How Much Better is Faster? Value Adjustments for Health-Improvement Sequences.

While the quality-adjusted life-year construct has advantages of simplicity and consistency, simplicity requires strong assumptions. In particular, standard assumptions result in health-state utility functions that are unrealistically linear and separable in risk and duration. Consequently, sequencing of a series of health improvements has no effect on the total value of the sequence because each increment is assessed independently of previous increments. Utility functions in nearly all other areas of applied economics are assumed to be nonlinear with diminishing marginal utility so it matters where an improvement occurs in a sequence. We construct a conceptual framework that that demonstrates how diminishing marginal utility for health improvements could affect preferences for different sequence patterns. Using this framework, we derive conditions for which the sum of conventional health-state utilities understates, overstates, or approximates the sequence-sensitive value of health improvements. These patterns suggest the direction and magnitude of possible adjustments to conventional value calculations. We provide numerical examples and identify recent studies whose results are consistent with the conceptual model.

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.

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.

Willingness to Accept Trade-Offs Among COVID-19 Cases, Social-Distancing Restrictions, and Economic Impact: A Nationwide US Study.

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<.01). 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.

Criteria and Process for Initiating and Developing an ISPOR Good Practices Task Force Report.

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.

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.

Variation Among Patients With Crohn's Disease in Benefit vs Risk Preferences and Remission Time Equivalents.

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.

Comparing the Noncomparable: The Need for Equivalence Measures That Make Sense in Health-Economic Evaluations.

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.

Effective Partnering in Conducting Benefit-Risk Patient Preference Studies: Perspectives From a Patient Advocacy Organization, a Pharmaceutical Company, and Academic Stated-Preference Researchers.

BACKGROUND: Formal incorporation of patients' perspectives is becoming increasingly important in medical product development and decision making. This article shares practical advice regarding how patient advocacy organizations, the pharmaceutical industry, and academic experts in stated-preference research can effectively partner on benefit-risk patient preference studies. METHODS: The authors partnered on a benefit-risk patient preference study related to the treatment of psoriasis. The authors from Duke Clinical Research Institute also share their experiences in collaborating with numerous other organizations in conducting benefit-risk patient preference studies. RESULTS: Upon initiation of the study partnership with appropriate experts, training is important to ensure all collaborators have a common understanding of the methodology, what objectives stated-preference methods can support, and expectations for the project. To the extent possible, partners should align on and document relevant clinical and logistical details prior to study implementation. During study implementation, partners should use good communication practices and document and maintain a record of any changes to the original plan. Presentation of the study results should be tailored to the particular audience, with the appropriate partner leading the presentation based on its format and audience. CONCLUSION: Partners from patient advocacy organizations, the pharmaceutical industry, and academia can effectively collaborate on benefit-risk patient preference studies with sufficient planning and ongoing communication. This article is a call for action for other organizations to engage in sharing of experiences regarding effective partnering in quantifying patient preferences in medical product development.

What are people willing to pay for whole-genome sequencing information, and who decides what they receive?

PURPOSE: Whole-genome sequencing (WGS) can be used as a powerful diagnostic tool as well as for screening, but it may lead to anxiety, unnecessary testing, and overtreatment. Current guidelines suggest reporting clinically actionable secondary findings when diagnostic testing is performed. We examined preferences for receiving WGS results. METHODS: A US nationally representative survey (n = 410 adults) was used to rank preferences for who decides (an expert panel, your doctor, you) which WGS results are reported. We estimated the value of information about variants with varying levels of clinical usefulness by using willingness to pay contingent valuation questions. RESULTS: The results were as follows: 43% preferred to decide themselves what information is included in the WGS report. 38% (95% confidence interval (CI): 33-43%) would not pay for actionable variants, and 3% (95% CI: 1-5%) would pay more than $1,000. 55% (95% CI: 50-60%) would not pay for variants for which medical treatment is currently unclear, and 7% (95% CI: 5-9%) would pay more than $400. CONCLUSION: Most people prefer to decide what WGS results are reported. Despite valuing actionable information more, some respondents perceive that genetic information could negatively impact them. Preference heterogeneity for WGS information should be considered in the development of policies, particularly to integrate patient preferences with personalized medicine and shared decision making.Genet Med 18 12, 1295-1302.

Choice Experiments to Quantify Preferences for Health and Healthcare: State of the Practice.

Stated-preference methods increasingly are used to quantify preferences in health economics, health technology assessment, benefit-risk analysis and health services research. The objective of stated-preference studies is to acquire information about trade-off preferences among treatment outcomes, prioritization of clinical decision criteria, likely uptake or adherence to healthcare products and acceptability of healthcare services or policies. A widely accepted approach to eliciting preferences is discrete-choice experiments. Patient, physician, insurant or general-public respondents choose among constructed, experimentally controlled alternatives described by decision-relevant features or attributes. Attributes can represent complete health states, sets of treatment outcomes or characteristics of a healthcare system. The observed pattern of choice reveals how different respondents or groups of respondents implicitly weigh, value and assess different characteristics of treatments, products or services. An important advantage of choice experiments is their foundation in microeconomic utility theory. This conceptual framework provides tests of internal validity, guidance for statistical analysis of latent preference structures, and testable behavioural hypotheses. Choice experiments require expertise in survey-research methods, random-utility theory, experimental design and advanced statistical analysis. This paper should be understood as an introduction to setting up a basic experiment rather than an exhaustive critique of the latest findings and procedures. Where appropriate, we have identified topics of active research where a broad consensus has not yet been established.

Patient Engagement at a Tipping Point-The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, "Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products".

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.

Valuations of genetic test information for treatable conditions: the case of colorectal cancer screening.

BACKGROUND: The value of the information that genetic testing services provide can be questioned for insurance-based health systems. The results of genetic tests oftentimes may not lead to well-defined clinical interventions; however, Lynch syndrome, a genetic mutation for which carriers are at an increased risk for colorectal cancer, can be identified through genetic testing, and meaningful health interventions are available via increased colonoscopic surveillance. Valuations of test information for such conditions ought to account for the full impact of interventions and contingent outcomes. OBJECTIVES: To conduct a discrete-choice experiment to elicit individuals' preferences for genetic test information. METHODS: A Web-enabled discrete-choice experiment survey was administered to a representative sample of US residents aged 50 years and older. In addition to specifying expenditures on colonoscopies, respondents were asked to make a series of nine selections between two hypothetical genetic tests or a no-test option under the premise that a relative had Lynch syndrome. The hypothetical genetic tests were defined by the probability of developing colorectal cancer, the probability of a false-negative test result, privacy of the result, and out-of-pocket cost. A model specification identifying necessary interactions was derived from assumptions of risk behavior and the decision context and was estimated using random-parameters logit. RESULTS: A total of 650 respondents were contacted, and 385 completed the survey. The monetary equivalent of test information was approximately $1800. Expenditures on colonoscopies to reduce mortality risks affected valuations. Respondents with lower income or who reported being employed significantly valued genetic tests more. CONCLUSION: Genetic testing may confer benefits through the impact of subsequent interventions on private individuals.

Adolescent girls' preferences for HPV vaccines: a discrete choice experiment.

PURPOSE: To measure adolescent girls' preferences over features of human papillomavirus (HPV) vaccines in order to provide quantitative estimates of the perceived benefits of vaccination and potential vaccine uptake. DESIGN/METHODOLOGY/APPROACH: A discrete choice experiment (DCE) survey was developed to measure adolescent girls' preferences over features of HPV vaccines. The survey was fielded to a U.S. sample of 307 girls aged 13-17 years who had not yet received an HPV vaccine in June 2008. FINDINGS: In a latent class logit model, two distinct groups were identified--one with strong preferences against vaccination which largely did not differentiate between vaccine features, and another that was receptive to vaccination and had well-defined preferences over vaccine features. Based on the mean estimates over the entire sample, we estimate that girls' valuation of bivalent and quadrivalent HPV vaccines ranged between $400 and $460 in 2008, measured as willingness-to-pay (WTP). The additional value of genital warts protection was $145, although cervical cancer efficacy was the most preferred feature. We estimate maximum uptake of 54-65%, close to the 53% reported for one dose in 2011 surveillance data, but higher than the 35% for three doses in surveillance data. RESEARCH LIMITATIONS/IMPLICATIONS: We conclude that adolescent girls do form clear opinions and some place significant value on HPV vaccination, making research on their preferences vital to understanding the determinants of HPV vaccine demand. ORIGINALITY/VALUE: DCE studies may be used to design more effective vaccine-promotion programs and for reassessing public health recommendations and guidelines as new vaccines are made available.

Healthy-days time equivalents for outcomes of acute rotavirus infections.

Rotavirus is the most common cause of severe gastroenteritis in infants and young children worldwide. Health-state utility measures used in economic evaluations of rotavirus vaccines do not reflect differences between mild and severe symptoms of rotavirus gastroenteritis and, therefore, do not adequately capture preferences for non-fatal outcomes associated with rotavirus common in industrialized countries. This paper describes the development and results of a survey specifically designed to develop quality-adjusted time equivalents for rotavirus gastroenteritis among a sample of parents with young children in the United States as an alternative to conventional QALY measures in assessing cost-effectiveness.