The History and Future of the “ISPOR Value Flower”: Addressing Limitations of Conventional Cost-Effectiveness Analysis

ObjectivesSince its publication as part of the 2018 ISPOR Special Task Force (STF) on US Value Assessments, the “ISPOR value flower,” with its petals highlighting elements that may be overlooked or underappreciated in conventional drug value assessments, has been discussed and debated. We review the history of the value flower, describe recent developments, and consider implications for future value assessments.MethodsWe discuss various antecedents to the value flower, as well as conceptual and empirical articles published in the past 4 years.ResultsSince the publication of the ISPOR STF report, researchers have provided more rigorous theoretical and mathematical foundations for certain novel value elements (eg, severity of illness, value of insurance, value of hope) through “generalized risk-adjusted cost-effectiveness analysis,” which incorporates risk aversion in people’s preferences and uncertainty in treatment outcomes. Empirical estimates are also emerging to support key elements, such as insurance value, real option value, value of hope, and value of knowing. Although health technology assessment bodies have applied or are considering certain elements (eg, severity modifiers to cost-effectiveness thresholds), other elements have yet to gain traction.ConclusionsFive years after the STF began its work, the development of novel value measures continues to evolve. Although it is encouraging to see supporting empirical studies emerging, more are needed. Additional efforts are also needed to illustrate how the estimates can be used in the deliberative processes that are integral to health technology assessments.     

A Bayesian Approach for Incorporating the EQ-5D Visual Analog Scale When Estimating the Health-Related Quality of Life

ObjectivesThe EuroQoL 3-level version of EQ-5D and 5-level version of EQ-5D questionnaires are often used to quantify health states. They include ordinal responses across 5 health dimensions (EQ-5D index) and an EQ-visual analog scale (EQ-VAS) overall health rating. We investigated the value of incorporating the EQ-VAS to update health utility estimates using a Bayesian framework.MethodsWe created a joint bivariate normal EQ-VAS and EQ-5D index utility model and compared this to a univariate normal EQ-5D index utility model. We tested these models for 1026 Sri Lankan patients with chronic kidney disease and 94 Australian patients with wounds. We validated our approach by simulating EQ-VAS and EQ-5D index responses and applying our Bayesian model and then comparing the modeled estimates to our observed data.ResultsThe combined model showed a reduction in estimate uncertainty for all respondents. Compared with the EQ-5D index-only model, the mean utility for Sri Lankan respondents dropped from 0.556 (0.534-0.579) to 0.540 (0.521-0.559) in men and increased from 0.489 (0.461-0.518) to 0.528 (0.506-0.550) in women, with reduced credible interval width by 13% and 23%, respectively. The mean utility in Australian respondents moved from 0.715 (0.633-0.800) to 0.716 (0.652-0.782) in men, and 0.652 (0.581-0.723) to 0.652 (0.593-0.711) in women, with reduced credible interval width by 23% and 17%, respectively. The credible interval width for simulated data also narrowed, ranging from 8.3 to 8.5%.ConclusionsIncluding the EQ-VAS through Bayesian methods can add value by reducing requisite sample sizes and decision uncertainty using small amounts of additional data that is often collected but rarely used.     

Suprachoroidal Injection of Triamcinolone Acetonide Injectable Suspension for the Treatment of Macular Edema Associated With Uveitis in the United States: A Cost-Effectiveness Analysis

ObjectivesSuprachoroidal injection of triamcinolone acetonide is the first Food and Drug Administration–approved treatment for macular edema associated with uveitis. A cost-effectiveness analysis was performed comparing this treatment with best supportive care (BSC) for the management of this indication from US Medicare and commercial payer perspectives.MethodsA patient-level simulation was developed per the patient characteristics and changes in best-corrected visual acuity letter scores observed in a phase III study of triamcinolone acetonide (PEACHTREE). The wholesale acquisition cost of triamcinolone acetonide was $1650/injection; suprachoroidal injection cost was assumed at $200/injection. Healthcare costs were informed by a US claims–based analysis. Mortality risk associated with severe vision loss and blindness was modeled by applying a hazard ratio to all-cause mortality rates of the US general population. Health-related quality of life weights, obtained from a regression model fitted to the Visual Function Questionnaire-25 data from PEACHTREE, were applied based on the best-corrected visual acuity scores of both eyes. Costs (2020 US dollar) and benefits were discounted at 3% annually. Incremental cost-effectiveness ratios were estimated over a 10-year horizon.ResultsIn the base-case, the incremental cost-effectiveness ratio comparing triamcinolone acetonide with BSC was $28 479 per quality-adjusted life-year gained. The wholesale acquisition cost for triamcinolone acetonide for suprachoroidal use was ～68%, ～56%, and ～27% below the willingness-to-pay thresholds of $150 000, $100 000, and $50 000 per quality-adjusted life-year gained, respectively. Results were robust in sensitivity and scenario analyses.ConclusionsTriamcinolone acetonide for suprachoroidal use is cost-effective compared with BSC for patients with macular edema associated with uveitis.     

Antimicrobial Resistance: Is Health Technology Assessment Part of the Solution or Part of the Problem?

Antimicrobial resistance is a serious challenge to the success and sustainability of our healthcare systems. There has been increasing policy attention given to antimicrobial resistance in the last few years, and increased amounts of funding have been channeled into funding for research and development of antimicrobial agents. Nevertheless, manufacturers doubt whether there will be a market for new antimicrobial technologies sufficient to enable them to recoup their investment. Health technology assessment (HTA) has a critical role in creating confidence that if valuable technologies can be developed they will be reimbursed at a level that captures their true value. We identify 3 deficiencies of current HTA processes for appraising antimicrobial agents: a methods-centric approach rather than problem-centric approach for dealing with new challenges, a lack of tools for thinking about changing patterns of infection, and the absence of an approach to epidemiological risks. We argue that, to play their role more effectively, HTA agencies need to broaden their methodological tool kit, design and communicate their analysis to a wider set of users, and incorporate long-term policy goals, such as containing resistance, as part of their evaluation criteria alongside immediate health gains.     

Reference Values and Psychometric Properties of the Quality of Life After Traumatic Brain Injury-Overall Scale in Italy,The Netherlands,and the United Kingdom

ObjectivesThe Quality of Life after Brain Injury-Overall Scale (QOLIBRI-OS) is a short screening instrument for assessing disease-specific health-related quality of life (HRQoL) after traumatic brain injury. To date, no reference values are available for the QOLIBRI-OS in general populations. Thus, this study aimed to establish reference values for the QOLIBRI-OS in general population samples from Italy, The Netherlands, and the United Kingdom.MethodsData were collected using an online survey. The total sample comprised 11759 participants, consisting of 3549 Italian, 3564 Dutch, and 4646 British subjects. In this sample, 49% of the total sample did not report any health complaints, whereas 51% had at least 1 chronic health condition. Reference values were deduced for the QOLIBRI-OS for health-condition–related samples and total general population samples per country. To ensure the comparability of these values, measurement invariance was assessed using a multigroup confirmatory factor analysis. Covariates characterizing the reference values were selected with the help of regression analyses.ResultsThe confirmatory factor analysis confirmed that the QOLIBRI-OS scores measured the same traumatic brain injury–specific HRQoL construct across the 3 countries. Healthy individuals reported significantly higher HRQoL than individuals with at least 1 chronic health condition. Older age and higher education levels were significantly associated with higher HRQoL.ConclusionsBecause the reference values displayed differences in terms of age and education level across the 3 countries, we recommend using country-specific reference values stratified by sociodemographic and health status in research and clinical practice.     

Towards an integrated theoretical framework for understanding water insecurity and gender-based violence in Low-and middle-income countries (LMICs)

Disparities in access to basic needs and resources such as water is largely borne out of power imbalance across scale. In examining these power dynamics in the context of health inequalities, scholars have deployed Feminist political ecology analytical framework to situate gender and other forms of vulnerability as emerging from unequal power relations, and political ecology of health to emphasise the health implications of inherent relational power in the distribution of resources. Although appealing, the two theoretical frameworks over time have proven to be limiting in the study of intersectional vulnerabilities such as gender-based violence and water insecurity which reflect multiple dimensions of unequal power structures. This study expands the theoretical space for the study of inequalities in health geography by demonstrating the utility of incorporating feminist political ecology with political ecology of health to form an integrated theoretical framework – Feminist Political Ecology of Health (FPEH). This proposed theoretical framework gives guidance for engaging with a suite of questions and methods related to multifaceted problems such as water insecurity and gender based-violence. The paper highlights these theoretical issues and then discusses how FPEH can enrich research on water security and gender-based violence in Low-and middle-income countries (LMICs).     

Veridical causal inference using propensity score methods for comparative effectiveness research with medical claims

Health Services and Outcomes Research Methodology - Medical insurance claims are becoming increasingly common data sources to answer a variety of questions in biomedical research. Although...     

Quality and Safety in Long-Term Care in Ontario: The Impact of Language Discordance

ObjectivesThis study compared quality indicators across linguistic groups and sought to determine whether disparities are influenced by resident-facility language discordance in long-term care.DesignPopulation-based retrospective cohort study using linked databases.Setting and ParticipantsRetrospective cohort of newly admitted residents of long-term care facilities in Ontario, Canada, between 2010 and 2016 (N=47,727). Individual residents' information was obtained from the Resident Assessment Instrument Minimum Data Set (RAI-MDS) to determine resident's primary language, clinical characteristics, and health care indicators.MeasuresMain covariates of interest were primary language of the resident and predominant language of the long-term care facility, which was determined using the French designation status as defined in the French Language Services Act. Primary outcomes were a set of quality and safety indicators related to long-term care: worsening of depression, falls, moderate-severe pain, use of antipsychotic medication, and physical restraints. Multivariable logistic regression models were used to assess the impact of resident's primary language, facility language, and resident-facility language discordance on each quality indicator.ResultsOverall, there were few differences between francophones and anglophones for quality and safety indicators. Francophones were more likely to report pain (10.9% vs 9.9%; P = .001) and be physically restrained (7.3% vs 5.2%; P < .001), whereas a greater proportion of anglophones experienced worsening of depressive symptoms (24.0% vs 22.9%; P = .001). However, quality indicators were generally worse for francophones in Non-Designated facilities, except for pain, which was more commonly reported by francophones in French-Designated facilities. Anglophones were more likely to be physically restrained in French-Designated facilities (6.7% vs 5.1%; P < .001).Conclusions and ImplicationsFor francophones, quality indicators tended to be worse in the presence of resident-facility language discordance. However, these findings did not persist after adjusting for individual- and facility-level characteristics, suggesting that the disparities observed at the population level cannot be attributed to linguistic factors alone.