PharmacoEconomics

BACKGROUND AND OBJECTIVES: There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they have not been summarised in a comprehensive manner. For all stakeholders working with rare diseases, it is important to be aware and understand these issues. The objective of this review is to identify the main challenges for the economic evaluation of orphan drugs in rare diseases...

BACKGROUND: The current use of health economic decision models in HTA is mostly confined to single use cases, which may be inefficient and result in little consistency over different treatment comparisons, and consequently inconsistent health policy decisions, for the same disorder. Multi-use disease models (MUDMs) (other terms: generic models, whole disease models, disease models) may offer a solution. However, much is uncertain about their definition and application. The current research aimed to develop a blueprint for the application of MUDMs...

BACKGROUND AND OBJECTIVE: Decision models for health technology assessment (HTA) are largely submitted to HTA agencies using commercial software, which has known limitations. The use of the open-source programming language R has been suggested because of its efficiency, transparency, reproducibility, and ability to consider complex analyses. However, its use in HTA remains limited. This qualitative study aimed to explore the main reasons for this slow uptake of R in HTA and identify tangible facilitators...

OBJECTIVES: Accurate risk stratification of patients with stage II and III colorectal cancer (CRC) prior to treatment selection enables limited health resources to be efficiently allocated to patients who are likely to benefit from adjuvant chemotherapy. We aimed to investigate the cost-effectiveness of a recently developed deep learning-based prognostic method, Histotyping, from the perspective of the Norwegian healthcare system. METHODS: Two partitioned survival models were developed to assess the cost-effectiveness of Histotyping for two treatment cohorts: patients with CRC stage II and III...

Value of Information (VOI) analyses calculate the economic value that could be generated by obtaining further information to reduce uncertainty in a health economic decision model. VOI has been suggested as a tool for research prioritisation and trial design as it can highlight economically valuable avenues for future research. Recent methodological advances have made it increasingly feasible to use VOI in practice for research; however, there are critical differences between the VOI approach and the standard methods used to design research studies such as clinical trials...

BACKGROUND: Assessing the cost-effectiveness of interventions targeting childhood excess weight requires estimates of the hazards of transitioning between weight status categories. Current estimates are based on studies characterized by insufficient sample sizes, a lack of national representativeness, and untested assumptions. OBJECTIVES: We sought to (1) estimate transition probabilities and hazard ratios for transitioning between childhood weight status categories, (2) test the validity of the underlying assumption in the literature that transitions between childhood bodyweight categories are time-homogeneous, (3) account for complex sampling procedures when deriving nationally representative transition estimates, and (4) explore the impact of child, maternal, and sociodemographic characteristics...

With an ever-increasing number of treatment options, the assessment of treatment sequences has become crucial in health technology assessment (HTA). This review systematically explores the multifaceted challenges inherent in evaluating sequences, delving into their interplay and nuances that go beyond economic model structures. We synthesised a 'roadmap' of literature from key methodological studies, highlighting the evolution of recent advances and emerging research themes. These insights were compared against HTA guidelines to identify potential avenues for future research...

BACKGROUND: The increasing number of studies that generate health state utility values (HSUVs) and the impact of HSUVs on cost-utility analyses make a robust tailored quality appraisal (QA) tool for systematic reviews of these studies necessary. OBJECTIVE: This study aimed to address conceptual issues regarding QA in systematic reviews of studies eliciting HSUVs by establishing a consensus on the definitions, dimensions and scope of a QA tool specific to this context...

INTRODUCTION: The rate of development and complexity of digital health interventions (DHIs) in recent years has to some extent outpaced the methodological development in economic evaluation and costing. Particularly, the choice of cost components included in intervention or program costs of DHIs have received scant attention. The aim of this study was to build a literature-informed checklist of program cost components of DHIs. The checklist was next tested by applying it to an empirical case, Mamma Mia, a DHI developed to prevent perinatal depression...

No abstract text is available yet for this article.

No abstract text is available yet for this article.

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...

No abstract text is available yet for this article.

No abstract text is available yet for this article.

INTRODUCTION: Prior discrete choice experiments (DCE) in oncology found that, on average, clinicians rank survival as the most important treatment attribute. We investigate heterogeneity in clinician preferences within the context of first-line treatment for advanced urothelial carcinoma in Spain, France, Italy, Germany, and the UK. METHODS: The online DCE included 12 treatment choice tasks, each comparing two hypothetical therapy profiles defined by treatment attributes: grade 3/4 treatment-related adverse events (TRAEs), induction and maintenance administration schedules, progression-free survival, and overall survival (OS)...

No abstract text is available yet for this article.

OBJECTIVES: This study aimed to develop population norms for three preference-accompanied measures [EQ-5D-5L, Patient-Reported Outcomes Measurement Information System (PROMIS)-preference scoring system (PROPr) and Short-Form Six-Dimension (SF-6D)] in Hungary. METHODS: In November 2020, an online cross-sectional survey was conducted among a representative sample of the Hungarian adult general population (n = 1631). Respondents completed the Hungarian versions of the EQ-5D-5L, PROMIS-29+2 version 2...

INTRODUCTION: Children may find self-reporting health-related quality of life (HRQoL) using patient-reported outcome measures (PROMs) presented in text-based formats difficult, particularly younger children and children with developmental delays or chronic illness. In such cases, pictorial PROMs (where pictorial representations are used alongside or to replace text) may offer a valid alternative. AIM: This systematic literature review focused on identifying and describing paediatric PROMs that incorporate pictorial approaches, providing children with more effective means to express their HRQoL...

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)...

OBJECTIVE: The aim of this study was to examine the validity, reliability and responsiveness of common generic paediatric health-related quality of life (HRQoL) instruments in children and adolescents with mental health challenges. METHODS: Participants were a subset of the Australian Paediatric Multi-Instrument Comparison (P-MIC) study and comprised 1013 children aged 4-18 years with attention-deficit/hyperactivity disorder (ADHD) (n = 533), or anxiety and/or depression (n = 480)...