Quality of Life in Economic Evaluations of Health

__Abstract__ Health care expenditures have increased rapidly over the last decades in the Netherlands, in absolute terms and as percentage of gross domestic product. Curbing the rising health care costs has proven to be a very sensitive and complex societal issue. An important driver of rising costs is the availability and use of new and expensive medical technologies, causing a greater number of patients to be treated with more expensive interventions. Typically, these interventions do not only increase expenditures but also contribute to societal health and well-being. Since health care expenditures are high on the political agenda, policy makers are interested in the relative effectiveness and efficiency of new medical interventions: do they achieve larger health effects than other treatments, and if so, at what additional costs? Economic evaluations address this question. In economic evaluations, costs and effects of alternative medical interventions are compared, to see whether the new intervention offers good ‘value for money’. Researchers that which to apply economic evaluations to health care interventions face several methodological challenges. This thesis deals with one particular challenge: the measurement of the benefit of health care interventions in terms of quality of life

From Good to Better: New Dutch Guidelines for Economic Evaluations in Healthcare

Many countries have national guidelines for performing economic evaluations in healthcare.1 These guidelines should ensure the comparability and quality of such evaluations, which should facilitate making well informed policy decisions regarding reimbursement of interventions. Given the developments in both the methodology and policy context of economic evaluation of healthcare interventions, these guidelines require periodical revision. Recently, the Dutch National Health Care Institute issued new guidance for economic evaluations in healthcare [1]. The new guidelines update and replace three separately published previous guidelines: those for pharmacoeconomic evaluation (latest version 2006), outcomes research (latest version 2008) as well as the Dutch costing manual (latest version 2010). In this editorial, we highlight the distinguishing features of the new Dutch guidelines. Moreover, we highlight which developments, in our opinion, are desirable in coming updates, but are still in development or controversial

Benefits of early highly effective versus escalation treatment strategies in relapsing multiple sclerosis estimated using a treatment-sequence model

BACKGROUND: Uncertainty about disproportionate impact on health care budgets limits implementation of early highly effective treatment (EHT) in multiple sclerosis (MS).OBJECTIVE: To estimate cost-effectiveness of escalation versus EHT disease-modifying treatment (DMT) sequences.METHODS: Using a health-economic approach, we analysed health benefits (relapse rate reduction, disability prevention), direct/indirect DMT and societal costs of escalation versus EHT DMT sequences. In scenario analyses, we allowed (1) earlier use of alemtuzumab (ALE) and (2) a single retreatment with cladribine (CLA).RESULTS: In our model, we showed that the ratio between costs and quality-adjusted life years (QALYs) for the most cost-effective EHT and escalation sequence results into a similar net health benefit with higher costs and also higher QALYs associated with an EHT versus escalation strategy. Earlier use of ALE is more cost-effective than in later lines, even when aggravating the impact of its side-effects tenfold. Retreatment with CLA was more cost-effective in both escalation and EHT sequences.CONCLUSIONS: Certain EHT sequences are equally cost-effective to escalation sequences and are likely to result in more health at uncertain additional costs. The favourable cost-benefit ratio of CLA and ALE suggests that a wider application of affordable highly effective therapies could promote the cost-effectiveness both EHT and escalation approaches.</p

Psychometric evaluation of the Dutch translation of the Overall Assessment of the Speaker’s Experience of Stuttering for adults (OASES-A-D)

Abstract: The Overall Assessment of the Speaker’s Experience of Stuttering for adults (OASES-A; Yaruss & Quesal, 2006, 2010) is a patient-reported outcome measure that was designed to provide a comprehensive assessment of “the experience of the stuttering disorder from the perspective of individuals who stutter” (Yaruss & Quesal, 2006, p.90). This paper reports on the translation process and evaluates the psychometric performance of a Dutch version of the OASES-A. Translation of the OASES-A into Dutch followed a standard forward and backward translation process. The Dutch OASES-A (OASES-A-D) was then administered to 138 adults who stutter. A subset of 91 respondents also evaluated their speech on a 10-point Likert scale. For another subset of 45 respondents, a clinician-based stuttering severity rating on a 5-point Likert scale was available. Thirty-two of the respondents also completed the Dutch S-24 scale (Brutten & Vanryckeghem, 2003). The OASES-A-D showed acceptable item properties. No ceiling effects were observed. For 30 out of 100 items, most of which were in Section IV (Quality of Life), floor effects were observed. Cronbach’s alpha coefficients for all sections and subsections surpassed the 0.70 criterion of good internal consistency and reliability. Concurrent validity was moderate to high. Construct validity was confirmed by distinct scores on the OASES-A-D for groups with different levels of stuttering severity as rated by the speakers themselves or by clinicians. These results suggest that the OASES-A-D is a reliable and valid measure that can be used to assess the impact of stuttering on Dutch adults who stutter

Benefits of early highly effective versus escalation treatment strategies in relapsing multiple sclerosis estimated using a treatment-sequence model

BACKGROUND: Uncertainty about disproportionate impact on health care budgets limits implementation of early highly effective treatment (EHT) in multiple sclerosis (MS).OBJECTIVE: To estimate cost-effectiveness of escalation versus EHT disease-modifying treatment (DMT) sequences.METHODS: Using a health-economic approach, we analysed health benefits (relapse rate reduction, disability prevention), direct/indirect DMT and societal costs of escalation versus EHT DMT sequences. In scenario analyses, we allowed (1) earlier use of alemtuzumab (ALE) and (2) a single retreatment with cladribine (CLA).RESULTS: In our model, we showed that the ratio between costs and quality-adjusted life years (QALYs) for the most cost-effective EHT and escalation sequence results into a similar net health benefit with higher costs and also higher QALYs associated with an EHT versus escalation strategy. Earlier use of ALE is more cost-effective than in later lines, even when aggravating the impact of its side-effects tenfold. Retreatment with CLA was more cost-effective in both escalation and EHT sequences.CONCLUSIONS: Certain EHT sequences are equally cost-effective to escalation sequences and are likely to result in more health at uncertain additional costs. The favourable cost-benefit ratio of CLA and ALE suggests that a wider application of affordable highly effective therapies could promote the cost-effectiveness both EHT and escalation approaches.</p

Mapping onto Eq-5 D for patients in poor health

<p>Abstract</p> <p>Background</p> <p>An increasing amount of studies report mapping algorithms which predict EQ-5 D utility values using disease specific non-preference-based measures. Yet many mapping algorithms have been found to systematically overpredict EQ-5 D utility values for patients in poor health. Currently there are no guidelines on how to deal with this problem. This paper is concerned with the question of why overestimation of EQ-5 D utility values occurs for patients in poor health, and explores possible solutions.</p> <p>Method</p> <p>Three existing datasets are used to estimate mapping algorithms and assess existing mapping algorithms from the literature mapping the cancer-specific EORTC-QLQ C-30 and the arthritis-specific Health Assessment Questionnaire (HAQ) onto the EQ-5 D. Separate mapping algorithms are estimated for poor health states. Poor health states are defined using a cut-off point for QLQ-C30 and HAQ, which is determined using association with EQ-5 D values.</p> <p>Results</p> <p>All mapping algorithms suffer from overprediction of utility values for patients in poor health. The large decrement of reporting 'extreme problems' in the EQ-5 D tariff, few observations with the most severe level in any EQ-5 D dimension and many observations at the least severe level in any EQ-5 D dimension led to a bimodal distribution of EQ-5 D index values, which is related to the overprediction of utility values for patients in poor health. Separate algorithms are here proposed to predict utility values for patients in poor health, where these are selected using cut-off points for HAQ-DI (> 2.0) and QLQ C-30 (< 45 average of QLQ C-30 functioning scales). The QLQ-C30 separate algorithm performed better than existing mapping algorithms for predicting utility values for patients in poor health, but still did not accurately predict mean utility values. A HAQ separate algorithm could not be estimated due to data restrictions.</p> <p>Conclusion</p> <p>Mapping algorithms overpredict utility values for patients in poor health but are used in cost-effectiveness analyses nonetheless. Guidelines can be developed on when the use of a mapping algorithms is inappropriate, for instance through the identification of cut-off points. Cut-off points on a disease specific questionnaire can be identified through association with the causes of overprediction. The cut-off points found in this study represent severely impaired health. Specifying a separate mapping algorithm to predict utility values for individuals in poor health greatly reduces overprediction, but does not fully solve the problem.</p

Distributional consequences of including survivor costs in economic evaluations

Medical interventions that increase life expectancy of patients result in additional consumption of non‐medical goods and services in ‘added life years’. This paper focuses on the distributional consequences across socio‐economic groups of including these costs in cost effectiveness analysis. In that context, it also highlights the role of remaining quality of life and household economies of scale. Data from a Dutch household spending survey was used to estimate non‐medical consumption and household size by age and educational attainment. Estimates of non‐medical consumption and household size were combined with life tables to estimate what the impact of including non‐medical survivor costs would be on the incremental cost effectiveness ratio (ICER) of preventing a death at a certain age. Results show that including non‐medical survivor costs increases estimated ICERs most strongly when interventions are targeted at the higher educated. Adjusting for household size (lower educated people less often live additional life years in multi‐person households) and quality of life (lower educated people on average spend added life years in poorer health) mitigates this difference. Ignoring costs of non‐medical consumption in economic evaluations implicitly favors interventions targeted at the higher educated and thus potentially amplifies socio‐economic inequalities in health