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Background: Value of information (VOI) is a tool that can be used to inform decisions concerning additional research in healthcare. VOI estimates the value of obtaining additional information and indicates the optimal design for additional research. Although it is recognized as good practice in handling uncertainty, it is still hardly used in decision making in the Netherlands.Objective: This paper aims to examine the potential value of VOI, barriers and facilitators and the way forward with the use of VOI in the decision-making process for reimbursement of pharmaceuticals in the Netherlands.Methods: Three focus group interviews were conducted with researchers, policy makers, and representatives of pharmaceutical companies.Results: The results revealed that although all stakeholders recognize the relevance of VOI, it is hardly used and many barriers to the performance and use of VOI were identified. One of these barriers is that not all uncertainties are easily incorporated in VOI, and the results may be biased if structural uncertainties are ignored. Furthermore, not all research designs indicated by VOI may be feasible in practice.Conclusions: To fully embed VOI into current decision-making processes, a threshold incremental cost-effectiveness ratio and guidelines that clarify when and how VOI should be performed are needed. In addition, it should be clear to all stakeholders how the results of VOI are used in decision making. [ABSTRACT FROM AUTHOR]

Purpose: To obtain insight into what kind of monitoring technologies exist to monitor activity in-home, what the characteristics and aims of applying these technologies are, what kind of research has been conducted on their effects and what kind of outcomes are reported. Methods: A systematic document search was conducted within the scientific databases Pubmed, Embase, Cochrane, PsycINFO and Cinahl, complemented by Google Scholar. Documents were included in this review if they reported on monitoring technologies that detect activities of daily living (ADL) or significant events, e.g. falls, of elderly people in-home, with the aim of prolonging independent living. Results: Five main types of monitoring technologies were identified: PIR motion sensors, body-worn sensors, pressure sensors, video monitoring and sound recognition. In addition, multicomponent technologies and smart home technologies were identified. Research into the use of monitoring technologies is widespread, but in its infancy, consisting mainly of small-scale studies and including few longitudinal studies. Conclusions: Monitoring technology is a promising field, with applications to the long-term care of elderly persons. However, monitoring technologies have to be brought to the next level, with longitudinal studies that evaluate their (cost-) effectiveness to demonstrate the potential to prolong independent living of elderly persons. [ABSTRACT FROM AUTHOR]

The authors discuss the need to make healthy decisions on the use and reimbursement of health programmes and the need to identify the uncertainties in healthcare decision making before the assessment of the programmes' effectiveness. They believe that the management of healthcare uncertainties can be improved by considering the knowledge and concepts on uncertainty management from other scientific disciplines. The types of uncertainty are cited including parameter uncertainty.

Objective: Point-of-care (POC) D-dimer tests have been developed to exclude deep venous thrombosis quickly and on the spot, but are known to have lower sensitivity compared with laboratory-based tests. Their cost-effectiveness is still unknown. Methods: We updated and extended a previously published Markov model to assess the cost-effectiveness of POC D-dimer tests ('Simplify', 'Cardiac', 'Triage' and 'Nycocard') compared with a laboratory-based latex assay to diagnose deep venous thrombosis in primary care. Results: The 'Laboratory' strategy resulted in 6.986 quality-adjusted life years at the cost of €8354 per patient. All POC D-dimer tests resulted in health outcomes similar to the 'Laboratory' strategy. The 'Simplify' strategy maximized cost savings (-€155 [95% CI: -€246 to -€83]). Conclusions: POC D-dimer tests yield similar health outcomes as laboratory-based testing procedures but can be performed more easily and at lower costs. Therefore, these tests are an alternative to laboratory-based testing and might be considered for exclusion of deep venous thrombosis in primary care. [ABSTRACT FROM AUTHOR]

Introduction: Post-thrombotic syndrome (PTS) is a serious complication of deep vein thrombosis (DVT) of the leg that affects 20-50% of patients. Once a patient experiences PTS there is no treatment that effectively reduces the debilitating complaints. Two randomised controlled trials showed that elastic compression stocking (ECS) therapy after DVT for 24 months can reduce the incidence of PTS by 50%. However, it is unclear whether all patients benefit to the same extent from ECS therapy or what the optimal duration of therapy for individual patients should be. ECS therapy is costly, inconvenient, demanding and sometimes even debilitating. Tailoring therapy to individual needs could save substantial costs. The objective of the IDEAL DVT study, therefore, is to evaluate whether tailoring the duration of ECS therapy on signs and symptoms of the individual patient is a safe and effective method to prevent PTS, compared with standard ECS therapy. Methods and analysis: A multicentre, single-blinded, allocation concealed, randomised, non-inferiority trial. A total of 864 consecutive patients with acute objectively documented proximal DVT of the leg are randomised to either standard duration of 24 months or tailored duration of ECS therapy following an initial therapeutic period of 6 months. Signs and symptoms of PTS are recorded at regular clinic visits. Furthermore, quality of life, costs, patient preferences and compliance are measured. The primary outcome is the proportion of patients with PTS at 24 months. Ethics and dissemination: Based on current knowledge the standard application of ECS therapy is questioned. The IDEAL DVT study will address the central questions that remain unanswered: Which individual patients benefit from ECS therapy and what is the optimal individual treatment duration? Primary ethics approval was received from the Maastricht University Medical Centre. Results: Results of the study will be disseminated via peer-reviewed publications and presentations at scientific conferences. [ABSTRACT FROM AUTHOR]

Abstract: Background: The objective of this study is to systematically review the literature on economic evaluations of interventions for the early diagnosis of Alzheimer's disease (AD) and related disorders and to describe their general and methodological characteristics. We focused on the diagnostic aspects of the decision models to assess the applicability of existing decision models for the evaluation of the recently revised diagnostic research criteria for AD. Methods: PubMed and the National Institute for Health Research Economic Evaluation database were searched for English-language publications related to economic evaluations on diagnostic technologies. Trial-based economic evaluations were assessed using the Consensus on Health Economic Criteria list. Modeling studies were assessed using the framework for quality assessment of decision-analytic models. Results: The search retrieved 2109 items, from which eight decision-analytic modeling studies and one trial-based economic evaluation met all eligibility criteria. Conclusions: Diversity among the study objective and characteristics was considerable and, despite considerable methodological quality, several flaws were indicated. Recommendations were focused on diagnostic aspects and the applicability of existing models for the evaluation of recently revised diagnostic research criteria for AD. [Copyright &y& Elsevier]

Correction to: Mogamulizumab for Previously Treated Mycosis Fungoides and Sézary Syndrome: An Evidence Review Group Perspective of a NICE Single Technology Appraisal 3.3 Cost-Effectiveness Evidence Submitted by the Company, 14th para, final sentence should be removed. Correction to: PharmacoEconomics (2021) https://doi.org/10.1007/s40273-021-01098-3 The following sentence has been added to the end of the abstract of the original online version "This decision was subsequently appealed, and an appeal decision has been reached.". [Extracted from the article]

Background: Health Technology Assessment (HTA) information, and in particular cost-effectiveness data is needed to guide decisions, preferably already in early stages of technological development. However, at that moment there is usually a high degree of uncertainty, because evidence is limited and different development paths are still possible. We developed a multi-parameter framework to assess dynamic aspects of a technology -still in development-, by means of scenario drafting to determine the effects, costs and cost-effectiveness of possible future diffusion patterns. Secondly, we explored the value of this method on the case of the clinical implementation of the 70-gene signature for breast cancer, a gene expression profile for selecting patients who will benefit most from chemotherapy. Methods: To incorporate process-uncertainty, ten possible scenarios regarding the introduction of the 70-gene signature were drafted with European experts. Out of 5 most likely scenarios, 3 drivers of diffusion (non-compliance, technical failure, and uptake) were quantitatively integrated in a decision-analytical model. For these scenarios, the cost-effectiveness of the 70-gene signature expressed in Incremental Cost-Effectiveness Ratios (ICERs) was compared to clinical guidelines, calculated from the past (2005) until the future (2020). Results: In 2005 the ICER was €1,9 million/quality-adjusted-life-year (QALY), meaning that the 70-gene signature was not yet cost-effective compared to the current clinical guideline. The ICER for the 70-gene signature improved over time with a range of €1,9 million to €26,145 in 2010 and €1,9 million to €11,123/QALY in 2020 depending on the separate scenario used. From 2010, the 70-gene signature should be cost-effective, based on the combined scenario. The uptake-scenario had strongest influence on the cost-effectiveness. Conclusions: When optimal diffusion of a technology is sought, incorporating process-uncertainty by means of scenario drafting into a decision model may reveal unanticipated developments and can demonstrate a range of possible cost-effectiveness outcomes. The effect of scenarios give additional information on the speed with cost effectiveness might be reached and thus provide a more realistic picture for policy makers, opinion leaders and manufacturers. [ABSTRACT FROM AUTHOR]

The original article can be found online at https://doi.org/10.1186/s13023-020-01666-4. Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to "TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases"). [Extracted from the article]

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This article discusses the challenges faced in assessing a disease model in the NICE single technology appraisal of tirzepatide for treating type 2 diabetes. The article highlights a significant mismatch between the decision problem and UK clinical practice, as well as the evidence from clinical trials. The use of disease models is advocated, but the assessment of the PRIME T2D model in this appraisal was hindered by limited transparency, adaptability, and confidentiality concerns. The article recommends using standard modeling languages, ensuring model adaptability, and taking extra validation steps to validate specific implementations of disease models. [Extracted from the article]

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. Methods: We elicited expert opinion using a two-round modified Delphi process. The panel consisted of experts and stakeholders in health economic modelling from various professional backgrounds. The first questionnaire concerned definition, terminology, potential applications, issues and recommendations for MUDMs and was based on an exploratory scoping review. In the second round, the panel members were asked to reconsider their input, based on feedback regarding first-round results, and to score issues and recommendations for priority. Finally, adding input from external advisors and policy makers in a structured way, an overview of issues and challenges was developed during two team consensus meetings. Results: In total, 54 respondents contributed to the panel results. The term 'multi-use disease models' was proposed and agreed upon, and a definition was provided. The panel prioritized 10 potential applications (with comparing alternative policies and supporting resource allocation decisions as the top 2), while 20 issues (with model transparency and stakeholders' roles as the top 2) were identified as challenges. Opinions on potential features concerning operationalization of multi-use models were given, with 11 of these subsequently receiving high priority scores (regular updates and revalidation after updates were the top 2). Conclusions: MUDMs would improve on current decision support regarding cost-effectiveness information. Given feasibility challenges, this would be most relevant for diseases with multiple treatments, large burden of disease and requiring more complex models. The current overview offers policy makers a starting point to organize the development, use, and maintenance of MUDMs and to support choices concerning which diseases and policy decisions they will be helpful for. [ABSTRACT FROM AUTHOR]