Expert Review of Pharmacoeconomics & Outcomes Research

INTRODUCTION: Suboptimal medication adherence is common among patients with cardiovascular diseases. We sought evidence on non-pharmacological interventions used to support adherence for patients with hypertension and/or dyslipidemia. METHODS: We searched MEDLINE, EMBASE, MEDLINE In-Process, ClinicalTrials.gov, EUCTR, and conference proceedings from July 2011-July 2021 to identify trials evaluating effects of health education, phone reminders, or digital interventions on medication adherence or persistence of adult patients with hypertension and/or dyslipidemia...

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OBJECTIVE: To explore the effect of traditional Chinese medicine (TCM) on the treatment of chronic kidney disease (CKD). METHODS: Databases were used for literature research until 16 December 2022, including PubMed, Cochrane Library, China National Knowledge Infrastructure (CNKI), and Embase. After full-text screening, data were extracted by two researchers independently. The Cochrane ROB tool was applied for quality assessment. The heterogeneity was tested using the Chi-squared-based Q statistic test and the I2 statistic...

INTRODUCTION: The aim of this study is to update a systematic review of instruments for evaluating the executive functions (EFs) in a pediatric population to assess their measurement properties. AREA COVERED: Studies describing evaluation tools of EFs were systematically searched on four electronic database: PubMed, EBSCO, Scopus and Web of Science. To be included studies had to be on a population aged 0 to 18 were included. The individuals were either healthy or presented a neurodevelopment disorder...

OBJECTIVE: To describe the impact of acute myeloid leukemia (AML) diagnosis on workplace absenteeism and disability days among patients and their caregivers. METHODS: This retrospective study included adults with newly-diagnosed AML (2009-2019) and adult caregivers of patients with newly-diagnosed AML, identified from the US Merative™ MarketScan® Commercial Database. The Merative MarketScan Health and Productivity Management Database provided linked patient-level records of workplace absence and short-term (STD) and long-term disability (LTD) data...

OBJECTIVES: The Asian PEONY trial showed that add-on pertuzumab to trastuzumab and chemotherapy significantly improved pathological complete response in the neoadjuvant treatment of patients with human epidermal growth factor receptor 2-positive (HER2+) early breast cancer (EBC). This study evaluated the cost-effectiveness of pertuzumab as an add-on therapy to trastuzumab and chemotherapy for neoadjuvant treatment of patients with HER2+ EBC in Singapore. METHODS: A six-state Markov model was developed from the Singapore healthcare system perspective, with a lifetime time horizon...

INTRODUCTION: Antibody-mediated rejection (ABMR) is a major cause of late kidney allograft failure, but its economic and humanistic impacts have not been well-characterized in the literature. AREAS COVERED: We reviewed available literature on economic burden (costs and healthcare resource use) and humanistic burden (health-related quality of life impacts [HRQOL] and utility estimates) in patients diagnosed with kidney transplant rejection; ABMR-specific studies were of particular interest...

INTRODUCTION: Biosimilars have improved access to biologic medicines; however, historical thinking may jeopardize the viability of future markets. AREAS COVERED: An expert panel of eight diverse European stakeholders provided insights about rethinking biosimilars and cost-savings, reducing patient access inequalities, increasing inter-market equity, and improving education. The insights reported here (Part 2) follow a study that provides perspectives on leveraging the holistic benefits of biosimilars for market sustainability based on independent survey results and telephone interviews of stakeholders from diverse biosimilar markets (Part 1)...

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INTRODUCTION: As drug prices are viewed to be opaque, there have been increasing societal demands on policy and decision makers to implement initiatives that promote drug price transparency. AREAS COVERED: This Perspective discusses what drug price transparency is and how it works in theory and in practice. EXPERT OPINION: Transparency on drug prices may target payers, patients and health care professionals; and may relate to prices at each stage in a drug's distribution system...

OBJECTIVES: Social care in the United Kingdom (UK) refers to care provided due to age, illness, disability, or other circumstances. Social care provision offers an intermediary step between hospital discharge and sufficient health for independent living, which subsequently helps with National Health Service (NHS) bed capacity issues. UK Health Technology Assessments (HTAs) do not typically include social care data, possibly due to a lack of high-quality, accessible social care data to generate evidence suitable for submissions...

OBJECTIVES: This study assessed the feasibility of using the Milano-Torino staging (MiToS)system for conducting economic evaluation to measurehealth outcomes in amyotrophic lateral sclerosis (ALS). METHODS: AMarkov model was developed using the MiToS system and evaluated with ahypothetical treatment versus standard of care. Health utilities and transitionprobabilities were derived from the literature. Four-time horizons (1, 5, 10,and 20 years) were examined...

OBJECTIVES: A public economic framework was used to explore lifetime government costs and benefits in relation to the Pediatric Immunization Program (PIP) in Belgium based on cases and deaths averted. METHODS: To estimate changes in net government revenue, we developed a decision-analytic model that quantifies lifetime tax revenues and transfers based on changes in morbidity and mortality arising from Belgium's Pediatric Immunization Program (PIP). The model considered differences in incidence rates with vaccines included in Belgium's PIP: compared with pre-vaccine era...

OBJECTIVE: The burden of diabetes on individuals, healthcare systems, and society must be explored to improve and sustain diabetes care. With this aim, we estimated both past and future diabetes-related direct health expenditures in Slovenia. METHODS: Analysis of expenditures from the healthcare payer perspective during the 2019-2022 period was based on individual patient data on expenditures for seven groups of diabetes-related medical conditions from the population-level database of the Health Insurance Institute of Slovenia...

INTRODUCTION: An avalanche of early stage cancer clinical trials is coming. The majority of these solely use surrogate outcomes that have not been validated against a target outcome of interest (e.g. overall survival). Current HTA guidance on surrogate outcome validation are not methodologically or practically conducive to this scenario. AREAS COVERED: We provide a high-level overview of methods, approaches, and conceptual thinking for making better use of limited evidence within early stage cancer HTA submissions...

INTRODUCTION: Approved biosimilars exhibit comparable efficacy, safety, and immunogenicity to reference products. This report provides perspectives on the societal value of biosimilars within Europe and potential factors that have influenced market dynamics. METHODS: An independent, self-administered survey or one-on-one in-depth interview was used to collect viewpoints about the impact of biosimilar medicines within European markets. Key insights were also sought from an expert panel of European stakeholders...

INTRODUCTION: The increasing availability of data and computing power has made machine learning (ML) a viable approach to faster, more efficient healthcare delivery. METHODS: A systematic literature review (SLR) of published SLRs evaluating ML applications in healthcare settings published between1 January 2010 and 27 March 2023 was conducted. RESULTS: In total 220 SLRs covering 10,462 ML algorithms were reviewed. The main application of AI in medicine related to the clinical prediction and disease prognosis in oncology and neurology with the use of imaging data...

OBJECTIVE: To assess the cost-effectiveness of sacituzumab govitecan for treating relapsed or refractory metastatic triple-negative breast cancer (TNBC) in Singapore. METHODS: A three-state partitioned survival model was developed to evaluate the cost-effectiveness of sacituzumab govitecan from a healthcare system perspective over 5 years. Clinical inputs were obtained from the ASCENT trial. Health state utilities were retrieved from the literature and direct costs were sourced from public healthcare institutions in Singapore...

BACKGROUND: Antineutrophil cytoplasmic antibody-associated vasculitis (AAV) are rare autoimmune diseases characterized by inflammation of blood vessels. This study aimed to assess the cost-utility of avacopan in combination with rituximab (RTX) or cyclophosphamide (CYC) compared with glucocorticoids (GC) for the treatment of severe, active AAV in Spain. METHODS: A 9-state Markov model was designed to reflect the induction of remission and sustained remission of AAV over a lifetime horizon...

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