Challenges and Opportunities in Interdisciplinary Research and Real-World Data for Treatment Sequences in Health Technology Assessments.

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.

Covariates of success in quitting smoking: a systematic review of studies from 2008 to 2021 conducted to inform the statistical analyses of quitting outcomes of a hospital-based tobacco dependence treatment service in the United Kingdom.

Background: Smoking cessation interventions are being introduced into routine secondary care in the United Kingdom (UK), but there are person and setting-related factors that could moderate their success in quitting smoking. This review was conducted as part of an evaluation of the QUIT hospital-based tobacco dependence treatment service ( https://sybics-quit.co.

Critical Appraisal of Decision Models Used for the Economic Evaluation of Bladder Cancer Screening and Diagnosis: A Systematic Review.

Background And Objective: Bladder cancer is common among current and former smokers. High bladder cancer mortality may be decreased through early diagnosis and screening. The aim of this study was to appraise decision models used for the economic evaluation of bladder cancer screening and diagnosis, and to summarise the main outcomes of these models.

Modelling the impact of the coronavirus pandemic on bowel cancer screening outcomes in England: A decision analysis to prepare for future screening disruption.

The English Bowel Cancer Screening Programme invites people between the ages of 60 and 74 to take a Faecal Immunochemical Test every two years. This programme was interrupted during the coronavirus pandemic. The research aimed: (1) to estimate the impact of colorectal cancer (CRC) Faecal Immunochemical Test screening pauses of different lengths and the actual coronavirus-related screening pause in England, and (2) to analyse the most effective and cost-effective strategies to re-start CRC screening to prepare for future disruptions.

Diagnostic accuracy of conventional oral examination for detecting oral cavity cancer and potentially malignant disorders in patients with clinically evident oral lesions: Systematic review and meta-analysis.

This systematic review evaluates the diagnostic accuracy of conventional oral examination (COE) versus incisional or excisional biopsy for the diagnosis of malignant and/or dysplastic lesions in patients with clinically evident lesions. Searches were conducted across five electronic databases from inception to January 2020. Meta-analyses were undertaken, where appropriate.

Calibrating Natural History of Cancer Models in the Presence of Data Incompatibility: Problems and Solutions.

The calibration of cancer natural history models is often challenged by a lack of representative calibration targets, forcing modellers to rely on potentially incompatible datasets. Using a microsimulation colorectal cancer model as an example, the purposes of this paper are to (1) highlight the reasons for uncertainty in calibration targets, (2) illustrate practical and generalisable approaches for dealing with incompatibility in calibration targets, and (3) discuss the importance of future research in the area of incorporating uncertainty in calibration. The low quality of data and differences in populations, outcome definitions, and healthcare systems may result in incompatibility between the model and the data.

The Cost-Effectiveness of Expanding the UK Newborn Bloodspot Screening Programme to Include Five Additional Inborn Errors of Metabolism.

Glutaric aciduria type 1, homocystinuria, isovaleric acidaemia, long-chain hydroxyacyl CoA dehydrogenase deficiency and maple syrup urine disease are all inborn errors of metabolism that can be detected through newborn bloodspot screening. This evaluation was undertaken in 2013 to provide evidence to the UK National Screening Committee for the cost-effectiveness of including these five conditions in the UK Newborn Bloodspot Screening Programme. A decision-tree model with lifetable estimates of outcomes was built with the model structure and parameterisation informed by a systematic review and expert clinical judgment.

Cost-Effectiveness Analysis of Point-of-Care Rapid Testing Versus Laboratory-Based Testing for Antenatal Screening of Syphilis in Brazil.

Objectives: Severe consequences of mother-to-child transmission of syphilis and high increasing incidence of congenital syphilis remains an important public health problem in Brazil. Our objective was to assess the cost-effectiveness of a rapid point-of-care test (RT) and treatment of positive mothers immediately compared with a laboratory-based standard test (ST) with treatment at next follow-up visit.

Methods: A decision analytic model was developed to estimate the incremental cost-effectiveness ratio (ICER) between antenatal syphilis screening strategies.

External Validation of a Colorectal Cancer Model Against Screening Trial Long-Term Follow-Up Data.

Background: The University of Sheffield School of Health and Related Research (ScHARR) Bowel Cancer Screening Model has been used previously to make decisions about colorectal cancer screening strategies in England.

Objectives: The objective of this study was to perform an external validation of the ScHARR model against long-term follow-up data about colorectal cancer (CRC) incidence and mortality reductions due to screening, from the Nottingham trial of guaiac faecal occult blood testing for CRC, and the UK Flexible Sigmoidoscopy Screening Trial.

Methods: The ScHARR model was adapted prior to validation to reflect the setting of each trial in terms of population characteristics, details of screening and surveillance programs, uptake of screening, and further investigations and study follow-up.

A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK.

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built.

Economic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme.

Background: A decision tree model was built to estimate the economic impact of introducing screening for X-linked adrenoleukodystrophy (X-ALD) into an existing tandem mass spectrometry based newborn screening programme. The model was based upon the UK National Health Service (NHS) Newborn Blood Spot Screening Programme and a public service perspective was used with a lifetime horizon. The model structure and parameterisation were based upon literature reviews and expert clinical judgment.

STAKEHOLDER INVOLVEMENT THROUGHOUT HEALTH TECHNOLOGY ASSESSMENT: AN EXAMPLE FROM PALLIATIVE CARE.

Objectives: Internationally, funders require stakeholder involvement throughout health technology assessment (HTA). We report successes, challenges, and lessons learned from extensive stakeholder involvement throughout a palliative care case study that demonstrates new concepts and methods for HTA.

Methods: A 5-step "INTEGRATE-HTA Model" developed within the INTEGRATE-HTA project guided the case study.

COMPREHENSIVE ASSESSMENT OF COMPLEX TECHNOLOGIES: INTEGRATING VARIOUS ASPECTS IN HEALTH TECHNOLOGY ASSESSMENT.

Objectives: Despite recent development of health technology assessment (HTA) methods, there are still methodological gaps for the assessment of complex health technologies. The INTEGRATE-HTA guidance for effectiveness, economic, ethical, socio-cultural, and legal aspects, deals with challenges when assessing complex technologies, such as heterogeneous study designs, multiple stakeholder perspectives, and unpredictable outcomes. The objective of this article is to outline this guidance and describe the added value of integrating these assessment aspects.

A cost-effectiveness analysis of condom distribution programmes for the prevention of sexually transmitted infections in England.

Background: Prevention of sexually transmitted infection (STI) incidence in England is a high priority, particularly among young people, men who have sex with men (MSM) and black ethnic minorities. An economic evaluation of condom distribution programmes (CDPs) to reduce STI transmission is presented.

Methods: An economic model using a Bernoulli process estimated the number of people acquiring an STI as a function of its prevalence, transmission rate, condom use, condom failure rate and number of sexual contacts.

A Framework for Developing the Structure of Public Health Economic Models.

Background: A conceptual modeling framework is a methodology that assists modelers through the process of developing a model structure. Public health interventions tend to operate in dynamically complex systems. Modeling public health interventions requires broader considerations than clinical ones.

Lay and professional stakeholder involvement in scoping palliative care issues: Methods used in seven European countries.

Background: Stakeholders are people with an interest in a topic. Internationally, stakeholder involvement in palliative care research and health technology assessment requires development. Stakeholder involvement adds value throughout research (from prioritising topics to disseminating findings).

A systematic literature review of the key challenges for developing the structure of public health economic models.

Objectives: To identify the key methodological challenges for public health economic modelling and set an agenda for future research.

Methods: An iterative literature search identified papers describing methodological challenges for developing the structure of public health economic models. Additional multidisciplinary literature searches helped expand upon important ideas raised within the review.

Avoiding and identifying errors and other threats to the credibility of health economic models.

Health economic models have become the primary vehicle for undertaking economic evaluation and are used in various healthcare jurisdictions across the world to inform decisions about the use of new and existing health technologies. Models are required because a single source of evidence, such as a randomised controlled trial, is rarely sufficient to provide all relevant information about the expected costs and health consequences of all competing decision alternatives. Whilst models are used to synthesise all relevant evidence, they also contain assumptions, abstractions and simplifications.

Using resource modelling to inform decision making and service planning: the case of colorectal cancer screening in Ireland.

Background: Organised colorectal cancer screening is likely to be cost-effective, but cost-effectiveness results alone may not help policy makers to make decisions about programme feasibility or service providers to plan programme delivery. For these purposes, estimates of the impact on the health services of actually introducing screening in the target population would be helpful. However, these types of analyses are rarely reported.

The Multiple Sclerosis Risk Sharing Scheme Monitoring Study--early results and lessons for the future.

Background: Risk sharing schemes represent an innovative and important approach to the problems of rationing and achieving cost-effectiveness in high cost or controversial health interventions. This study aimed to assess the feasibility of risk sharing schemes, looking at long term clinical outcomes, to determine the price at which high cost treatments would be acceptable to the NHS.

Methods: This case study of the first NHS risk sharing scheme, a long term prospective cohort study of beta interferon and glatiramer acetate in multiple sclerosis (MS) patients in 71 specialist MS centres in UK NHS hospitals, recruited adults with relapsing forms of MS, meeting Association of British Neurologists (ABN) criteria for disease modifying therapy.

Use of expert knowledge in evaluating costs and benefits of alternative service provisions: a case study.

Objectives: A treatment pathway model was developed to examine the costs and benefits of the current bowel cancer service in England and to evaluate potential alternatives in service provision. To use the pathway model, various parameters and probability distributions had to be specified. They could not all be determined from empirical evidence and, instead, expert opinion was elicited in the form of statistical quantities that gave the required information.

A stated preference binary choice experiment to explore NICE decision making.

Objective: To explore whether the National Institute for Health and Clinical Excellence (NICE) takes account of concerns other than just incremental cost effectiveness in commissioning healthcare services.

Method: A stated preference binary choice experiment was used to explore the preferences of members of NICE's Appraisal Committees for incremental cost effectiveness, the degree of uncertainty surrounding incremental costs and health outcomes, the age of beneficiaries, baseline health-related quality of life (HR-QOL) and the availability of alternative therapies when considering whether to recommend health technologies.

Results: A logit modelling analysis of Committee members' stated preferences suggested that increases in the incremental cost-effectiveness ratio and economic uncertainty, and the availability of other therapies was associated with statistically significant reductions in the odds of a positive recommendation (p < 0.

Option appraisal of population-based colorectal cancer screening programmes in England.

Objectives: To estimate the effectiveness, cost-effectiveness and resource impact of faecal occult blood testing (FOBT) and flexible sigmoidoscopy (FSIG) screening options for colorectal cancer to inform the Department of Health's policy on bowel cancer screening in England.

Methods: We developed a state transition model to simulate the life experience of a cohort of individuals without polyps or cancer through to the development of adenomatous polyps and malignant carcinoma and subsequent death in the general population of England. The costs, effects and resource impact of five screening options were evaluated: (a) FOBT for individuals aged 50-69 (biennial screening); (b) FOBT for individuals aged 60-69 (biennial screening); (c) once-only FSIG for individuals aged 55; (d) once-only FSIG for individuals aged 60; and (e) once-only FSIG for individuals aged 60, followed by FOBT for individuals aged 61-70 (biennial screening).

Economics of tandem mass spectrometry screening of neonatal inherited disorders.

Objectives: The aim of this study was to evaluate the cost-effectiveness of neonatal screening for phenylketonuria (PKU) and medium-chain acyl-coA dehydrogenase (MCAD) deficiency using tandem mass spectrometry (tandem MS).

Methods: A systematic review of clinical efficacy evidence and cost-effectiveness modeling of screening in newborn infants within a UK National Health Service perspective was performed. Marginal costs, life-years gained, and cost-effectiveness acceptability curves are presented.