PharmacoEconomics Open

OBJECTIVES: To develop a value set reflecting the United States (US) general population's preferences for health states described by the Functional Assessment of Cancer Therapy (FACT) eight-dimensions preference-based multi-attribute utility instrument (FACT-8D), derived from the FACT-General cancer-specific health-related quality-of-life (HRQL) questionnaire. METHODS: A US online panel was quota-sampled to achieve a general population sample representative by sex, age (≥ 18 years), race and ethnicity...

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BACKGROUND: Gastric cancer patients with peritoneal carcinomatosis (PC) have a poor prognosis, with a median overall survival of 10 months when treated with systemic chemotherapy only. Cohort studies showed that cytoreductive surgery with hyperthermic intraperitoneal chemotherapy (CRS/HIPEC) might improve the prognosis for gastric cancer patients with limited PC. Besides generating trial data on clinical effectiveness, it is crucial to timely collect information on economic aspects to guide the reimbursement decision-making process...

BACKGROUND: The aim of this study was to pool multiple data sets to build a patient-centric, data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) to estimate disease trajectory across patient lifetime under current standard of care in future economic evaluations. The study was conducted as part of Project HERCULES, a multi-stakeholder collaboration to develop tools to support health technology assessments of new treatments for DMD. METHODS: Health states were informed by a review of NHMs for DMD and input from clinicians, patients and caregivers, and defined using common outcomes in clinical trials and real-world practice...

AIM: This study assessed the cost-effectiveness of neoadjuvant pembrolizumab plus chemotherapy followed by adjuvant pembrolizumab versus neoadjuvant chemotherapy plus placebo followed by adjuvant placebo for patients with high-risk, early-stage, triple-negative breast cancer (TNBC) from a Swiss third-party payer perspective over a lifetime horizon (51 years). MATERIALS AND METHODS: A transition model with four health states (event-free, locoregional recurrence, distant metastasis, and death) was developed to assess the cost-effectiveness of pembrolizumab plus chemotherapy versus chemotherapy alone for the treatment of high-risk, early-stage TNBC...

BACKGROUND: Dementia affects about 55 million people worldwide. Demographic change and shifting lifestyles challenge the organization of dementia care. A discrete choice experiment (DCE) was conducted to elicit preferences for living arrangements in dementia in urban and rural regions of Germany. METHODS: Preliminary work included review of previous literature and focus groups. The DCE consists of seven attributes (group size, staff qualifications, organization of care, activities offered, support of religious practice, access to garden, consideration of food preferences) with three levels each...

BACKGROUND: The aim of this study was to assess health care resource utilization (HRU) and costs associated with delayed pulmonary arterial hypertension (PAH) diagnosis in the United States. METHODS: Eligible adults with newly diagnosed PAH from Optum's de-identified Clinformatics® Data Mart Database (2016-2021) were assigned to mutually exclusive cohorts based on time between first PAH-related symptom and first PAH diagnosis (i.e., ≤12 months' delay, >12 to ≤24 months' delay, >24 months' delay)...

BACKGROUND: Alzheimer's disease (AD) has been associated with great healthcare and non-healthcare resource consumption. The aim of this study was to estimate the burden of AD in Spain according to disease severity from a societal perspective. METHODS: A self-administered questionnaire was designed by the researchers and completed by the informal caregivers of patients with AD, reporting data on themselves as caregivers and on the AD patients for whom they care. The patients' sociodemographic and clinical data, their healthcare and non-healthcare resource consumption in the previous 12 months, and the impact of the disease on labor productivity were compiled...

BACKGROUND: We conducted a study to investigate the role of health insurance plans on biosimilar adoption among commercially insured patients in the USA. Flexible and rigid health plans may exhibit differing biosimilar coverage due to variations in cost considerations, formulary design, and provider networks. OBJECTIVE: To identify the characteristics of switchers and biosimilar initiators for six biologic-biosimilar pairs. METHODS: Using claims data from 2015 to 2019, we implement sequential regression models to assess the role of health plans on biosimilars adoption...

INTRODUCTION: Immunocompromised (IC) adults are at increased risk of developing herpes zoster (HZ) and HZ-related complications due to therapy or underlying disease. This study evaluated the cost effectiveness of recombinant zoster vaccine (RZV) versus no vaccine for the prevention of HZ in hematopoietic stem cell transplant (HSCT) recipients and other IC adults aged ≥ 18 years in the United States (US). METHODS: A static Markov model simulated cohorts of IC individuals using a 1-year cycle length and 30-year time horizon to estimate the cost effectiveness of RZV...

BACKGROUND/AIMS: The use and costs of health care rise substantially in the months prior to death, and although the use of palliative care services may be expected to lead to less costly care, the evidence is mixed. We analysed the costs of care over the last year of life and the extent to which these are associated with the use and duration of specialist palliative care (SPC) for decedents who died from cancer or another life-limiting illness. METHODS: The decedents were participants in a cohort study of older residents of the state of New South Wales, Australia...

BACKGROUND: Cetuximab and panitumumab, two anti-EGFR therapies, are widely used for third-line therapy of metastatic colorectal cancer (mCRC) with wild-type KRAS, but there remains uncertainty around their cost effectiveness. The objective of this analysis was to conduct a real-world cost-effectiveness analysis of the policy change introducing KRAS testing and third-line anti-EGFR therapy mCRC in British Columbia (BC), Canada. METHODS: We conducted secondary analysis of administrative data for a cohort of mCRC patients treated in BC in 2006-2015...

OBJECTIVE: We aimed to investigate whether individuals' trade-offs between vaccine effectiveness and vaccine safety vary if they are asked to consider the perspective of a policymaker making decisions for others compared with the decisions they would make for themselves. METHOD: A web-enabled discrete choice experiment survey was administered between 1 April and 1 May 2022 to participants recruited from the general population of two Southeast Asian countries (Indonesia and Vietnam)...

BACKGROUND: Few studies have estimated cumulative health care costs post-diagnosis for individuals with autism spectrum disorder (ASD). OBJECTIVES: Using an incidence-based approach, the objective of this analysis was to estimate cumulative costs of ASD to the Ontario health care system of children and adolescents. METHODS: Using administrative health records from Ontario, Canada's most populous province, a retrospective, population-based, incident cohort study of children and adolescents aged 0-19 years old diagnosed with ASD was undertaken to estimate cumulative health care costs of ASD to the health care system from 2010 to 2019...

BACKGROUND: Dementia prevalence is increasing, with no cure at present. Drug therapies have potential side effects and risk of mortality. People with dementia are frequently offered non-pharmacological interventions to improve quality of life and relieve symptoms. Identifying which interventions are cost-effective is important due to finite resources in healthcare services. AIMS: The aims were to review published economic evaluations of community and nursing home non-pharmacological interventions for people with mild cognitive impairment or dementia and assess the usefulness of these evaluations for decision making in health services, for use by policy and local and national decision makers...

As part of the National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation programme, Novartis submitted evidence to support the use of onasemnogene abeparvovec as a treatment option for patients with pre-symptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival of motor neuron (SMN) 1 gene and up to three copies of the SMN2 gene. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned to act as the External Assessment Group (EAG)...

BACKGROUND AND OBJECTIVES: Heart failure (HF) is one of the leading causes of morbidity and mortality, and economic burden on the healthcare system. The aim of this study was to estimate the current hospital resource utilization and costs for HF patients in France. METHODS: This retrospective cohort study included adult HF patients hospitalized in France between January 1, 2019 and December 31, 2019. Data related to sociodemographic characteristics, number and duration of hospital stays, use of medical procedures or expensive and innovative drugs/medical devices included in the "liste-en-sus", and comorbidities were retrieved from the French national hospital discharge database...

BACKGROUND AND AIMS: Risperidone is used in autism spectrum disorder (ASD) to manage aggressive behavior. Budget impact analysis (BIA) assists managers in promoting more sustainable health systems; however, it is unclear whether BIAs underestimate or overestimate the estimates derived from real-world data. This study aimed to compare the estimated BIA values of risperidone use and the monitoring of adverse events in ASD using theoretical and real data. METHODS: Analyses were conducted based on the clinical protocol and the Brazilian therapeutic guidelines for ASD...

BACKGROUND: To utilize EQ-5D in economic evaluations, a societal-based value set is needed. To date, no value sets exist for any EQ-5D instrument in Pakistan. Previous EQ-5D studies conducted in Pakistan 'borrowed' health preferences developed in other countries. However, for a value set to be valid for Pakistani population, it should represent the preferences of the Pakistani population, and culture and living standards of Pakistan. OBJECTIVE: The aim of this study was to derive a Pakistani EQ-5D-3L value set...

BACKGROUND: Non-small cell lung cancer (NSCLC) is the predominant histological subtype of lung cancer and is the leading cause of cancer-related deaths globally. Quality of life is an important consideration for patients and current treatments can adversely affect health-related quality of life (HRQoL). OBJECTIVE: The objectives of this systematic literature review (SLR) were to identify and provide a comprehensive catalogue of published health state utility values (HSUVs) in patients with early-stage NSCLC and to understand the factors impacting on HSUVs in this indication...