PharmacoEconomics最新文献

Objective: The aim of this study was to compare the psychometric performance of common generic paediatric health-related quality-of-life instrument descriptive systems (PedsQL generic core 4.0, EQ-5D-Y-3L, EQ-5D-Y-5L, Child Health Utility 9D [CHU9D], Assessment of Quality of Life 6D [AQoL-6D], and Health Utilities Index Mark 3 [HUI3]) by child age, report type, and health status.

Methods: Data for children aged 5-18 years were from the Australian Paediatric Multi-Instrument Comparison study. Ceiling effects, test-retest reliability, known-group validity, convergent and divergent validity, and responsiveness were assessed in the total sample and by child age (5-12 years vs 13-18 years), report type (self- vs proxy report), and health status. Instruments were scored using an exploratory level sum score (LSS) approach.

Results: Survey data were available for 5945 children, with follow-up data available for 2346 children. The EQ-5D-Y-3L demonstrated ceiling effects. The PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D demonstrated acceptable test-retest reliability. All instruments demonstrated known-group, convergent, and divergent validity. The EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D demonstrated responsiveness to improvements in health and the PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D to worsening health. The AQoL-6D and HUI3 had inconclusive test-retest reliability and responsiveness evidence due to small sample size. Importantly, ceiling effects, test-retest reliability and responsiveness varied by subgroup.

Conclusion: Results reflect instrument performance using LSSs, which may differ to utility scores. In the total sample, the EQ-5D-Y-5L and CHU9D descriptive systems demonstrated evidence of good performance (i.e., meeting prespecified criteria) across all psychometric attributes tested. Performance varied by child age and report type, indicating room for considerations by population and study.

Background: Widely used generic instruments to measure paediatric health-related quality of life (HRQoL) include the EQ-5D-Y-5L, Child Health Utility 9 Dimension (CHU-9D), Paediatric Quality of Life Inventory (PedsQL) and Health Utilities Index (HUI). There are similarities and differences in the content of these instruments, but there is little empirical evidence on how the items they contain relate to each other, and to an overarching model of HRQoL derived from their content.

Objective: This study aimed to explore the dimensionality of the instruments using exploratory factor analysis (EFA).

Methods: Data from the Australian Paediatric Multi-Instrument Comparison (P-MIC) Study were used. EQ-5D-Y-5L, CHU-9D, PedsQL and HUI data were collected via proxy or child self-report data. EFA was used to investigate the underlying domain structure and measurement relationship. Items from the four instruments were pooled and domain models were identified for self- and proxy-reported data. The number of factors was determined based on eigenvalues greater than 1. A correlation cut-off of 0.32 was used to determine item loading on a given factor, with cross-loading also considered. Oblique rotation was used.

Results: Results suggest a six-factor structure for the proxy-reported data, including emotional functioning, pain, daily activities, physical functioning, school functioning, and senses, while the self-report data revealed a similar seven-factor structure, with social functioning emerging as an additional factor.

Conclusion: We provide evidence of differences and similarities between paediatric HRQoL instruments and the aspects of health being measured by these instruments. The results identified slight differences between self- and proxy-reported data in the relationships among items within the resulting domains.

Introduction: Few preference-weighted instruments are available to measure health-related quality of life in young children (2-4 years of age). The EQ-5D-Y-3L and EQ-5D-Y-5L were recently modified for this purpose.

Objective: The aim of this study was to test the psychometric properties of these adapted versions for use with parent proxies of children aged 2-4 years and to compare their performance with the original versions. It was hypothesised that the adapted instrument wording would result in improved psychometric performance.

Methods: Survey data of children aged 2-4 years were obtained from the Australian Paediatric Multi-Instrument Comparison study. Distributional and psychometric properties tested included feasibility, convergence, distribution of level scores, ceiling effects, known-group validity (Cohen's D effect sizes for prespecified groups defined by the presence/absence of special healthcare needs [SHCNs]), test-retest reliability (intraclass correlation coefficients [ICCs]), and responsiveness (standardised response mean [SRM] effect sizes for changes in health). Level sum scores were used to provide summary outcomes. Supplementary analysis using utility scores (from the Swedish EQ-5D-Y-3L value set) were conducted for the adapted and original EQ-5D-Y-3L, and no value sets were available for the EQ-5D-Y-5L.

Results: A total of 842 parents of children aged 2-4 years completed the survey. All instruments were easy to complete. There was strong convergence between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed more responses in the severe levels of the five EQ-5D-Y dimensions, particularly in the usual activity and mobility dimensions (EQ-5D-Y-5L: mobility level 1: adapted n = 478 [83%], original n = 253 [94%]; mobility level 4/5: adapted n = 17 [2.9%], original n = 4 [1.5%)]). The difference in the distribution of responses was more evident in children with SHCNs. Assessment of known-group validity showed a greater effect size for the adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L compared with the original instruments (EQ-5D-Y-5L: adapted Cohen's D = 1.01, original Cohen's D = 0.83) between children with and without SHCNs. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed improved reliability at 4-week follow-up, with improved ICCs (EQ-5D-Y-5L: adapted ICC = 0.83, original ICC = 0.44). The responsiveness of all instruments moved in the hypothesised direction for better or worse health at follow-up. Probability of superiority analysis showed little/no differences between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. Supplementary psychometric analysis of the adapted and original EQ-5D-Y-3L using utilities showed comparable findings with analyses using level sum scores.

Conclusions: The findings suggest improved psychometric performance of the adapted version of

Background

The availability of increasingly advanced and expensive new health technologies puts considerable pressure on publicly financed healthcare systems. Decisions to not—or no longer—reimburse a health technology from public funding may become inevitable. Nonetheless, policymakers are often pressured to amend or revoke negative reimbursement decisions due to the public disagreement that typically follows such decisions. Public disagreement may be reinforced by the publication of pictures of individual patients in the media. Our aim was to assess the effect of depicting a patient affected by a negative reimbursement decision on public disagreement with the decision.

Methods

We conducted a discrete choice experiment in a representative sample of the public (n = 1008) in the Netherlands and assessed the likelihood of respondents’ disagreement with policymakers’ decision to not reimburse a new pharmaceutical for one of two patient groups. We presented a picture of one of the patients affected by the decision for one patient group and “no picture available” for the other group. The groups were described on the basis of patients’ age, health-related quality of life (HRQOL) and life expectancy (LE) before treatment, and HRQOL and LE gains from treatment. We applied random-intercept logit regression models to analyze the data.

Results

Our results indicate that respondents were more likely to disagree with the negative reimbursement decision when a picture of an affected patient was presented. Consistent with findings from other empirical studies, respondents were also more likely to disagree with the decision when patients were relatively young, had high levels of HRQOL and LE before treatment, and large LE gains from treatment.

Conclusions

This study provides evidence for the effect of depicting individual, affected patients on public disagreement with negative reimbursement decisions in healthcare. Policymakers would do well to be aware of this effect so that they can anticipate it and implement policies to mitigate associated risks.

Background: Assessment of quality of life (QoL) in people living with sleep disorders using questionnaires is necessary to compare intervention benefits. Knowledge of the content and concepts covered by specific QoL instruments is essential to determine which instruments are best suited for conducting economic evaluations of sleep-related interventions.

Objectives: This review aims to identify the QoL instruments that have been applied in economic evaluations of sleep disorder interventions and compare their conceptual overlap and content coverage using the framework of the International Classification of Functioning, Disability and Health (ICF).

Methods: A systematic review of full economic evaluations in sleep published in peer-reviewed journals from conception to 30 May, 2023 was conducted. MEDLINE, PsychInfo, ProQuest, Cochrane, Scopus, CINAHL, Web of Science and Emcare were searched for eligible studies. Studies incorporating either generic or sleep-specific QoL instruments as the primary or secondary measures of effectiveness within a full economic evaluation were included. Quality appraisal against the JBI Critical Appraisal Checklist for Economic Evaluations and EURONHEED checklists and mapping of QoL items to ICF categories were performed by two reviewers, with a third helping settle any potential differences.

Results: Sixteen instruments were identified as having been used in sleep health economic evaluations. The EQ-5D-3L, Epworth Sleepiness Scale, and Insomnia Severity Index were the most widely used, but the latter two are predominantly diagnostic tools and not specifically designed to guide economic evaluations. Other instruments with broader ICF content coverage have been least used, and these include the Sleep Apnea Quality of Life Index, Functional Outcomes of Sleep Questionnaire, 15 Dimensions, Short-Form 6 Dimensions, 12-item Short Form Survey, 36-item Short Form Survey and the GRID Hamilton Rating Scale for Depression.

Conclusions: This study provides an overview of current QoL instruments used in economic evaluations of sleep with respect to their content coverage. A combination of generic and sleep-specific instruments with broader ICF content coverage is recommended for such evaluations.

Background: Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, with up to 32% of patients with NSCLC harboring an epidermal growth factor receptor (EGFR) mutation. NSCLC harboring an EGFR mutation has a dedicated treatment pathway, with EGFR tyrosine kinase inhibitors and platinum-based chemotherapy often being the therapy of choice.

Objective: The aim of this study was to systemically review and summarize economic models of first-line treatments used for locally advanced or metastatic NSCLC harboring EGFR mutations, as well as to identify areas for improvement for future models.

Methods: Literature searches were conducted via Ovid in PubMed, MEDLINE, MEDLINE In-Process, Embase, Evidence-Based Medicine Reviews: Health Technology Assessment, Evidence-Based Medicine Reviews: National Health Service Economic Evaluation Database, and EconLit. An initial search was conducted on 19 December 2022 and updated on 11 April 2023. Studies were selected according to predefined criteria using the Population, Intervention, Comparator, Outcome and Study design (PICOS) framework.

Results: Sixty-seven articles were included in the review, representing 59 unique studies. The majority of included models were cost-utility analyses (n = 52), with the remaining studies being cost-effectiveness analyses (n = 4) and a cost-minimization analysis (n = 1). Two studies incorporated both a cost-utility and cost-minimization analysis. Although the model structure across studies was consistently reported, justification for this choice was often lacking.

Conclusions: Although the reporting of economic models in NSCLC harboring EGFR mutations is generally good, many of these studies lacked sufficient reporting of justification for structural choices, performing extensive sensitivity analyses and validation in economic evaluations. In resolving such gaps, the validity of future models can be increased to guide healthcare decision making in rare indications.