évaluation médico économique

Background and Objectives Valuing children’s health states for use in economic evaluations is globally relevant and is of particular relevance in jurisdictions where a cost-utility analysis is the preferred form of analysis for decision making. Despite this, the challenges with valuing child health mean that there are many remaining questions for debate about the approach to elicitation of values. The aim of this paper was to identify and describe the methods used to value children’s health states and the specific issues that arise in the use of these methods. Conclusions Differences in reporting limited the conclusions that can be formed about which methods are most suitable for eliciting preferences for children’s health and the influence of differing perspectives and values. Difficulties encountered in drawing conclusions from the data (such as lack of consensus and poor reporting making it difficult for users to choose and interpret available values) suggest that reporting guidelines are required to improve the consistency and quality of reporting of studies that value children’s health using preference-based techniques.

Facilitating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework – Corrigendum - Volume 38 Issue 1

Background There is evidence to suggest that the proportion of missing values is slightly higher in the older population resulting in lower completion rates of the EQ-5D. However, existing studies rarely provide a within-sample comparison of feasibility properties across age groups to quantify this difference. Hence, this study examines feasibility properties of the EQ-5D-3L and 5L in the general population and explores the impact of age on the completion of EQ-5D instruments. Methods We pool five waves from the English GP Patient Survey, where respondents self-report their health in either EQ-5D-3L or 5L. Descriptive analysis was undertaken to analyse the distribution and proportion of missing values and completion rates stratified by age and EQ-5D version; logistic regression models were specified to quantify the impact of age, gender and potential long-term conditions on the completion of each of the EQ-5D instruments. Results The total sample comprises ~ 4.36 million observations, of which 2.88 million respondents report their health in 5L and 1.47 million in 3L, respectively. Respondents over 64 years have slightly more missing values in each dimension than younger respondents. The highest share was observed for the oldest age group in the dimension anxiety/depression (3L 9.1% vs. 5L 7.6%), but was otherwise below 5%. Consequently, completion rates (observed and predicted) decreased with older age and at a higher rate after the age of 64; this was more pronounced for the 3L. Conclusion Evidence from our study suggests that both the EQ-5D-3L and 5L have good feasibility properties. In comparison to younger populations there appears to be a higher proportion of respondents with incomplete responses beyond the age of 64 years. Overall, the 5L version compares more favourably in terms of missing values, completion rates as well as with regard to the expected probability of an incomplete descriptive system.

In the valuation of EQ-5D-Y-3L, adult respondents are asked to complete composite time trade-off (cTTO) tasks for a 10-year-old child. Earlier work has shown that cTTO utilities elicited in such a child perspective are generally higher than when adults take their own perspective. We explore how differences in time preference in child and adult perspectives could explain this effect. Furthermore, as cTTO valuation in a child perspective involves explicit consideration of immediate death for a child, we also consider how cTTO utilities could be affected by decision-makers lexicographically avoiding death in children. We report the results of an experiment in which 219 respondents valued 5 health states in both adult and child perspectives with either a standard cTTO or a lead-time TTO only approach, in which immediate death is less focal. Time preferences were measured in both perspectives. Our results suggest that utilities were lower when lead-time TTO, rather than cTTO, was used. We find large heterogeneity in time preference in both perspectives, with predominantly negative time preference. The influence of time preferences on utilities, however, was small, and correcting for time preferences did not reduce differences between utilities elicited in both perspectives. Surprisingly, we found more evidence for differences in utilities between adult and child perspectives when lead-time TTO was used. Overall, these results suggest that time and lexicographic preferences affect time trade-off valuation in child and adult perspectives, but are not the explanation for differences between these perspectives. We discuss the implications of our findings for EQ-5D-Y-3L valuation.

The Government of Canada plans to implement new controls on the prices of patented drugs sold in Canada. The literature indicates that such controls d…

Discrete choice experiments (DCEs) are increasingly used to elicit preferences for health and healthcare. Although many applications assume preference…

In economic evaluations, quality of life is measured using patient-reported outcome measures (PROMs), such as the EQ-5D-5L. A key assumption for the v…

The development process of economic evaluation guidelines in low- and middle-income countries: a systematic review - Volume 38 Issue 1

This study aimed to investigate how multicriteria decision analysis (MCDA) could complement cost-effectiveness analysis (CEA) to support investment de…

Guidelines of economic evaluations suggest that probabilistic analysis (using probability distributions as inputs) provides less biased estimates than deterministic analysis (using point estimates) owing to the non-linear relationship of model inputs and model outputs. However, other factors can also impact the magnitude of bias for model results. We evaluate bias in probabilistic analysis and deterministic analysis through three simulation studies. The simulation studies illustrate that in some cases, compared with deterministic analyses, probabilistic analyses may be associated with greater biases in model inputs (risk ratios and mean cost estimates using the smearing estimator), as well as model outputs (life-years in a Markov model). Point estimates often represent the most likely value of the parameter in the population, given the observed data. When model parameters have wide, asymmetric confidence intervals, model inputs with larger likelihoods (e.g., point estimates) may result in less bias in model outputs (e.g., costs and life-years) than inputs with lower likelihoods (e.g., probability distributions). Further, when the variance of a parameter is large, simulations from probabilistic analyses may yield extreme values that tend to bias the results of some non-linear models. Deterministic analysis can avoid extreme values that probabilistic analysis may encounter. We conclude that there is no definitive answer on which analytical approach (probabilistic or deterministic) is associated with a less-biased estimate in non-linear models. Health economists should consider the bias of probabilistic analysis and select the most suitable approach for their analyses.

Background Multiple sclerosis imposes a heavy burden on the person who suffers from it and on the relatives, due to the caregiving load involved. The objective was to analyse whether the inclusion of social costs in economic evaluations of multiple sclerosis-related interventions changed results and/or conclusions. Methods A systematic review was launched using Medline and the Cost-Effectiveness Analysis Registry of Tufts University (2000–2019). Included studies should: (1) be an original study published in a scientific journal, (2) be an economic evaluation of any multiple sclerosis-related intervention, (3) include productivity losses and/or informal care costs (social costs), (4) be written in English, (5) use quality-adjusted life years as outcome, and (6) separate the results according to the perspective applied. Results Twenty-nine articles were selected, resulting in 67 economic evaluation estimations. Social costs were included in 47% of the studies. Productivity losses were assessed in 90% of the estimations (the human capital approach was the most frequently used method), whereas informal care costs were included in nearly two-thirds of the estimations (applying the opportunity and the replacement-cost methods equally). The inclusion of social costs modified the figures for incremental costs in 15 estimations, leading to a change in the conclusions in 10 estimations, 6 of them changing from not recommended from the healthcare perspective to implemented from the societal perspective. The inclusion of social costs also altered the results from cost-effective to dominant in five additional estimations. Conclusions The inclusion of social costs affected the results/conclusions in multiple sclerosis-related interventions, helping to identify the most appropriate interventions for reducing its economic burden from a broader perspective.

Many countries use uniform cost-effectiveness criteria to determine whether to adopt a new medical technology for the entire population. This approach…

Involvement of patients and medical professionals in assessment of relative effectiveness (relative effectiveness assessment) contributes to an effici…