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

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

Can time heal all wounds? An empirical assessment of adaptation to functional limitations in an older population

Chronic diseases and functional limitations may have serious and persistent consequences for one's quality of life (QoL). Over time, however, their negative impact on QoL may diminish because of adaptation. Understanding how much people adapt helps to correctly separate the effects attributable to interventions from those arising from adaptation and thus facilitates a better estimation of the effects of disease and treatment on QoL. To date, however, there is little empirical evidence on adaptation in older populations. In particular, it is unclear to which extent dimensions of QoL like health and overall experience with life are influenced by adaptation. This paper studies adaptation to functional limitations in 5000 respondents of the Survey of Health, Ageing and Retirement in Europe (SHARE) who develop disabilities during the span of the 5 waves of data collection between 2004 and 2015. To examine the association between time since the onset of functional limitations and self-perceived health and life satisfaction, a fixed effects ordered logit model is used. We found evidence supporting adaptation in life satisfaction, corresponding to a return to pre-onset levels of life satisfaction. Also in the self-perceived health dimension, adaptation does occur, but it does not occur fast enough to offset the negative changes in underlying health. This means that observational studies that measure one of these two outcome measures should be aware that part or all of the effects found are due to adaptation

Burden of providing informal care for patients with atrial fibrillation

Objectives: Patients with atrial fibrillation (AF) have rapid and irregular heart rates, increasing the risk of comorbidities and mortality. Next to formal medical care, many patients receive informal care from their social environment. The objective of this study was to examine the well-being and economic burden of providing informal care to patients with AF in the UK, Italy, and Germany. Methods: Caregivers of patients with AF completed an online survey based on the iMTA Valuation of Informal Care Questionnaire, with questions about their caregiving situation, perceived burden of caregiving, and absence from work due to health problems resulting from caregiving. Care-related quality-of-life utilities were calculated using the Care-related Quality of Life instrument and associated tariffs. Societal costs of caregiving were calculated based on the proxy good method. Results: A total of 585 caregivers participated in this study. On average, caregivers provided 33 hours of informal care per week to patients (SD 29 hours). On a scale from 0 to 10, their self-rated burden was 5.4. The average Care-related Quality of Life utility was 72. Caregivers primarily indicated problems with daily activities, mental health, and physical health. Still, the vast majority of caregivers (87%) derived fulfillment from providing care. Weekly societal costs of caregiving were on average V636. Comorbidities contributed substantially to the caregiver time and burden. Conclusions: Caring for a patient with AF is associated with substantial objective and subjective burden, but a

Estimating Informal Caregiving Time from Patient EQ-5D Data: The Informal CARE Effect (iCARE) Tool

Background: Families and friends provide a considerable proportion of care for patients and elderly people. Caregiving can have substantial effects on caregivers’ lives, health, and well-being. However, because clinical trials rarely assess these effects, no information on caregiver burden is available when evaluating the cost effectiveness of treatments. Objective: This study develops an algorithm for estimating caregiver time using information that is typically available in clinical trials: the EQ-5D scores of patients and their gender. Methods: Four datasets with a total of 8012 observations of dyads of caregivers and a gamma model with a log-link estimated with the Bayesian approach were used to estimate the statistical association between patient scores on the EQ-5D-3L dimensions and the numbers of hours of care provided by caregivers during the previous week. The model predicts hours of care as mean point estimates with 95% credible intervals or entire distributions. Results: Model predictions of hours of care based on the five EQ-5D dimensions ranged from 13.06 (12.7–14.5) h/week for female patients reporting no health problems but receiving informal care to 52.82 (39.38–66.26) for male patients with the highest level of problems on all EQ-5D dimensions. Conclusions: The iCARE algorithm developed in this study allows researchers who only have patient-level EQ-5D data to estimate the mean hours of informal care received per week, including a 95% Bayesian credible interval. Caregiver time can be multiplied with a monetary value for caregiving, enabling the inclusion of informal care costs in economic evaluations. We recommend using the tool for samples that fall within the confidence intervals of the characteristics of our samples: men (age range 47.0–104.2 years), women (age range 55–103 years)

Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision

The emergence of gene therapies challenge health economists to evaluate interventions that are often provided to a small patient population with a specif

Health-related quality of life and its determinants in patients with metastatic renal cell carcinoma

__Purpose:__ Based on improvements of progression-free survival (PFS), new agents for metastatic renal cell carcinoma (mRCC) have been approved. It is assumed that one of the benefits is a delay in health-related quality of life (HRQoL) deterioration as a result of a delay in progression of disease. However, little data are available supporting this relationship. This study aims to provide insight into the most important determinants of HRQoL (including progression of disease) of patients with mRCC. __Methods:__ A patient registry (PERCEPTION) was created to evaluate treatment of patients with (m)RCC in the Netherlands. HRQoL was measured, using the EORTC QLQ-C30 and EQ-5D-5L, every 3 months in the first year of participation in the study, and every 6 months in the second year. Participation started as soon as possible following a diagnosis of (m)RCC. Random effects models were used to study associations between HRQoL and patient and disease characteristics, symptoms and treatment. __Results:__ Eighty-seven patients with mRCC completed 304 questionnaires. The average EORTC QLQ-C30 global health status was 69 (SD, 19) before progression and 61 (SD, 22) after progression of disease. Similarly, the average EQ-5D utility was 0.75 (SD, 0.19) before progression and 0.66 (SD, 0.30) after progression of disease. The presence of fatigue, pain, dyspnoea, and the application of radiotherapy were associated with significantly lower EQ-5D utilities. __Conclusions:__ Key drivers for reduced HRQoL in mRCC are disease symptoms. Since symptoms increase with progression of disease, targeted therapies that increase PFS are expected to postpone reductions in HRQoL in mRCC

Severity-Adjusted Probability of Being Cost Effective

Background In the context of priority setting, a diferential cost-efectiveness threshold can be used to refect a higher societal willingness to pay for quality-adjusted life-year gains in the worse of. However, uncertainty in the estimate of severity can lead to problems when evaluating the outcomes of cost-efectiveness analyses. Objectives This study standardizes the assessment of severity, integrates its uncertainty with the uncertainty in cost-efectiveness results and provides decision makers with a new estimate: the severity-adjusted probability of being cost efective. Methods Severity is expressed in proportional and absolute shortfall and estimated using life tables and country-specifc EQ-5D values. We use the three severity-based cost-efectiveness thresholds (€20.000, €50.000 and €80.000, per QALY) adopted in The Netherlands. We exemplify procedures of integrating uncertainty with a stylized example of a hypothetical oncology treatment. Results Applying our methods, taking into account the uncertainty in the cost-efectiveness results and in the estimation of severity identifes the likelihood of an intervention being cost efective when there is uncertainty about the appropriate severity-based cost-efectiveness threshold. Conclusions Higher willingness-to-pay thresholds for severe diseases are implemented in countries to refect societal concerns for an equitable distribution of resources. However, the estimates of severity are uncertain, patient populations are heterogeneous, and this can be accounted for with the severity-adjusted probability of being cost efective proposed in this study. The application to the Netherlands suggests that not adopting the new method could result in incorrect decisions in the reimbursement of new health technologies

A cost-effectiveness threshold based on the marginal returns of cardiovascular hospital spending

Traditionally, threshold levels of cost‐effectiveness have been derived from willingness‐to‐pay studies, indicating the consumption value of health (v‐thresholds). However, it has been argued that v‐thresholds need to be supplemented by so‐called k‐thresholds, which are based on the marginal returns to health care. The objective of this research is to estimate a k‐threshold based on the marginal returns to cardiovascular disease (CVD) hospital care in the Netherlands. To estimate a k‐threshold for hospital care on CVD, we proceed in two steps: First, we estimate the impact of hospital spending on mortality using a Bayesian regression modelling framework, using data on CVD mortality and CVD hospital spending by age and gender for the period 1994–2010. Second, we use life tables in combination with quality of life data to convert these estimates into a k‐threshold expressed in euros per quality‐adjusted life year gained. Our base case estimate resulted in an estimate of 41,000 per quality‐adjusted life year gained. In our sensitivity analyses, we illustrated how the incorporation of prior evidence into the estimation pushes estimates downwards. We conclude that our base case estimate of the k‐threshold may serve as a benchmark value for decision making in the Netherlands as well as for future research regarding k‐thresholds