Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research

OBJECTIVES: Faster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements (PBRSA), to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes. One important reason for risk-sharing is to resolve differences of opinion between innovators and payers about a technology's underlying value. To date, there has been no formal attempt to set out the circumstances in which risk-sharing can address these differences...

OBJECTIVE: Calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) are novel high-cost treatments for the prevention of migraine. This study presents data on utilisation, expenditure and treatment patterns with CGRP mAbs available under a managed access protocol in Ireland, to a cohort of treatment refractory patients (failed three or more previous treatments) with chronic migraine. METHODS: Data was extracted from the Primary Care Reimbursement Service (PCRS) High Tech claims database and special drug request online system, and analysed using Microsoft Excel and SAS...

OBJECTIVES: Health-related Quality of Life (HRQoL) instruments for cardiovascular diseases (CVD) have been commonly used to measure important patient-reported outcomes (PROs) in clinical trials and practices. This study aimed at systematically identifying and assessing the content validity of CVD-specific HRQoL instruments in clinical studies. METHODS: The research team searched CINAHL, Embase, and PubMed from inception to January 20, 2022. The research team included studies that reported the development and content validity for CVD-specific instruments...

OBJECTIVES: We aim to assess the psychometric performance and added value of nine existing bolt-ons (breathing problems, cognition, hearing, self-confidence, skin irritation, sleep, social relationships, tiredness and vision) for the EQ-5D-5L in a general population sample. METHODS: The EQ-5D-5L, nine bolt-ons, SF-6Dv1, PROMIS-29+2, PROMIS Global Health and Satisfaction With Life Scale were completed in an online cross-sectional survey among a general adult population sample in Hungary (n=1587)...

OBJECTIVE: To evaluate the cost-effectiveness of anti-vascular endothelial growth factor drugs (anti-VEGFs) compared with panretinal photocoagulation (PRP) for treating proliferative diabetic retinopathy (PDR) in the UK. METHODS: A discrete event simulation model was developed, informed by individual patient data meta-analysis. The model captures treatment effects on best corrected visual acuity in both eyes, and the occurrence of diabetic macular oedema (DMO) and vitreous haemorrhage...

OBJECTIVES: This commentary seeks to improve the design and analysis of trials undertaken to obtain approval of drugs for treatment of rare diseases. METHODS: Methodological analysis reveals that use of hypothesis testing in the FDA drug approval process is harmful. Conventional asymmetric error probabilities bias the approval process against approval of new drugs. Hypothesis testing is inattentive to the relative magnitudes of losses to patient welfare when Types I and II errors occur...

OBJECTIVE: Inclusion of relevant effectiveness and safety outcomes in economic evaluation of health technologies is required to aid efficient healthcare decisions. Our objective was to identify the key issues related to the inclusion of adverse events (AEs) in economic evaluation and explore perspectives for good practice recommendations to handle these issues. METHODS: We focused on the frequently encountered methodological issues related to the integration of AEs in economic evaluations of health technologies...

OBJECTIVES: Health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multi-criteria decision analysis (MCDA) approach. METHODS: We constructed an MCDA-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (n=23), we refined previously generated criteria and developed rank-based weights...

OBJECTIVES: This study aims to show the application of flexible statistical methods in real-world cost-effectiveness analyses applied in the cardiovascular field, focusing specifically on the use of PCSK9 inhibitors for hyperlipidaemia. METHODS: The proposed method allowed us to use an electronic health database to emulate a target trial for cost-effectiveness analysis using multi-state modelling and microsimulation. We formally established the study design and provided precise definitions of the causal measures of interest, while also outlining the assumptions necessary for accurately estimating these measures using the available data...

OBJECTIVES: Assessing the value of tumor-agnostic therapies (TAD) is challenging given the potential variability in treatment effects, trials with small sample sizes, different standards-of-care (SoC), and lack of comparative data from single-arm basket trials. Our study developed and applied novel methods to assess the value of pembrolizumab compared to SoC to inform coverage decisions. METHODS: We developed a partitioned survival model to evaluate the cost-utility of pembrolizumab for previously treated patients with 8 advanced or metastatic MSI-H/dMMR cancers from a U...

OBJECTIVES: Critics of quality-adjusted life years (QALYs) argue that it discriminates against older individuals. However, little empirical evidence has been produced to inform this debate. To compare published cost effectiveness analyses (CEAs) on patients aged ≥65 years and those aged <65 years. METHODS: We used the Tufts Cost-Effectiveness Analysis Registry to identify CEAs published in Medline between 1976 and 2021. Eligible CEAs were categorized according to age (≥65 years versus <65 years)...

OBJECTIVES: To conduct a review of existing methods used to incorporate life cycle drug pricing (LCDP) in cost-effectiveness analyses (CEAs), identify common methodological challenges, and suggest modeling approaches for prospectively implementing LCDP in CEA. METHODS: Two complementary searches were conducted in PubMed®, combined with hand searching and reference mining, to identify English language full-text articles that explored (1) how drug prices change over time and (2) methods used to apply dynamic pricing in cost-effectiveness models (CEMs)...

OBJECTIVES: To systematically review the psychometric properties of the Geriatric Oral Health Assessment Index (GOHAI) across age groups using the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) methodology. METHODS: Data: English peer-reviewed articles reporting studies of the development, translation, or validation of GOHAI. SOURCES: PubMed, Web of Science and EMBASE from Jan 1990 until 31. December 2023...

OBJECTIVE: Several measures have been used or developed to capture the health and well-being of caregivers, including the EQ Health and Well-being (EQ-HWB) and its short form, EQ-HWB-S. This study aimed to evaluate the psychometric properties and construct validity of the EQ-HWB/EQ-HWB-S in a US caregiver population. METHODS: A cross-sectional survey was conducted involving 504 caregivers. Eligible participants were 18+ years old, provided unpaid care to a relative/friend aged 18+ in the past six months, and spent on average of at least one hour per week caregiving...

OBJECTIVES: Pelvic organ prolapse is the descent of one or more reproductive organs from their normal position, causing associated negative symptoms. One conservative treatment option is pessary management. The aim of this study was to investigate the cost-effectiveness of pessary self-management (SM) when compared to clinic-based care (CBC). A decision-analytic model was developed to extend the economic evaluation. METHODS: A randomised controlled trial with health economic evaluation...

OBJECTIVE: In 2018, Rwanda launched a national program to eliminate the hepatitis C virus (HCV). We aim to assess the impact of the program to date and identify strategies to achieve the World Health Organization's HCV elimination goals by 2030. METHODS: We developed a microsimulation model to simulate Rwanda's HCV epidemic from 2015 through 2050 and evaluated temporal trends in HCV infection, prevalence, mortality, and the total cost of care for scenarios that could achieve HCV elimination by 2030...

OBJECTIVES: The EQ Health and Wellbeing, EQ-HWB (25-item) and the EQ-HWB-S (9-item), are new generic measures of health and wellbeing. The purpose of this study was to examine the measurement properties of the EQ-HWB and EQ-HWB-S measures in relation to the EQ-5D-5L among the Italian general population. METHODS: A cross-sectional survey was conducted from October 2020 to February 2021, followed by secondary analysis of collected data from Italian adults. This analysis included response pattern distributions, correlation strength, and known group comparison (KGC)...

No abstract text is available yet for this article.

OBJECTIVES: Time trade-off (TTO) and discrete choice experiment (DCE) preference-elicitation techniques can be administered using face-to-face interviews (F2F), unassisted online (UO) surveys, or remote-assisted (RA) interviews. The objective of this study was to explore how the mode of administration affects the quality and reliability of preference-elicitation data. METHODS: EQ-5D-5L health states were valued using composite TTO (cTTO) and DCE approaches by the UK general population...

OBJECTIVES: The EQ-5D-Y-5L is a generic preference-based measure of health-related quality of life (HRQoL) for children. This study aimed to describe the distributional properties, test-retest reliability, and convergent validity of the EQ-5D-Y-5L in children with intellectual disability (ID). METHODS: Caregivers of children with ID (aged 4 to 18 years) completed an online survey including a proxy-report EQ-5D-Y-5L, the Quality of life Inventory-Disability (QI-Disability), and disability-appropriate measures corresponding to the EQ-5D dimensions: mobility (MO), self-care (SC), usual activities (UA), pain/discomfort (PD), and worry/sadness/unhappiness (WSU)...