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

OBJECTIVES: To quantify health fluctuations, identify affected health-related quality of life (HRQoL) dimensions, and evaluate if fluctuations affect the HRQoL instruments recall period adherence in people living with dementia (PlwD). METHODS: Caregivers of PlwD completed a daily diary for 14 days, documenting if PlwD's health was better or worse than the day before and the affected HRQoL dimensions. Health fluctuation was categorized into low (0-4 fluctuations in 14 days), moderate (5-8) and high (9-14)...

OBJECTIVES: The primary focus of this research is the proposition of a methodological framework for the clinical application of the long coronavirus disease (COVID) Symptom and Severity Score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals suffering from long COVID. METHODS: A three-stage process was used to develop, validate, and establish scoring standards for the LC-SSS...

OBJECTIVES: This study aims to provide subjective well-being (SWB) population norms in Hungary and explore the contribution of explanatory factors of SWB inequality among the Hungarian adult general population. METHODS: The data originated from a large representative internet-based cross-sectional survey in Hungary, which was conducted in 2020. We applied validated multi-item instruments for measuring SWB, namely SWLS and WHO-5. Multiple linear regressions were employed to examine the relationship between demographic-socioeconomic-health status and both well-being instruments...

OBJECTIVES: The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) FL demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared to standard of care (SOC) to manage r/r FL patients who have had at least two prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective. METHODS: A three-state partitioned survival cost-effectiveness model was developed with a lifetime horizon...

OBJECTIVES: This study aims to systematically review evidence on the cost-effectiveness of chimeric antigen receptor (CAR)-T therapies for patients with cancer. METHODS: Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments and economic evaluations that compared costs and effects of CAR-T therapy in cancer patients were included. Two reviewers independently screened studies, extracted data, synthesized results, and critically appraised studies using the Philips checklist...

OBJECTIVES: Health economic (HE) models are often considered as "black boxes" because they are not publicly available and lack transparency, which prevents independent scrutiny of HE models. Additionally, validation efforts and validation status of HE models are not systematically reported. Methods to validate HE models in absence of their full underlying code are therefore urgently needed to improve health policy making.This study aimed to develop and test a generic dashboard to systematically explore the workings of HE models and validate their model parameters and outcomes...

OBJECTIVES: The Inflation Reduction Act (IRA), enacted in 2022, brings substantial reforms to the US healthcare system, particularly regarding Medicare. A key aspect includes the introduction of Medicare price negotiation. The objective of this commentary is to explore the implications of the IRA for US pharmaceutical companies, with a specific focus on the role of real-world evidence (RWE) in the context of Medicare reforms. METHODS: The commentary employs a qualitative analysis of the IRA's provisions related to healthcare and pharmaceutical regulation, focusing on how these reforms change the evidence requirements for pharmaceutical companies...

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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)...