significant attribute (ß, 9.59 [95% CrI, 9.20-10.00] vs ß, 0.41 [95% CrI, 0-0.80]). Respondents also preferred fewer rare adverse events (ß, 6.35 [95% CrI, 5.74-6.86), fewer mild side effects (ß, 5.49; 95% CrI, 5.12-5.87), 1 dose (ß, 5.41; 95% CrI, 5.04-5.78), FDA approval (ß, 6.01; 95% CrI, 5.64-6.41), and shorter waiting times (ß, 5.67; 95% CrI, 4.87-6.48). Results were very similar when framing the question as adult or child vaccination, with slightly stronger preference for fewer rare adverse events for children. Latent class analysis revealed 4 groups of respondents: (1) individuals sensitive to safety and regulatory status, (2) individuals sensitive to convenience, (3) individuals who carefully considered all attributes in making their choices, and (4) individuals who rejected the vaccine. Conclusions and Relevance: In this survey study of US adults, the identification of 4 distinct preference groups provides new information to guide communications to support vaccine decision making. In particular, the group that prioritize convenience (less time required for vaccination and fewer doses) may present an opportunity to create actionable strategies to increase vaccination uptake for both adult and pediatric populations.

Estimating Joint Health State Utility Algorithms Under Partial Information.

OBJECTIVES: We explored the performance of existing joint health state utility estimators when data are not available on utilities that isolate single-condition health states excluding any co-occurring condition. METHODS: Using data from the National Epidemiologic Survey on Alcohol and Related Conditions-III, we defined 2 information sets: (1) a full-information set that includes the narrowly defined health state utilities used in most studies that test the performance of joint health state utility estimators, and (2) a limited information set that includes only the more broadly defined health state utilities more commonly available to researchers. We used an example of alcohol use disorder co-occurring with cirrhosis of the liver, depressive disorder, or nicotine use disorder to illustrate our analysis. RESULTS: We found that the performance of joint health state utility estimators is appreciably different under limited information than under full information. Full-information estimators typically overestimate the joint state utility, whereas limited-information estimators underestimate the joint state utility, except for the minimum estimator, which is overestimated in all cases. CONCLUSIONS: Researchers using joint health state utility estimators should understand the information set available to them and use methodological guidance appropriate for that information set. We recommend the minimum estimator under limited information based on its ease of use, consistency (and therefore a predictable direction of bias), and lower root mean squared error.

Alcohol consumption and health-related quality of life in the US during the COVID-19 pandemic: a US national survey.

BACKGROUND: Alcohol consumption has changed during the COVID-19 pandemic yet the impacts on alcohol-related outcomes, and specifically health-related quality of life, are not completely known. Our objective was to assess the association between alcohol consumption and health-related quality of life (HRQOL) during the COVID-19 pandemic. METHOD: We conducted an on-line/telephone survey of three cross-sectional samples of US adults during a nine-month stretch of the pandemic, from August 2020 through April 2021, collecting data on drinking-current quantity/frequency and change since prior to pandemic, HRQOL (using the SF-6D), and perceived impact of the pandemic on respondents' lives-overall impact and disruptions across various dimensions (job loss, school closures, social isolation, loss of income). We pooled the data from the three administrations and applied survey weights to reflect the US population. We described drinking behavior and pandemic impact, and regressed HRQOL on alcohol consumption risk level (per World Health Organization categories), change in drinking since pre-pandemic, and pandemic impact using weighted least squares, controlling for respondents' demographic characteristics. We tested the significance of categorical variables using Wald tests at a p-value of 0.05. RESULTS: Among 3,125 respondents, weighted to reflect the US population, 68% reported drinking during the pandemic and 40% reported a change in drinking from pre-pandemic level (either increased or decreased). Mean HRQOL among our sample was 0.721 (SD 0.003). Any change in drinking from pre-pandemic level was independently associated with significantly lower HRQOL compared to never drinking (pre or during pandemic), from - 0.0251 points for decreased/stopped drinking to -0.0406 points for increased drinking (combined levels' Wald test F = 10.62, p < 0.0000). COVID-19 pandemic related impacts/disruptions were associated with HRQOL decrements ranging from - 0.0834 to -0.1340 (Wald test F = 64.34, p < 0.0000). CONCLUSION: The US population HRQOL was substantially lower during the pandemic than reported a decade earlier (mean = 0.79 in 2012-13). While pandemic-related impacts and disruptions may explain a large part of this decrement, changes in drinking-and the associated implications of such changes-might also play a role. Both individuals who reduced their drinking during the pandemic and those who increased consumption may be at risk of poor HRQOL.

The COVID-19 pandemic has precipitated changes in drinking that may be positive or negative depending on who is affected and how. We conducted a survey of over 3,000 adults in the US during the middle of the pandemic to understand drinking and quality of life. We found that US adults' quality of life was worse during the pandemic than in prior years, and people who either increased or decreased the amount that they drank were particularly worse-off. People who reported being severely impacted by the pandemic, however, were also much worse-off in terms of quality of life, and actually more so than people whose drinking habits changed. We need to pay attention to how drinking is connected to stressful events such as the pandemic, and make sure to attend to people who change their drinking in either direction as this may indicate underlying problems.

Health Utility of Drinkers' Family Members: A Secondary Analysis of a US Population Data Set.

Background. Problematic alcohol use is known to harm individuals surrounding the drinker. This study described the health utility of people who reported having a family member(s) whom they perceived as a "problem drinker."Methods. We conducted a secondary analysis of the US National Epidemiologic Survey of Alcohol and Related Conditions Wave 3 (NESARC-III, 2012-13) data to estimate the independent associations of a family member's problem drinking on the respondent's health utility, also known as health-related quality of life, assessed via the SF-6D. Participants included 29,159 noninstitutionalized adults, of whom 21,808 reported perceiving a family member or members as having a drinking problem at any point in that person's life. Respondent drinking was assessed via self-report and diagnostic interview. We used population-weighted multivariate regression to estimate disutility. Results. After adjusting for the respondent's own alcohol consumption, alcohol use disorder (AUD), family structure, and sociodemographic characteristics, the mean decrement in SF-6D score associated with perceiving a family member as a problem drinker ranged from 0.033 (P < 0.001) for a spouse/partner to 0.023 (P < 0.001) for a grandparent, sibling, aunt, or uncle. The mean decrement in SF-6D score from having AUD oneself was 0.039 (P < 0.001). Conclusions. Perceived problem drinking within one's family is associated with statistically significant losses in health utility, the magnitude of which is dependent on relationship type. The adverse consequences associated with problem drinking in the family may rival having AUD oneself. Implications. Family-oriented approaches to AUD interventions may confer outsize benefits, especially if focused on the spouse or partner. Economic evaluation of alcohol misuse could be made more accurate through the inclusion of family spillover effects. Highlights: Spillover effects from problem drinking in the family vary by relationship type.One's perception of their spouse or child as having a drinking problem is associated with a utility decrement of equal magnitude to having alcohol use disorder oneself.Medical decision makers should consider the outsize effects of family spillovers in treatment decisions in the context of alcohol consumption, particularly among spouses and children of problem drinkers.Economic evaluation should consider how to incorporate family spillover effects from problem drinking in alcohol-related models.

The relative value of carer and patient quality of life: A person trade-off (PTO) study.

Carer quality of life is increasingly considered alongside patient quality of life in economic evaluation. Important questions remain about how to value carer and patient quality of life effects alongside one another. In this study, we estimated the relative social value of two conceptualisations of carer quality of life (health-related and care-related) compared to patient quality of life. Relative valuations were estimated using a person trade-off (PTO) study with 990 representative members of the UK public. Participants chose between hypothetical services that improved the quality of life of carers and patients, iterating to a point of indifference. Overall 84% of participants completing the task were willing to trade patient and carer quality of life effects. Relative to a reference point of 1 for patient health-related quality of life, we estimated a social value of 0.74 for carer health-related quality of life effects and 0.69 for carer care-related quality of life effects. In conclusion, public preferences appear to support the inclusion of carer quality of life effects within economic evaluation. The results provide a means to value different carer quality of life outcomes in economic evaluation, where such values are needed and deemed appropriate.

Intergenerational Decision Making: The Role of Family Relationships in Medical Decision Making.

A symposium held at the 42nd annual Society for Medical Decision Making conference on October 26, 2020, focused on intergenerational decision making. The symposium covered existing research and clinical experiences using formal presentations and moderated discussion and was attended by 43 people. Presentations focused on the roles of pediatric patients in decision making, caregiver decision making for a child with complex medical needs, caregiver involvement in advanced care planning, and the inclusion of spillover effects in economic evaluations. The moderated discussion, summarized in this article, highlighted existing resources and gaps in intergenerational decision making in four areas: decision aids, economic evaluation, participant perspectives, and measures. Intergenerational decision making is an understudied and poorly understood aspect of medical decision making that requires particular attention as our society ages and technological advances provide new innovations for life-sustaining measures across all stages of the lifespan.

Estimating an exchange-rate between care-related and health-related quality of life outcomes for economic evaluation: An application of the wellbeing valuation method.

Quality of life outcomes for family carers and patients may be measured in different ways within the same economic evaluation. We used the wellbeing valuation method to calculate "exchange rates" between care-related outcomes (the Carer Experience Scale and CarerQoL-7D) and health-related (the EQ-5D-5L) outcomes. Data on quality of life outcomes were collected through a postal quality of life survey in the UK. A random effects model was used to estimate carers' wellbeing as a function of their EQ-5D-5L, Carer Experience Scale (or CarerQoL-7D) and a set of control variables. When life satisfaction was used as the measure of wellbeing, a one-point gain in the Carer Experience Scale (0-100 scale) was equivalent (in wellbeing terms) to a 0.014 gain in EQ-5D-5L value; and a one point gain in the CarerQoL-7D (0-100 scale) was equivalent to a 0.033 gain in EQ-5D-5L. The exchange rate values were reduced when capability was used as the measure of wellbeing. The exchange rates estimated in this study offer a means to place carer and patient outcomes, measured via different quality of life instruments, on a common scale, although there are important issues to consider in operationalising the technique.

How Patients Choose a Laryngologist: A Pilot Stated Preference Study.

OBJECTIVE: Patients consider many factors when deciding how to receive medical care. This study used best-worst scaling (BWS), a technique novel to otolaryngology, to quantitatively examine preferences among patients choosing a laryngologist. Our objective was to quantify in a pilot cohort the relative importance patients place on a variety of attributes when seeking a laryngologist. STUDY DESIGN: BWS survey. SETTING: Academic voice clinic. METHODS: New patients were recruited to take a computerized BWS survey developed using attributes derived from patient input, expert opinion, and literature review. Attributes were grouped into 4 categories: physician reputation, physician qualifications, hospital-related factors, and other nonclinical factors. Responses were analyzed using multinomial logit regression to determine importance scores and associations with other variables. RESULTS: Eighty-seven of 93 patients recruited participated (93.5% response rate). Physician qualifications were the most important attributes to patients, with specialty laryngology training receiving the highest importance score (20.8; 95% CI, 20.2 to 21.5; P < .0001). Recommendations from referring physicians (15.6; 95% CI, 14.3 to 16.9) and use of cutting-edge technology (11.9; 95% CI, 10.7 to 13.1) were the second and third most important, respectively. Least important were nonclinical factors, including wait time to get an appointment (4.3; 95% CI, 2.8 to 5.8) and convenience of office location (1.5; 95% CI, 0.9 to 2.1). Just over half of patients (51.2%) reported willingness to wait 4 weeks for an appointment with a laryngologist. Older patients were less concerned with convenience-related factors. CONCLUSION: Nonclinical factors were less important to patients than clinical factors, and laryngology-specific training was paramount. Stated preference methodologies can elucidate underlying preferences and help providers make care more patient centered.

Family-level impact of genetic testing: integrating health economics and ethical, legal, and social implications.

Tweetable abstract Health economics and ELSI can be better integrated to consider the family impacts of genetic and genomic testing.

Health-related quality of life of alcohol use disorder with co-occurring conditions in the US population.

BACKGROUND: Alcohol use disorder (AUD) commonly co-occurs with other health conditions or other substance use, complicating our understanding of the health-related quality of life (HRQoL) of AUD. We described the HRQoL of alcohol use disorder in the presence of co-occurring conditions and identified the contribution of each. METHODS: Secondary analysis of National Epidemiologic Survey on Alcohol and Related Conditions III data, consisting of 36,309 non-institutionalized US adults; descriptive and regression analysis. HRQoL measured via the SF-6D; AUD via the Alcohol Use Disorder and Associated Disabilities Interview Schedule (AUDADIS-5); physical, mental health, and substance use disorders/conditions as reported or assessed via AUDADIS-5. RESULTS: AUD was independently associated with lower HRQoL for individuals experiencing co-occurring conditions. Compared to no AUD, past year AUD reduced SF-6D score by 0.0304 (SE = 0.0027) and prior-to-past-year AUD reduced SF-6D by 0.0163 (SE = 0.0023). AUD's co-occurring conditions were independently associated with lower HRQoL, beyond the reduction from AUD: any co-occurring physical health condition was associated with a 0.062 point reduction in SF-6D score (SE = 0.0023), any mental health condition with a 0.084 point reduction (SE = 0.0025), and any substance use disorder with a 0.038 point reduction (SE = 0.0023). CONCLUSIONS: AUD's association with diminished HRQoL may be explained in large part by the presence of co-occurring conditions among individuals reporting AUD, as these co-occurring conditions are associated with substantial decrements in HRQoL-often eclipsing the magnitude of the decrements associated with AUD alone. Alcohol use interventions endeavoring to improve HRQoL should consider the entirety of an individual to design patient-centered care.

SF-6D utility scores for alcohol use disorder status and alcohol consumption risk levels in the US population.

AIMS: To estimate US population health utilities for subgroups defined by alcohol use disorder (AUD) status and consumption level. DESIGN: Cross-sectional survey. SETTING: Community settings in the United States (i.e. excluding institutional settings). PARTICIPANTS: A total of 36,042 adults (age 18+) in non-institutional settings in the United States. MEASUREMENTS: We used 12-item Short Form Survey (SF-12) data from the National Epidemiologic Survey on Alcohol and Related Conditions-III to calculate mean Short Form-6 dimension (SF-6D) utility scores across World Health Organization alcohol consumption risk levels-very high risk, high risk, medium risk, low risk and an additional abstinent level-for three groups: (1) the general population (n = 36,042), (2) individuals with life-time AUD (n = 9925) and (3) individuals with current AUD (n = 5083), and assessed minimally important differences (MIDs) between consumption levels. Each group is a subset of the previous group. FINDINGS: The general population's mean SF-6D utility was higher than that of individuals with life-time or current AUD across all consumption risk levels (0.79 versus 0.76 for both AUD groups). For all groups, SF-6D utilities increased as consumption risk level decreased to non-abstinent levels, and reducing consumption from very high risk to any lower level was associated with a statistically significant and meaningful improvement in utility. For individuals with life-time or current AUD, becoming abstinent from high-, medium- and low-risk levels was associated with significantly and meaningfully worse utilities. CONCLUSIONS: Higher alcohol consumption risk levels appear to be associated with lower health index scores for the general population and individuals with a history of alcohol use disorder, meaning that higher alcohol consumption is associated with worse health-related quality of life.

MyPref: pilot study of a novel communication and decision-making tool for adolescents and young adults with advanced cancer.

PURPOSE: Adolescents and young adults (AYAs) with cancer report feeling ill-informed about their cancer treatment options. Tools are needed to inform AYAs about treatment choices and amplify the AYA's voice in medical decision-making. We developed MyPref, a conjoint-analysis based tool that quantifies AYA preferences for future cancer treatments. METHODS: We conducted a staged pilot study of MyPref utilizing an intervention mixed methods design. AYAs and their parent or trusted person (PTP) completed MyPref and received a summary report of their preferences for treatment-related factors. Participants later completed the Preparation for Decision Making Scale and MyPref Experience Questionnaire and engaged in semi-structured interviews. Oncologists reported on the perceived accuracy and utility of MyPref. We used a weaving technique for presenting mixed methods data. RESULTS: Fifteen AYAs with advanced cancer, 7 PTPs, and 12 providers participated in this pilot; 32 (94%) completed all study items. AYA/PTPs stated study participation was useful and believed MyPref allowed for improved understanding of treatment factors and consideration, organization, and visualization of preferences. All providers agreed that MyPref made them think about patient's preferences and 9 (75%) reported they planned to change their approach to discussions about preferences for future treatments. CONCLUSION: MyPref is an objective way to estimate AYA and PTP preferences for future treatment characteristics. This novel tool may be a useful way to engage AYAs and PTPs in discussions around preferences for treatment and prepare AYAs for future decision-making. We are currently evaluating this tool longitudinally to determine the impact on actual treatment decisions.

Parental Considerations Regarding Cure and Late Effects for Children With Cancer.

BACKGROUND: More than 80% of children with cancer become long-term survivors, yet most survivors experience late effects of treatment. Little is known about how parents and physicians consider late-effects risks against a potential survival benefit when making treatment decisions. METHODS: We used a discrete choice experiment to assess the importance of late effects on treatment decision-making and acceptable trade-offs between late-effects risks and survival benefit. We surveyed 95 parents of children with cancer and 41 physicians at Dana-Farber/Boston Children's Cancer and Blood Disorders Center to assess preferences for 5 late effects of treatment: neurocognitive impairment, infertility, cardiac toxicity, second malignancies, and impaired growth and development. RESULTS: Each late effect had a statistically significant association with treatment choice, as did survival benefit (P < .001). Avoidance of severe cognitive impairment was the most important treatment consideration to parents and physicians. Parents also valued cure and decreased risk of second malignancies; physician decision-making was driven by avoidance of second malignancies and infertility. Both parents and physicians accepted a high risk of infertility (parents, a 137% increased risk; physicians, an 80% increased risk) in exchange for a 10% greater chance of cure. CONCLUSIONS: Avoidance of severe neurocognitive impairment was the predominant driver of parent and physician treatment preferences, even over an increased chance of cure. This highlights the importance of exploring parental late-effects priorities when discussing treatment options.

The health utility of mild and severe dysphonia.

OBJECTIVES/HYPOTHESIS: The impact of disease states can be measured using health state utilities, which are values that reflect economic preferences for health outcomes. Utilities for dysphonia have not been studied using direct methods. The objective of this project was to establish the baseline health utilities of mild and severe dysphonia from a societal perspective. STUDY DESIGN: Direct utility elicitation survey. METHODS: Four health states (monocular blindness, binocular blindness, mild dysphonia, and severe dysphonia) were evaluated by a convenience sample of adults recruited from the general public with three computer-aided estimation techniques (visual analog scale [VAS], standard gamble [SG], and time trade-off [TTO]). Standardized descriptions and voice recordings from multiple dysphonic patients were employed. Perfect health was defined as a utility of 1, with death 0. Analysis of variance with post hoc pairwise comparison was used to calculate significant differences between health states. RESULTS: Three hundred participants were surveyed, and 225 (75.0%) responses met quality thresholds. Severe dysphonia (VAS = 48.3, SG = 0.810, TTO = 0.798) was valued significantly worse than monocular blindness (VAS = 56.2, SG = 0.834, TTO = 0.839) on the VAS (P < .001) and equivalent on SG and TTO; it was preferred over binocular blindness (VAS = 25.7, SG = 0.631, TTO = 0.622; P < .001) with all methods. Mild dysphonia evaluated favorably with all methods to the other health states (VAS = 78.5, SG = 0.902, TTO = 0.908; P < .001). CONCLUSIONS: Voice disorders may have a measurable impact on utility, with severe dysphonia valued equivalently to monocular blindness. Mild dysphonia has a utility decrement from perfect health. These estimates are critical for quality-of-life assessment and could be used to assess cost-effectiveness of treatments for voice disorders. LEVEL OF EVIDENCE: NA Laryngoscope, 130:1256-1262, 2020.

Spillover Effects on Caregivers' and Family Members' Utility: A Systematic Review of the Literature.

BACKGROUND: A growing body of research has identified health-related quality-of-life effects for caregivers and family members of ill patients (i.e. 'spillover effects'), yet these are rarely considered in cost-effectiveness analyses (CEAs). OBJECTIVE: The objective of this study was to catalog spillover-related health utilities to facilitate their consideration in CEAs. METHODS: We systematically reviewed the medical and economic literatures (MEDLINE, EMBASE, and EconLit, from inception through 3 April 2018) to identify articles that reported preference-based measures of spillover effects. We used keywords for utility measures combined with caregivers, family members, and burden. RESULTS: Of 3695 articles identified, 80 remained after screening: 8 (10%) reported spillover utility per se, as utility or disutility (i.e. utility loss); 25 (30%) reported a comparison group, either population values (n = 9) or matched, non-caregiver/family member or unaffected individuals' utilities (n = 16; 3 reported both spillover and a comparison group); and 50 (63%) reported caregiver/family member utilities only. Alzheimer's disease/dementia was the most commonly studied disease/condition, and the EQ-5D was the most commonly used measurement instrument. CONCLUSIONS: This comprehensive catalog of utilities showcases the spectrum of diseases and conditions for which caregiver and family members' spillover effects have been measured, and the variation in measurement methods used. In general, utilities indicated a loss in quality of life associated with being a caregiver or family member of an ill relative. Most studies reported caregiver/family member utility without any comparator, limiting the ability to infer spillover effects. Nevertheless, these values provide a starting point for considering spillover effects in the context of CEA, opening the door for more comprehensive analyses.

Health utilities and parental quality of life effects for three rare conditions tested in newborns.

BACKGROUND: Measurement of health utilities is required for economic evaluations. Few studies have evaluated health utilities for rare conditions; even fewer have incorporated disutility that may be experienced by caregivers. This study aimed to (1) estimate health utilities for three rare conditions currently recommended for newborn screening at the state or federal level, and (2) estimate the disutility, or spillover, experienced by parents of patients diagnosed with a rare, heritable disorder. METHODS: A stated-preference survey using a time trade-off approach elicited health utilities for Krabbe disease, phenylketonuria, and Pompe disease at varying stages (mild, moderate, severe) and onset of disease symptoms (infancy, childhood, and adulthood). We recruited respondents from a nationally representative community sample (n = 862). Respondents valued disease specific health states in three consecutive question frames: (1) adult health state (> = 18 years of age), (2) child health state (< 18 years of age), and (3) as a parent of a child with a condition (parent spillover state). Corresponding mean utilities were calculated for plausible disease states in adulthood and childhood. Mean disutility was estimated for parental spillover. Predictors of utilities were evaluated using a negative binomial regression model. RESULTS: More severe conditions and infant health states received lower estimated utility and greater estimated disutility among parents. Conditions with the lowest estimated health utilities were severe infantile Pompe disease (0.40, CI: 0.34-0.46) and infantile Krabbe disease (0.37, CI: 0.32-0.43). Disutility was evident for all conditions evaluated (range: 0.07-0.19). CONCLUSIONS: Rare childhood conditions are associated with substantial estimated losses in quality of life. Evidence of disutility among parents further warrants the inclusion of spillover effects in cost-effectiveness analyses. Continued research is needed to assess and measure the effects of childhood disease from a family perspective.

Joint Utility Estimators in Substance Use Disorders.

BACKGROUND: Although co-occurring conditions are common with substance use disorders (SUDs), estimation methods for joint health state utilities have not yet been tested in this context. OBJECTIVES: To compare joint health state utility estimators in SUD to inform economic evaluation. METHODS: We conducted two Internet-based surveys of US adults to collect community perspective standard gamble utilities for SUD and common co-occurring conditions. We evaluated six conditions as they occur individually and four combinations of these as they occur in tandem. We applied joint utility estimators using the six individual conditions' utilities to compare their performance relative to the observed combination states' utilities. We assessed performance with bias (estimated utility minus observed utility) and root mean square error (RMSE). RESULTS: Using 3892 utilities from 1502 respondents, the minimum estimator was statistically unbiased (i.e., the 95% confidence interval included 0) for all combination states that we measured. The maximum estimator was unbiased for two states and the linear index and adjusted decrement estimators were unbiased for one state. The maximum estimator had the smallest RMSE for two combination states (back pain and prescription opioid misuse [0.0004] and injection crack and injection opioid use [0.0007]); the linear index and minimum estimators had the smallest RMSE for one combination state each. The additive and multiplicative estimators had the largest RMSE for all states. CONCLUSIONS: Our results demonstrate the usefulness of the minimum estimator in this context, and confirm the inadequacy of the additive and multiplicative estimators. Further research is needed to extend these results to other SUD states.