Myelodysplasia in a psoriasis patient receiving etanercept : Cause or coincidence?

Peer reviewedFinal Accepted Versio

Basal Insulin Regimens for Adults with Type 1 Diabetes Mellitus : A Cost-Utility Analysis

Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.OBJECTIVES: To assess the cost-effectiveness of basal insulin regimens for adults with type 1 diabetes mellitus in England. METHODS: A cost-utility analysis was conducted in accordance with the National Institute for Health and Care Excellence reference case. The UK National Health Service and personal and social services perspective was used and a 3.5% discount rate was applied for both costs and outcomes. Relative effectiveness estimates were based on a systematic review of published trials and a Bayesian network meta-analysis. The IMS CORE Diabetes Model was used, in which net monetary benefit (NMB) was calculated using a threshold of £20,000 per quality-adjusted life-year (QALY) gained. A wide range of sensitivity analyses were conducted. RESULTS: Insulin detemir (twice daily) [iDet (bid)] had the highest mean QALY gain (11.09 QALYs) and NMB (£181,456) per patient over the model time horizon. Compared with the lowest cost strategy (insulin neutral protamine Hagedorn once daily), it had an incremental cost-effectiveness ratio of £7844/QALY gained. Insulin glargine (od) [iGlarg (od)] and iDet (od) were ranked as second and third, with NMBs of £180,893 and £180,423, respectively. iDet (bid) remained the most cost-effective treatment in all the sensitivity analyses performed except when high doses were assumed (>30% increment compared with other regimens), where iGlarg (od) ranked first. CONCLUSIONS: iDet (bid) is the most cost-effective regimen, providing the highest QALY gain and NMB. iGlarg (od) and iDet (od) are possible options for those for whom the iDet (bid) regimen is not acceptable or does not achieve required glycemic control.Peer reviewe

Moving the needle—what does RSAP look for and what does it aim to do?

Peer reviewe

A Review of Economic Models Submitted to NICE's Technology Appraisal Programme, for Treatments of T1DM & T2DM.

Background: In the UK, 4.7 million people are currently living with diabetes. This is projected to increase to 5 million by 2025. The direct and indirect costs of T1DM and T2DM are rising, and direct costs already account for approximately 10% of the National Health Service (NHS) budget. Objective: The aim of this review is to assess the economic models used in the context of NICE's Technology Appraisals (TA) Programme of T1DM and T2DM treatments, as well as to examine their compliance with the American Diabetes Association's (ADA) guidelines on computer modelling. Methods: A review of the economic models used in NICE's TA programme of T1DM and T2DM treatments was undertaken. Relevant TAs were identified through searching the NICE website for published appraisals completed up to April 2021. The review also examined the associated Evidence Review Group (ERG) reports and Final Appraisal Documents (FAD), which are publicly accessible. ERG reports were scrutinised to identify major issues pertaining to the economic modelling. The FAD documents were then examined to assess how these issues reflected on NICE recommendations. Results: Overall, 10 TAs pertaining to treatments of T1DM and T2DM were identified. Two TAs were excluded as they did not use economic models. Seven of the 8 included TAs related to a novel class of oral antidiabetic drugs (OADs), gliflozins, and one to continuous subcutaneous insulin infusion (CSII) devices. There is a lack of recent, robust data informing risk equations to enable the derivation of transition probabilities. Despite uncertainty surrounding its clinical relevance, bodyweight/BMI is a key driver in many T2DM-models. HbA1c's reliability as a predictor of hard outcomes is uncertain, chiefly for macrovascular complications. The external validity of T1DM is even less clear. There is an inevitable trade-off between the sophistication of models' design, their transparency and practicality. Conclusion: Economic models are essential tools to support decision-making in relation to market access and ascertain diabetes technologies' cost effectiveness. However, key structural and methodological issues exist. Models' shortcomings should be acknowledged and contextualised within the framework of technology appraisals. Diabetes medications and other technologies should also be subject to regular and consistent re-appraisal to inform disinvestment decisions. Artificial intelligence could potentially enhance models' transparency and practicality

Cost Impact Analysis of Using The Improve® Tool For Venous Thromboembolism Risk Assessment In Medical Patients Admitted to The UK NHS Hospitals to Inform NICE Clinical Guideline Recommendation

© 2018 The Author(s). This an open access work distributed under the terms of the Creative Commons Attribution Licence (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.Peer reviewedFinal Accepted Versio

Diagnostics and treatments of COVID-19: two-year update to a living systematic review of economic evaluations

Objectives: As the initial crisis of the COVID-19 pandemic recedes, healthcare decision makers are likely to want to make rational evidence-guided choices between the many interventions now available. We sought to update a systematic review to provide an up-to-date summary of the cost-effectiveness evidence regarding tests for SARS-CoV-2 and treatments for COVID-19.Methods: Key databases, including MEDLINE, EconLit and Embase, were searched on 3 July 2023, 2 years on from the first iteration of this review in July 2021. We also examined health technology assessment (HTA) reports and the citations of included studies and reviews. Peer-reviewed studies reporting full health economic evaluations of tests or treatments in English were included. Studies were quality assessed using an established checklist, and those with very serious limitations were excluded. Data from included studies were extracted into predefined tables.Results: The database search identified 8,287 unique records, of which 54 full texts were reviewed, 28 proceeded for quality assessment, and 15 were included. Three further studies were included through HTA sources and citation checking. Of the 18 studies ultimately included, 17 evaluated treatments including corticosteroids, antivirals and immunotherapies. In most studies, the comparator was standard care. Two studies in lower-income settings evaluated the cost effectiveness of rapid antigen tests and critical care provision. There were 17 modelling analyses and 1 trial-based evaluation.Conclusion: A large number of economic evaluations of interventions for COVID-19 have been published since July 2021. Their findings can help decision makers to prioritise between competing interventions, such as the repurposed antivirals and immunotherapies now available to treat COVID-19. However, some evidence gaps remain present, including head-to-head analyses, disease-specific utility values, and consideration of different disease variants.Systematic Review Registration: [https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021272219], identifier [PROSPERO 2021 CRD42021272219]

Potential Barriers of Patient Involvement in Health Technology Assessment in Central and Eastern European Countries

Patients' perspectives are important to identify preferences, estimate values and appreciate unmet medical needs in the process of research and development and subsequent assessment of new health technologies. Patient and public involvement in health technology assessment (HTA) is essential in understanding and assessing wider implications of coverage and reimbursement decisions for patients, their relatives, caregivers, and the general population. There are two approaches to incorporating the patients' voice in HTA, preferably used in a mix. In the first one, patients, caregivers and/or their representatives directly participate at discussions in different stages of the HTA process, often at the same table with other stakeholders. Secondly, patient involvement activities can be supported by evidence on patient value and experience collected directly from patients, caregivers and/or their representatives often by patient groups Patient involvement practices, however, are limited in Central and Eastern European (CEE) countries without clear methodology or regulatory mechanisms to guide patient involvement in the HTA process. This poses the question of transferability of practices used in other countries, and might call for the development of new CEE-specific guidelines and methods. In this study we aim to map potential barriers of patient involvement in HTA in countries of the CEE region

Effectiveness and cost effectiveness of pharmacist input at the ward level: a systematic review and meta-analysis

Background Pharmacists play important role in ensuring timely care delivery at the ward level. The optimal level of pharmacist input, however, is not clearly defined. Objective To systematically review the evidence that assessed the outcomes of ward pharmacist input for people admitted with acute or emergent illness. Methods The protocol and search strategies were developed with input from clinicians. Medline, EMBASE, Centre for Reviews and Dissemination, The Cochrane Library, NHS Economic Evaluations, Health Technology Assessment and Health Economic Evaluations databases were searched. Inclusion criteria specified the population as adults and young people (age >16 years) who are admitted to hospital with suspected or confirmed acute or emergent illness. Only randomised controlled trials (RCTs) published in English were eligible for inclusion in the effectiveness review. Economic studies were limited to full economic evaluations and comparative cost analysis. Included studies were quality-assessed. Data were extracted, summarised. and meta-analysed, where appropriate. Results Eighteen RCTs and 7 economic studies were included. The RCTs were from USA (n=3), Sweden (n=2), Belgium (n=2), China (n=2), Australia (n=2), Denmark (n=2), Northern Ireland, Norway, Canada, UK and Netherlands. The economic studies were from UK (n=2), Sweden (n=2), Belgium and Netherlands. The results showed that regular pharmacist input was most cost effective. It reduced length-of-stay (mean= -1.74 days [95% CI: -2.76, -0.72], and increased patient and/or carer satisfaction (Relative Risk (RR) =1.49 [1.09, 2.03] at discharge). At £20,000 per quality-adjusted life-year (QALY)-gained cost-effectiveness threshold, it was either cost-saving or cost-effective (Incremental Cost Effectiveness Ratio (ICER) =£632/ QALY-gained). No evidence was found for 7-day pharmacist presence. Conclusions Pharmacist inclusion in the ward multidisciplinary team improves patient safety and satisfaction and is cost-effective when regularly provided throughout the ward stay. Research is needed to determine whether the provision of 7-day service is cost-effective.Peer reviewe

Protocol for the development of SPIRIT and CONSORT extensions for randomised controlled trials with surrogate primary endpoints: SPIRIT-SURROGATE and CONSORT-SURROGATE

Introduction Randomised controlled trials (RCTs) may use surrogate endpoints as substitutes and predictors of patient-relevant/participant-relevant final outcomes (eg, survival, health-related quality of life). Translation of effects measured on a surrogate endpoint into health benefits for patients/participants is dependent on the validity of the surrogate; hence, more accurate and transparent reporting on surrogate endpoints is needed to limit misleading interpretation of trial findings. However, there is currently no explicit guidance for the reporting of such trials. Therefore, we aim to develop extensions to the SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) and CONSORT (Consolidated Standards of Reporting Trials) reporting guidelines to improve the design and completeness of reporting of RCTs and their protocols using a surrogate endpoint as a primary outcome. Methods and analysis The project will have four phases: phase 1 (literature reviews) to identify candidate reporting items to be rated in a Delphi study; phase 2 (Delphi study) to rate the importance of items identified in phase 1 and receive suggestions for additional items; phase 3 (consensus meeting) to agree on final set of items for inclusion in the extensions and phase 4 (knowledge translation) to engage stakeholders and disseminate the project outputs through various strategies including peer-reviewed publications. Patient and public involvement will be embedded into all project phases. Ethics and dissemination The study has received ethical approval from the University of Glasgow College of Medical, Veterinary and Life Sciences Ethics Committee (project no: 200210051). The findings will be published in open-access peer-reviewed publications and presented in conferences, meetings and relevant forums

Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment

There is growing interest in using observational data to assess the safety, effectiveness, and cost effectiveness of medical technologies, but operational, technical, and methodological challenges limit its more widespread use. Common data models and federated data networks offer a potential solution to many of these problems. The open-source Observational and Medical Outcomes Partnerships (OMOP) common data model standardises the structure, format, and terminologies of otherwise disparate datasets, enabling the execution of common analytical code across a federated data network in which only code and aggregate results are shared. While common data models are increasingly used in regulatory decision making, relatively little attention has been given to their use in health technology assessment (HTA). We show that the common data model has the potential to facilitate access to relevant data, enable multidatabase studies to enhance statistical power and transfer results across populations and settings to meet the needs of local HTA decision makers, and validate findings. The use of open-source and standardised analytics improves transparency and reduces coding errors, thereby increasing confidence in the results. Further engagement from the HTA community is required to inform the appropriate standards for mapping data to the common data model and to design tools that can support evidence generation and decision making