Design and analysis of health state valuation data for model-based economic evaluations and for economic evaluations alongside clinical trials

2016 ◽  
Author(s):  
John Brazier ◽  
Julie Ratcliffe ◽  
Joshua A. Salomon ◽  
Aki Tsuchiya
Keyword(s):  
Clinical Trials ◽  
General Population ◽  
Sample Size ◽  
Final Section ◽  
Health State Valuation ◽  
Economic Evaluations ◽  
Health State ◽  
Mode Of Administration ◽  
Administration Timing ◽  
Analytic Models

This chapter focuses upon the needs of two approaches, economic evaluations based on decision analytic models, and those alongside clinical trials in terms of the collection and analysis of health state values. The first section of the chapter presents requirements that are likely to be common to any study in which health state values are collected from patients and/or members of the general population, including: who to ask, mode of administration, timing of assessments, sample size, and handling uncertainty. The second section of the chapter considers issues specific to trial-based economic evaluations, and the final section considers issues specific to the design and analysis of health state valuation data for economic models.

scholarly journals Binary Choice Health State Valuation and Mode of Administration: Head-to-Head Comparison of Online and CAPI

2013 ◽  
Vol 16 (1) ◽  
pp. 104-113 ◽  
Author(s):  
Brendan Mulhern ◽  
Louise Longworth ◽  
John Brazier ◽  
Donna Rowen ◽  
Nick Bansback ◽  
...  
Keyword(s):  
Health State Valuation ◽  
Binary Choice ◽  
Health State ◽  
Mode Of Administration

scholarly journals Implausible States: Prevalence of EQ-5D-5L States in the General Population and Its Effect on Health State Valuation

2020 ◽  
Vol 40 (6) ◽  
pp. 735-745 ◽  
Author(s):  
Ole Marten ◽  
Brendan Mulhern ◽  
Nick Bansback ◽  
Aki Tsuchiya
Keyword(s):  
General Population ◽  
Secondary Data ◽  
Patient Survey ◽  
Health State Valuation ◽  
Health State ◽  
Health States ◽  
Data Set ◽  
Value Sets ◽  
Routine Removal ◽  
The Impact

The EQ-5D is made up of health state dimensions and levels, in which some combinations seem less “plausible” than others. If “implausible” states are used in health state valuation exercises, then respondents may have difficulty imagining them, causing measurement error. There is currently no standard solution: some valuation studies exclude such states, whereas others leave them in. This study aims to address 2 gaps in the literature: 1) to propose an evidence-based set of the least prevalent two-way combinations of EQ-5D-5L dimension levels and 2) to quantify the impact of removing perceived implausible states from valuation designs. For the first aim, we use data from 2 waves of the English General Practitioner Patient Survey ( n = 1,639,453). For the second aim, we remodel a secondary data set of a Discrete Choice Experiment (DCE) with duration that valued EQ-5D-5L and compare across models that drop observations involving different health states: 1) implausible states as defined in the literature, 2) the least prevalent states identified in stage 1, and 3) randomly select states, alongside 4) a model that does not drop any observations. The results indicate that two-way combinations previously thought to be implausible actually exist among the general population; there are other combinations that are rarer, and removing implausible states from an experimental design of a DCE with duration leads to value sets with potentially different characteristics depending on the criterion of implausible states. We advise against the routine removal of implausible states from health state valuation studies.

Planning the Size of Clinical Trials with Allowance for Patient Noncompliance

1990 ◽  
Vol 29 (03) ◽  
pp. 243-246 ◽  
Author(s):  
M. A. A. Moussa
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sample Size ◽  
Event Rate ◽  
Survival Functions ◽  
Time Intervals ◽  
Patient Noncompliance ◽  
Event Rates ◽  
Larger Sample

AbstractVarious approaches are considered for adjustment of clinical trial size for patient noncompliance. Such approaches either model the effect of noncompliance through comparison of two survival distributions or two simple proportions. Models that allow for variation of noncompliance and event rates between time intervals are also considered. The approach that models the noncompliance adjustment on the basis of survival functions is conservative and hence requires larger sample size. The model to be selected for noncompliance adjustment depends upon available estimates of noncompliance and event rate patterns.

scholarly journals The measurement performance of the EQ-5D-5L versus EQ-5D-3L in patients with hidradenitis suppurativa

2021 ◽  
Author(s):  
Alex Bató ◽  
Valentin Brodszky ◽  
L. Hunor Gergely ◽  
Krisztián Gáspár ◽  
Norbert Wikonkál ◽  
...  
Keyword(s):  
Convergent Validity ◽  
Hidradenitis Suppurativa ◽  
Clinical Care ◽  
Intraclass Correlation ◽  
Life Quality ◽  
Measurement Properties ◽  
Economic Evaluations ◽  
Health State ◽  
Cross Sectional Survey ◽  
Cross Sectional

Abstract Purpose Hidradenitis suppurativa (HS) is a chronic inflammatory skin disease that affects up to 1% of the population in Europe. The EQ-5D is the most commonly used generic instrument for measuring health-related quality of life among HS patients. This study aims to compare the measurement properties of the two adult versions of EQ-5D (EQ-5D-3L and EQ-5D-5L) in patients with HS. Methods We recruited 200 consecutive patients with HS (mean age 37 years, 38% severe or very severe HS) to participate in a multicentre cross-sectional survey. Patients completed the EQ-5D-3L, EQ-5D-5L, Dermatology Life Quality Index (DLQI) and Skindex-16 questionnaires. Results More than twice as many different health state profiles occurred in the EQ-5D-5L compared to the EQ-5D-3L (101 vs. 43). A significant reduction in ceiling effect was found for the mobility, self-care and usual activities dimensions. A good agreement was established between the EQ-5D-3L and EQ-5D-5L with an intraclass correlation coefficient of 0.872 (95% CI 0.830–0.903; p < 0.001) that was confirmed by a Bland-Altman plot. EQ-5D-5L improved both the absolute and relative informativity in all dimensions except for anxiety/depression. EQ-5D-3L and EQ-5D-5L demonstrated similar convergent validity with DLQI and Skindex-16. EQ-5D-5L was able to better discriminate between known groups of patients based on the number of comorbidities and disease severity (HS-Physician's Global Assessment). Conclusion In patients with HS, the EQ-5D-5L outperformed the EQ-5D-3L in feasibility, ceiling effects, informativity and known-groups validity for many important clinical characteristics. We recommend using the EQ-5D-5L in HS patients across various settings, including clinical care, research and economic evaluations.

scholarly journals Do patient characteristics matter when calculating sample size for eczema clinical trials?

2021 ◽  
Author(s):  
L. Howells ◽  
S. Gran ◽  
J. R. Chalmers ◽  
B. Stuart ◽  
M. Santer ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Sample Size ◽  
Patient Characteristics

scholarly journals NCOG-36. FIRST HEALTH UTILITIES OF GLIOBLASTOMA PATIENTS USING TTFIELDS TREATMENT

2020 ◽  
Vol 22 (Supplement_2) ◽  
pp. ii137-ii137
Author(s):  
Gordon Chavez ◽  
Christina Proescholdt
Keyword(s):  
Population Sample ◽  
National Health System ◽  
Medical Decision ◽  
Economic Evaluations ◽  
Health State ◽  
High Quality ◽  
Health States ◽  
Tumor Treating Fields ◽  
Health State Utilities ◽  
The Uk

Abstract BACKGROUND Despite the importance of Health State Utilities for health policy and medical decision making, there are no publications that provide high quality utility values estimated from glioblastoma multiforme (GBM) patients. Published health economic evaluations for GBM treatments rely on utilities determined by Garside et al. (2007), which used the standard gamble method in healthy panel members of the UK National Health System. There are no published utilities for GBM estimated from a general population sample, and there are no utility estimates whatsoever for Tumor Treating Fields (TTFields) users. METHODS We designed a study to remedy this major deficit by eliciting utilities directly from GBM patients using the EuroQol 5-Dimension (EQ-5D) survey. The EQ-5D is a widely used and NICE-recommended tool for the estimation of health state utilities. The survey is composed of a questionnaire that asks patients to specify their health state along 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Statistical models provided by EuroQol’s network of researchers convert this data into health state utility estimates. RESULTS The EQ-5D questionnaire is administered to active patients using TTFields treatment during the study duration, allowing the elicitation of health preference measures for different glioblastoma health states based on: progression status (progressed vs. non-progressed), current treatments (TTFields only vs. TTFields + others) and time-from-diagnosis (0-12 months vs. &gt; 12 months) CONCLUSION These results are important for understanding the patient preferences using TTFields treatment and communicating these preferences to decision makers. This study is the first to provide direct, high quality utility measures in glioblastoma patients using TTFields treatment.

scholarly journals Valuation of the EQ-5D-3L in Russia

2021 ◽  
Author(s):  
Vitaly Omelyanovskiy ◽  
Nuriya Musina ◽  
Svetlana Ratushnyak ◽  
Tatiana Bezdenezhnykh ◽  
Vlada Fediaeva ◽  
...  
Keyword(s):  
Health Problems ◽  
Cost Utility ◽  
Economic Evaluations ◽  
Computer Assisted ◽  
Health State ◽  
Primary Analysis ◽  
Health States ◽  
Value Set ◽  
Model Combining ◽  
Life Years

Abstract Purpose The most widely used generic questionnaire to estimate the quality of life for yielding quality-adjusted life years in economic evaluations is EQ-5D. Country-specific population value sets are required to use EQ-5D in economic evaluations. The aim of this study was to establish an EQ-5D-3L value set for Russia. Methods A representative sample aged 18+ years was recruited from the Russia`s general population. Computer-assisted face–to–face interviews were conducted based on the standardized valuation protocol using EQ-Portable Valuation Technology. Population preferences were elicited utilizing both composite time trade-off (cTTO) and discrete choice experiment (DCE) techniques. To estimate the value set, a hybrid regression model combining cTTO and DCE data was used. Results A total of 300 respondents who successfully completed the interview were included in the primary analysis. 120 (40.0%) respondents reported no health problems of any dimension, and 56 (18.7%) reported moderate health problems in one dimension of the EQ‐5D‐3L. Median self-rated health using EQ‐VAS was 80 with IQR 70–90. Comparing cTTO and DCE-predicted values for 243 health states resulted in a similar pattern. This supports the use of hybrid models. The predicted value based on the preferred model for the worst health state “33333” was −0.503. Mobility dimension had the most significant impact on the utility decrement, and anxiety/depression had the lowest decrement. Conclusion Determining a Russian national value set may be considered the first step towards promoting cost-utility analysis use to increase comparability among studies and improve the transferability of healthcare decision-making in Russia.

scholarly journals Economic evaluations in paediatric dentistry clinical trials

2020 ◽  
Vol 31 (S1) ◽  
pp. 56-65
Author(s):  
Helen Jessica Rogers ◽  
Raiza Dias Freitas ◽  
Morgan James Beeson ◽  
Christopher Robert Vernazza
Keyword(s):  
Clinical Trials ◽  
Paediatric Dentistry ◽  
Economic Evaluations
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