A structured process for the validation of a decision-analytic model: application to a cost-effectiveness model for risk-stratified national breast screening.

Background: Decision-makers require knowledge of the strengths and weaknesses of decision-analytic models used to evaluate healthcare interventions to be able to confidently use the results of such models to inform policy. A number of aspects of model validity have previously been described, but no systematic approach to assessing the validity of a model has been proposed. This study aimed to consolidate the different aspects of model validity into a step-by-step approach to assessing the strengths and weaknesses of a decision-analytic model.

Trade-offs between overall survival and side effects in the treatment of metastatic breast cancer: eliciting preferences of patients with primary and metastatic breast cancer using a discrete choice experiment.

Objectives: There has been a recent proliferation in treatment options for patients with metastatic breast cancer. Such treatments often involve trade-offs between overall survival and side effects. Our study aims to estimate the trade-offs that could be used to inform decision-making at the individual and policy level.

Global serum profiling: an opportunity for earlier cancer detection.

The advances in cancer research achieved in the last 50 years have been remarkable and have provided a deeper knowledge of this disease in many of its conceptual and biochemical aspects. From viewing a tumor as a 'simple' aggregate of mutant cells and focusing on detecting key cell changes leading to the tumorigenesis, the understanding of cancer has broadened to consider it as a complex organ interacting with its close and far surroundings through tumor and non-tumor cells, metabolic mechanisms, and immune processes. Metabolism and the immune system have been linked to tumorigenesis and malignancy progression along with cancer-specific genetic mutations.

Variation in hospital cost trajectories at the end of life by age, multimorbidity and cancer type.

Background: Approximately thirty thousand people in Scotland are diagnosed with cancer annually, of whom a third live less than one year. The timing, nature and value of hospital-based healthcare for patients with advanced cancer are not well understood. The study's aim was to describe the timing and nature of hospital-based healthcare use and associated costs in the last year of life for patients with a cancer diagnosis.

Clinical validation of a spectroscopic liquid biopsy for earlier detection of brain cancer.

Background: Diagnostic delays impact the quality of life and survival of patients with brain tumors. Earlier and expeditious diagnoses in these patients are crucial to reduce the morbidities and mortalities associated with brain tumors. A simple, rapid blood test that can be administered easily in a primary care setting to efficiently identify symptomatic patients who are most likely to have a brain tumor would enable quicker referral to brain imaging for those who need it most.

Feasibility study for interactive reporting of network meta-analysis: experiences from the development of the MetaInsight COVID-19 app for stakeholder exploration, re-analysis and sensitivity analysis from living systematic reviews.

Background: Network meta-analysis (NMA) has been increasingly adopted worldwide by Cochrane reviews, guideline developers and decision-making bodies to identify optimal treatment choices. However, NMA results are often produced statically, not allowing stakeholders to 'dig deeper' and interrogate with their own judgement. Additionally, amid the COVID-19 pandemic, unnecessary or duplicated reviews have been proposed which analyse from the same pool of evidence.

From Spreadsheets to Script: Experiences From Converting a Scottish Cardiovascular Disease Policy Model into R.

Given the advantages in transparency, reproducibility, adaptability and computational efficiency in R, there is a growing interest in converting existing spreadsheet-based models into an R script for model re-use and upskilling training among health economic modellers. The objective of this exercise was to convert the Scottish Cardiovascular Disease (CVD) Policy Model from Excel to R and discuss the lessons learnt throughout this process. The CVD model is a competing risk state transition cohort model.

The impact of the Covid-19 pandemic on breast cancer early detection and screening.

The COVID-19 pandemic affects mortality and morbidity, with disruptions expected to continue for some time, with access to timely cancer-related services a concern. For breast cancer, early detection and treatment is key to improved survival and longer-term quality of life. Health services generally have been strained and in many settings with population breast mammography screening, efforts to diagnose and treat breast cancers earlier have been paused or have had reduced capacity.

Content of Health Economics Analysis Plans (HEAPs) for Trial-Based Economic Evaluations: Expert Delphi Consensus Survey.

Objectives: Health economics analysis plans (HEAPs) currently lack consistency, with uncertainty surrounding appropriate content. We aimed to develop a list of essential items that should be included in HEAPs for economic evaluations conducted alongside randomized trials.

Methods: A list of potential items for inclusion was developed by examining existing HEAPs.

Early economic evaluation to guide the development of a spectroscopic liquid biopsy for the detection of brain cancer.

Objectives: An early economic evaluation to inform the translation into clinical practice of a spectroscopic liquid biopsy for the detection of brain cancer. Two specific aims are (1) to update an existing economic model with results from a prospective study of diagnostic accuracy and (2) to explore the potential of brain tumor-type predictions to affect patient outcomes and healthcare costs.

Methods: A cost-effectiveness analysis from a UK NHS perspective of the use of spectroscopic liquid biopsy in primary and secondary care settings, as well as a cost-consequence analysis of the addition of tumor-type predictions was conducted.

Healthcare use and costs in the last year of life: a national population data linkage study.

Background: People who are nearing the end of life are high users of healthcare. The cost to providers is high and the value of care is uncertain.

Objectives: To describe the pattern, trajectory and drivers of secondary care use and cost by people in Scotland in their last year of life.

Long-term outcome of partial P450 side-chain cleavage enzyme deficiency in three brothers: the importance of early diagnosis.

Objective: CYP11A1 mutations cause P450 side-chain cleavage (scc) deficiency, a rare form of congenital adrenal hyperplasia with a wide clinical spectrum. We detail the phenotype and evolution in a male sibship identified by HaloPlex targeted capture array.

Family Study: The youngest of three brothers from a non-consanguineous Scottish family presented with hyperpigmentation at 3.

Chemotherapy effectiveness in trial-underrepresented groups with early breast cancer: A retrospective cohort study.

Background: Adjuvant chemotherapy in early stage breast cancer has been shown to reduce mortality in a large meta-analysis of over 100 randomised trials. However, these trials largely excluded patients aged 70 years and over or with higher levels of comorbidity. There is therefore uncertainty about whether the effectiveness of adjuvant chemotherapy generalises to these groups, hindering patient and clinician decision-making.

Barriers and Facilitators to Model Replication Within Health Economics.

Background: Model replication is important because it enables researchers to check research integrity and transparency and, potentially, to inform the model conceptualization process when developing a new or updated model.

Objective: The aim of this study was to evaluate the replicability of published decision analytic models and to identify the barriers and facilitators to replication.

Methods: Replication attempts of 5 published economic modeling studies were made.

Real-world evidence was feasible for estimating effectiveness of chemotherapy in breast cancer: a cohort study.

Objective: Evidence-based guidelines recommend adjuvant chemotherapy in early stage breast cancer whenever treatment benefit is considered sufficient to outweigh the associated risks. However, many groups of patients were either excluded from or underrepresented in the clinical trials that form the evidence base for this recommendation. This study aims to determine whether using administrative health care data-real world data-and econometric methods for causal analysis to provide "real world evidence" (RWE) are feasible methods for addressing this gap.

Independent validation of the PREDICT breast cancer prognosis prediction tool in 45,789 patients using Scottish Cancer Registry data.

Background: PREDICT is a widely used online prognostication and treatment benefit tool for patients with early stage breast cancer. The aim of this study was to conduct an independent validation exercise of the most up-to-date version of the PREDICT algorithm (version 2) using real-world outcomes from the Scottish population of women with breast cancer.

Methods: Patient data were obtained for all Scottish Cancer Registry (SCR) records with a diagnosis of primary invasive breast cancer diagnosed in the period between January 2001 and December 2015.

Survival estimates stratified by the Nottingham Prognostic Index for early breast cancer: a systematic review and meta-analysis of observational studies.

Background: Estimates of survival for women diagnosed with early staged breast cancer are available based on stratification into prognostic categories defined using the Nottingham Prognostic Index (NPI). This review aimed to identify and summarize the estimated survival statistics from separate sources in the literature and to explore the extent of between-study heterogeneity in survival estimates.

Methods: Observational studies in women diagnosed with early and locally advanced breast cancer reporting overall survival by NPI category were identified using a systematic literature search.

Health economic evaluation of a serum-based blood test for brain tumour diagnosis: exploration of two clinical scenarios.

Objectives: To determine the potential costs and health benefits of a serum-based spectroscopic triage tool for brain tumours, which could be developed to reduce diagnostic delays in the current clinical pathway.

Design: A model-based health pre-trial economic assessment. Decision tree models were constructed based on simplified diagnostic pathways.

Evaluation of a Stratified National Breast Screening Program in the United Kingdom: An Early Model-Based Cost-Effectiveness Analysis.

Objectives: To identify the incremental costs and consequences of stratified national breast screening programs (stratified NBSPs) and drivers of relative cost-effectiveness.

Methods: A decision-analytic model (discrete event simulation) was conceptualized to represent four stratified NBSPs (risk 1, risk 2, masking [supplemental screening for women with higher breast density], and masking and risk 1) compared with the current UK NBSP and no screening. The model assumed a lifetime horizon, the health service perspective to identify costs (£, 2015), and measured consequences in quality-adjusted life-years (QALYs).

How do individuals' health behaviours respond to an increase in the supply of health care? Evidence from a natural experiment.

The efficacy of the management of long-term conditions depends in part on whether healthcare and health behaviours are complements or substitutes in the health production function. On the one hand, individuals might believe that improved health care can raise the marginal productivity of their own health behaviour and decide to complement health care with additional effort in healthier behaviours. On the other hand, health care can lower the cost of unhealthy behaviours by compensating for their negative effects.

Discrete choice experiments of pharmacy services: a systematic review.

Background Two previous systematic reviews have summarised the application of discrete choice experiments to value preferences for pharmacy services. These reviews identified a total of twelve studies and described how discrete choice experiments have been used to value pharmacy services but did not describe or discuss the application of methods used in the design or analysis. Aims (1) To update the most recent systematic review and critically appraise current discrete choice experiments of pharmacy services in line with published reporting criteria and; (2) To provide an overview of key methodological developments in the design and analysis of discrete choice experiments.

Valuing Preferences for the Process and Outcomes of Clinical Genetics Services: A Pilot Study.

Background: Understanding preferences for the process and outcomes of clinical genetics services (CGS) is a first step to developing these services appropriately.

Aim: The aim of this study was to quantify the relative importance of attributes defining the process of service delivery and the patient outcomes of CGS.

Methods: An online hybrid conjoint analysis discrete choice experiment (CA-DCE) was piloted in a purposive sample (n = 37) of CGS patients and non-patients to identify (i) service attributes (n = 13) perceived to facilitate informed decision making; (ii) relative preferences for six attributes (5 process, 1 outcome: ability to make an informed decision).

Health economics education in undergraduate medical degrees: an assessment of curricula content and student knowledge.

Objective: To define the structure and content of health economics teaching in undergraduate medical degrees in the UK, and identify and quantify differences in student knowledge, with a view to informing the health economics curricula.

Methods: Semi-structured interviews with senior teaching staff in three Medical Schools, a review of course documentation, and an online survey to assess student knowledge. The survey was scored and mean scores were compared across medical schools, year of study, and teaching components, including the professional background of the teachers.