Health Technology Assessment Research Articles

Erratum for The Effectiveness of Spiritual Competence Training on the Psychological Well-being of Preschool Teachers. Health Technology Assessment in Action [Health Tech Asmnt Act. 2023; 6(2)

The Article Abstract is not available

Erratum for Evaluation of Validity Indices of Spiritual Competence Questionnaire. Health Technology Assessment in Action [Health Tech Asmnt Act. 2021; 5(1)

The Article Abstract is not available

Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia.

Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations. A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation. The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations. This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.

Outcomes of guidelines from health technology assessment organizations in community-based primary care: a systematic mixed studies review.

Health technology assessment (HTA) organizations generate guidelines to inform healthcare practices toward improved health outcomes. This review sought to identify and classify outcomes of guidelines from HTA organizations within published research. We performed a systematic mixed studies review of empirical studies that (a) referred to a published guideline from an HTA organization and (b) reported an outcome resulting from a guideline. We searched the published literature in English or French within seven databases. Outcome types were classified within five dimensions of an existing framework for online health information (e.g., relevance, cognitive/affective impact, and use). Subdimensions were inductively developed. A two-phase sequential data synthesis was performed. Phase 1: a hybrid deductive-inductive thematic analysis identified the types of outcomes and displayed their relationships on a concept map. Phase 2: descriptive statistics were tabulated by the type of outcome. A total of 6,719 records were retrieved through searches on 6 February 2023. After screening, we included 120 observational studies (twenty-one qualitative, ninety-four quantitative, and five mixed methods). Phase 1 identified twenty-nine types of outcomes. The most frequently reported outcomes were within the organizational dimension (reported in ninety-four studies). The most common subdimensions were "Referrals" (thirty-eight occurrences), the "Quality of Prescriptions" (fifteen occurrences), and the "Quality of Diagnosis" (eight occurrences). For Phase 2, we could only generate descriptive statistics on seventeen outcomes. These were almost equally distributed among positive, neutral, and negative effects. Our results contribute to knowledge about the outcomes of HTA guidelines and options for documenting and measuring them in future evaluations.

Challenges associated with access to recently developed hemophilia treatments in routine care: perspectives of healthcare professionals.

The treatment landscape for haemophilia continues to rapidly develop, and expectations for future treatment success are high. There is limited information on the challenges to accessing new and innovative therapies. The aim of this study was to explore challenges with accessing haemophilia treatment from the perspective of healthcare professionals (HCPs). A crosssectional study design was used. A pilot-tested, online survey was distributed to haemophilia treatment centres in Australia, Canada, France, Italy, New Zealand, Republic of Ireland, Turkey, the United States, and the United Kingdom. The questionnaire covered questions on product access, economic considerations, health technology assessment requirements, and patient organization involvement. The results were analyzed descriptively using SPSS. A total of 154 HCPs completed the questionnaire. There was heterogeneity across countries, regions, and centres regarding HCPs' knowledge of access to novel recently developed treatments. Notable limitations to access were reported such as differences in access based on age of patient and type of product, economic considerations, and the growing influence of HTA bodies. Many countries have a hemophilia patient organization that does not have a vote at the decision-making table. There is a need to empower HCPs to better understand national healthcare structures and decisions that lead to access limitations. Requirements from HTA bodies must be understood to optimally design clinical studies and value generation of treatment options. This may strengthen the haemophilia treatment centre's voice to collectively mandate for exchange with key involved individuals, such as the payers and politicians for the provision of optimal therapy.

Indirect Treatment Comparisons in Healthcare Decision Making: A Targeted Review of Regulatory Approval, Reimbursement, and Pricing Recommendations Globally for Oncology Drugs in 2021-2023.

Indirect treatment comparisons (ITCs) evaluate novel treatments compared to appropriate comparators when direct evidence is unavailable or infeasible. The objective of this study was to highlight the prevalence of different ITC methods in oncology drug submissions and to provide insights into how ITCs have been used in recent regulatory approval, reimbursement recommendations, or pricing decisions across various regions and diverse assessment frameworks. A targeted literature review was conducted to identify assessment documents for oncology drug submissions that included ITCs. This included hand searches of the websites of four regulatory bodies and four health technology assessment (HTA) agencies with varying assessment frameworks across North America, Europe, and Asia-Pacific. A total of 185 documents were included for synthesis. Documents were retrieved from all four HTA agencies and the European Medicines Agency (EMA), the only regulatory body with eligible records. Within these, 188 unique submissions included a total of 306 supporting ITCs of various methods. Authorities more frequently favored anchored or population-adjusted ITC techniques for their effectiveness in data adjustment and bias mitigation. Furthermore, ITCs in orphan drug submissions more frequently led to positive decisions compared to non-orphan submissions. This review highlights the crucial role and widespread use of ITCs in global healthcare decision-making, particularly when direct evidence is lacking, and in the discernment of market-specific clinical benefits. This work contributes to bolstering the credibility and recognition of ITCs across regulatory and HTA agencies of diverse regions and assessment frameworks.

The Use of Real-World Data for Estimating Relative Treatment Effects in NICE Health Technology Assessment Submissions: A Review.

This paper investigates the current use of real-world data (RWD) for estimating relative treatment effects in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions. This review included 64 HTA submissions, which accounted for approximately 11% of the total NICE HTA submissions between January 2016 and December 2023. The main sources of RWD considered in the submissions were disease registries and electronic health records. RWD were primarily used to provide an external control arm to enable comparisons within single-arm trials and to inform long-term treatment effects when extrapolating survival data beyond the trial follow-up. Adjustments for potential systematic differences between treatment groups have improved over time; however, approximately one-third of the submissions still relied on unadjusted treatment comparisons. We found that approximately 10% of NICE HTA submissions used RWD to directly inform treatment effects estimation. Over one-third of the submissions relied on naïve and/or unadjusted treatment comparisons, which may have introduced biases. To ensure that RWD provide sound evidence for HTA, submissions should follow published guidelines, including the NICE real-world evidence (RWE)framework.

Valuing the Societal Impact of Medicines and Other Health Technologies: A User Guide to Current Best Practices.

This study argues that value assessment conducted from a societal perspective should rely on the Generalized Cost-Effectiveness Analysis (GCEA) framework proposed herein. Recently developed value assessment inventories- such as the Second Panel on Cost-Effectiveness's "impact inventory" and International Society of Pharmacoeconomics Outcomes Research (ISPOR) "value flower"- aimed to more comprehensively capture the benefits and costs of new health technologies from a societal perspective. Nevertheless, application of broader value elements in practice has been limited in part because quantifying these elements can be complex, but also because there have been numerous methodological advances since these value inventories have been released (e.g. generalized and risk-adjusted cost effectiveness). To facilitate estimation of treatment value from a societal perspective, this paper provides an updated value inventory- called the GCEA value flower- and a user guide for implementing GCEAfor health economics researchers and practitioners. GCEA considers 15 broader value elements across four categories: (i)uncertainty, (ii) dynamics, (iii)beneficiary, and (iv) additional value components. The uncertainty category incorporates patient risk preferences into value assessment. The dynamics category petals account for the evolution of real-world treatment value (e.g. option value) and includes drug pricing trends (e.g. future genericization). The beneficiary category accounts for the fact health technologies can benefit others (e.g. caregivers) and also that society may care to whom health benefits accrue (e.g. equity). Finally, GCEA incorporates additional broader sources of value (e.g. community spillovers, productivity losses). This GCEA user guide aims to facilitate both the estimation of each of these value elements and the incorporation of these values into health technology assessment when conducted from a societal perspective.

Economic evaluations of technology-based interventions used to provide care support for people with mild dementia or mild cognitive impairment and their caregivers: A systematic review.

The cost-effectiveness of interventions is a key issue owing to the limited resources of healthcare services. To conduct a systematic review of economic evaluations of technology-based healthcare interventions in care support for people with dementia or mild cognitive impairment (MCI) and their caregivers, and of the tools used to assess effectiveness and costs. The following databases were used: PubMed, National Health Service Economic Evaluation Database, and Health Technology Assessment. A total of 207 articles from 2012 to 2024 were identified and then screened. Seventeen studies were included, of which nine were study protocols. Almost half (n = 8) the interventions were multicomponent. The most common components used in the interventions were cognitive stimulation, physical functioning and continuing support. Regarding the efficiency results of these interventions, only three studies provided a full economic evaluation. The most frequent tools in the economic evaluations used to measure effectiveness (measured in quality-adjusted life years) and costs were the European Quality of Life-5 Dimensions and Resource Utilization in Dementia instruments, respectively. Most of the interventions evaluated were cost-effective. However, these results should be interpreted with caution, given the scarcity of the literature, and further economic evaluations of technology-based healthcare interventions for people with mild dementia or MCI care support and their caregivers are therefore needed. Additionally, a meta-analysis could not be performed due to the heterogeneity of the data.

Assessing Methods for Adjusting Estimates of Treatment Effectiveness for Patient Nonadherence in the Context of Time-to-Event Outcomes and Health Technology Assessment: A Simulation Study.

We aim to assess the performance of methods for adjusting estimates of treatment effectiveness for patient nonadherence in the context of health technology assessment using simulation methods. We simulated trial datasets with nonadherence, prognostic characteristics, and a time-to-event outcome. The simulated scenarios were based on a trial investigating immunosuppressive treatments for improving graft survival in patients who had had a kidney transplant. The primary estimand was the difference in restricted mean survival times in all patients had there been no nonadherence. We compared generalized methods (g-methods; marginal structural model with inverse probability of censoring weighting [IPCW], structural nested failure time model [SNFTM] with g-estimation) and simple methods (intention-to-treat [ITT] analysis, per-protocol [PP] analysis) in 90 scenarios each with 1,900 simulations. The methods' performance was primarily assessed according to bias. In implementation nonadherence scenarios, the average percentage bias was 20% (ranging from 7% to 37%) for IPCW, 20% (8%-38%) for SNFTM, 20% (8%-38%) for PP, and 40% (20%-75%) for ITT. In persistence nonadherence scenarios, the average percentage bias was 26% (9%-36%) for IPCW, 26% (14%-39%) for SNFTM, 26% (14%-36%) for PP, and 47% (16%-72%) for ITT. In initiation nonadherence scenarios, the percentage bias ranged from -29% to 110% for IPCW, -34% to 108% for SNFTM, -32% to 102% for PP, and between -18% and 200% for ITT. In this study, g-methods and PP produced more accurate estimates of the treatment effect adjusted for nonadherence than the ITT analysis did. However, considerable bias remained in some scenarios. Randomized controlled trials are usually analyzed using the intention-to-treat (ITT) principle, which produces a valid estimate of effectiveness relating to the underlying trial, but when patient adherence to medications in the real world is known to differ from that observed in the trial, such estimates are likely to result in a biased representation of real-world effectiveness and cost-effectiveness.Our simulation study demonstrates that generalized methods (g-methods; IPCW, SNFTM) and per-protocol analysis provide more accurate estimates of the treatment effect than the ITT analysis does, when adjustment for nonadherence is required; however, even with these adjustment methods, considerable bias may remain in some scenarios.When real-world adherence is expected to differ from adherence observed in a trial, adjustment methods should be used to provide estimates of real-world effectiveness.

Health Technology Assessment for digital health technologies in India: A framework for action.

Health Technology Assessment for digital health technologies in India: A framework for action.

Patient experiences of treatment-resistant depression (TRD): A systematic review and qualitative meta-synthesis

Treatment-resistant depression (TRD) occurs when depressive symptoms persist after a patient has tried at least two antidepressants at an appropriate dose for an adequate length of time, as judged by their clinician. Understanding what it is like to live with and seek care for TRD can inform treatment planning and contribute to health technology assessments for depression-related therapies. Our objective was to systematically review this evidence through an investigation of qualitative literature about the experiences of people who have TRD. We searched MEDLINE, CINAHL, PsycINFO, and the Web of Science Core Collection. Publications eligible for inclusion were English-language primary empirical qualitative studies or the qualitative component of mixed methods studies examining adults’ experiences with TRD or with specific treatment options for TRD. Two reviewers independently screened and extracted data, as well as critically appraised included studies using the Standards for Reporting Qualitative Research (SRQR) instrument. This review was guided by integrative meta-synthesis. Data analysis consisted of a staged coding process similar to grounded theory. The protocol was registered in PROSPERO prior to the search (record ID: CRD42022356813). Sixteen studies were included from screening 18,568 results. Two studies focused on the overall experience of TRD and 14 explored experiences of specific treatments such as ketamine. Patients described the depressive symptoms they experienced, a sense of futility and desperation to find an effective treatment, and their experiences with new, sometimes experimental, therapies including deriving benefits beyond the clinical. Overall, few studies have examined the patient experience of TRD, reflecting the clinical complexity of this patient population. As most literature coalesced around experiences of specific treatments, greater examination of the overall experience of TRD is needed to inform clinical practice, and administrative and funding policy decisions for interventions that may be effective for this patient population. PROSPERO registration number: CRD42022356813.

A Systematic Literature Review of Modelling Approaches to Evaluate the Cost Effectiveness of PET/CT for Therapy Response Monitoring in Oncology.

This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature. This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications. The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias. Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addressed appropriately. Further research should include more advanced and patient-level modelling approaches to accurately represent the complex context of clinical practice and, therefore, to be meaningful to support decision making. This review is registered in PROSPERO, the international prospective register of systematic reviews funded by the National Institute for Health Research, with CRD42023402581.

Evaluating progress towards implementation of the European HTA Regulation: Insights generated from the European Access Academy's multi-stakeholder survey

ObjectivesWe conducted a multi-stakeholder survey to assess stakeholders' perceptions of the progress made towards the implementation of the European Regulation on Health Technology Assessment (EU HTA R) and to identify and prioritize the remaining challenges for implementation. MethodsUsing two iterative Delphi cycles, an exploratory, semi-quantitative survey was developed to identify how stakeholders 1) experienced preparatory activities of the Regulation, and 2) prioritized remaining challenges for successful implementation. The survey was distributed among the network of the European Access Academy and via social media to ensure coverage of key stakeholders. Descriptive analyses were performed on quantitative response items, and relative importance was calculated for ranking items. Free-text responses supplemented participants' answers to quantitative questions. ResultsN = 61 responses were received from N = 15 countries including Global/ EU-wide institutions (Patients and Patients’ representatives: 7; Clinicians’ representatives: 5; Regulators: 3; Health Technology Developers (HTDs): 20; HTA bodies: 10; Payers: 7; Policy Makers: 2; Academic representatives: 7). The majority of respondents were aware of preparatory activities, with 74 % (N = 45) observing the drafting of Guidance Documents and 64 % (N = 39) noting the establishment of the Coordination Group. Respondents ranked the success of preparatory activities neutral with a slight tendency towards a positive ranking. Key challenges were Member States' readiness for Joint Clinical Assessments, HTA capacity/ capability constraints, and the applicability/ feasibility of the methodological framework. ConclusionsThis study identified the key remaining challenges for the successful establishment of the EU HTA process. Key findings emphasize the readiness of national systems and procedures as pivotal factors. Balancing operational efficiency with strategic objectives, including the development of a European Value Framework, is imperative for harnessing the full potential of the joint HTA process and enhancing patient access to innovative technologies on a pan-European scale. Public interest summaryIn January 2022, the European Union (EU) adopted the European Regulation on Health Technology Assessment (HTA). By harmonizing HTA practices and promoting collaboration across Member States, the joint procedure as set out in the regulation aims to improve efficient use of resources and ensure long-term sustainability of EU-wide HTA cooperation. Close to reaching the half-way mark of the preparation phase in mid-2023, we conducted a multi-stakeholder survey to assess perceptions of the progress made towards implementation of a joint procedure and to prioritize remaining challenges. The responses indicated a neutral to positive perception of the preparatory activities (i.e., establishing a coordination group and a stakeholder network, and creating guidance documents). Respondents prioritized challenges related to the readiness of Member States, limitations in HTA capacity and capability, and applicability of the assessment methods. Further research is needed to define targeted strategies for specific stakeholder groups to effectively address the identified challenges.

Perceptions of indirect treatment comparisons as an evidence base in oncology decision-making: results of an international survey of health technology assessment and payer decision-makers.

Aim: Health technology assessment (HTA) and payer organizations are often faced with early decision-making in oncology. To design and conduct robust indirect treatment comparisons (ITCs), it is important to better understand HTA and payer decision-maker perceptions of ITCs. Here we aim to describe what individuals with HTA and payer experience see as the acceptability of ITCs for HTA and payer organization coverage and reimbursement decision-making. Materials & methods: This survey included 30 current and former HTA and payer decision-makers from five countries: Australia, France, Germany, the UK (n=5 each) and the US(n=10). Main outcomes included the ratings of acceptance of ITCs and the presence of well-defined methodological guidance for ITCs. Results: ITCs are generally accepted by participants in Australia and the UK but are more likely evaluated on a case-by-case basis in France, Germany and the US. Four of five participants in Germany and the UK, two of five in Australia and one of five in France reported that well-defined and prescribed criteria regarding the use of ITCs were in place. Conclusion: There is a need for harmonization of methods used to assess ITCs by HTA and payers, especially in the rapidly evolving treatment landscape in oncology.

Generative AI for Health Technology Assessment: Opportunities, Challenges, and Policy Considerations - an ISPOR Working Group Report

ObjectiveTo provide an introduction to the uses of generative Artificial Intelligence (AI) and foundation models, including large language models (LLMs), in the field of health technology assessment (HTA). MethodsWe reviewed applications of generative AI in three areas: systematic literature reviews, real world evidence (RWE) and health economic modeling. Results(1) Literature reviews: generative AI has the potential to assist in automating aspects of systematic literature reviews by proposing search terms, screening abstracts, extracting data and generating code for meta-analyses; (2) Real World Evidence (RWE): generative AI can facilitate automating processes and analyze large collections of real-world data (RWD) including unstructured clinical notes and imaging; (3) Health economic modeling: generative AI can aid in the development of health economic models, from conceptualization to validation. Limitations in the use of foundation models and LLMs include challenges surrounding their scientific rigor and reliability, the potential for bias, implications for equity, as well as nontrivial concerns regarding adherence to regulatory and ethical standards, particularly in terms of data privacy and security. Additionally, we survey the current policy landscape and provide suggestions for HTA agencies on responsibly integrating generative AI into their workflows, emphasizing the importance of human oversight and the fast-evolving nature of these tools. ConclusionsWhile generative AI technology holds promise with respect to HTA applications, it is still undergoing rapid developments and improvements. Continued careful evaluation of their applications to HTA is required. Both developers and users of research incorporating these tools, should familiarize themselves with their current capabilities and limitations.

Managing experts’ conflicts of interest in the EU Joint Clinical Assessment

ObjectiveThis article critically evaluates the European Commission’s 2024 Implementing Regulation (IR) on conflicts of interest (COIs) management for stakeholders in the European Union (EU) Joint Clinical Assessment (JCA), with a focus on individual experts such as clinicians and patient representatives.Key findingsThe IR is the first EU-level framework to assess COIs in the context of health technology assessment (HTA). The regulation requires experts involved in the JCA to submit annual declarations of interest for both financial and non-financial interests and presents a matrix on whether these conflicts should disqualify them from participating in the joint work. We compared the IR to COIs-management approaches from other European national HTA bodies and found that the IR is closely modelled after the French guidelines. Concerns include potential over-representation of experts from a small number of countries, lack of guidance on organisational COIs, and ambiguities in how the size of financial interests are disclosed. Unclear resource allocation for enforcement could also hinder compliance.ConclusionsThe IR marks progress in EU-wide HTA collaboration, but improvements in transparency, expert diversity, and comprehensive COIs management are needed to ensure impartiality in the JCA process.

Economic effects of priority setting in healthcare: a scoping review of current evidence

ObjectivesStudy objective was to map the current literature on the economic effects of priority setting at the system level in healthcare.DesignThe study was conducted as a scoping review.Data sourcesScopus electronic database was searched in June 2023.Eligibility criteriaWe included peer-reviewed articles published 1 January 2020–1 January 2023. All study designs that contained empirical evidence on the financial effects or opportunity costs of healthcare priority setting were included excluding disease, condition, treatment, or patient group-specific studies.Data extraction and synthesisTwo independent researchers screened the articles, and two additional researchers reviewed the full texts and extracted data. We used Joanna Briggs Institute checklists to assess the quality of qualitative, quasi-experimental and economic evaluations and the mixed methods appraisal tool for the mixed method studies. Synthesis was done qualitatively and through descriptive statistics.Results8869 articles were screened and 15 fulfilled the inclusion criteria. The most common study focus was health technology assessment (7/15). Other contexts were opportunity costs, effects of programme budgeting and marginal analysis, and disinvestment initiatives. Priority setting activities analysed in the studies did not achieve cost savings or cost containment (4/15) or have mixed findings at best (8/15). Only five studies found some indication of cost savings, cost containment or increased efficiency. Also, many of the studies consider costs only indirectly or qualitatively.ConclusionsAll in all, there is very little research addressing the pressing question of whether explicit priority setting and priority-setting methods can support cost containment on a health service system level (regional or national). There is limited evidence of the economic effects of priority setting.

Health Technology Assessment of Nailfold Capillaroscopy and Digital Retina Imaging in Diabetes Screening

AimThis study aims to identify the criteria for the evaluation of medical technologies in diabetes screening and then perform health technology assessment (HTA) for two technologies: digital retina imaging (DRI) and Nailfold Capillaroscopy (NFC). MethodsA multicriteria decision-making tool measuring attractiveness using a categorical-based evaluation technique (MACBETH) was used to calculate and compare alternatives. A focus group of ten decision-makers with more than ten years of experience in health technology was used for the decision-making. The Delphi Method was used to get a consensus about comparing criteria and alternatives to these criteria. The sampling method used in the study was the nonprobability purposive sampling method. ResultsThe study concluded that clinical efficacy (0.42) is the most important criterion for evaluating medical technologies, followed by cost-effectiveness (0.27). At the same time, ethical considerations were found to be less important (0.03). The sensitivity analysis concluded that the decision is sensitive to clinical efficacy and cost-effectiveness changes. The study found that none of the alternatives dominates others as an option for early detection of diabetes through vascular changes. LimitationsThis study primarily uses systemic literature review methods to identify criteria for evaluating alternatives. There may be additional criteria for evaluation. ConclusionThis study provides an approach for a multicriteria comparison of medical devices. The study findings are useful for public health professionals and health policymakers. Plain Language SummaryThis study assesses nailfold capillaroscopy (NFC) as a substitute for digital retina imaging (DRI) in diabetes screening using the MACBETH multicriteria decision-making tool. The research identifies five key evaluation criteria: clinical efficacy, cost-effectiveness, patient needs, user safety, and ethical considerations. A focus group of experienced health technology decision-makers was used to compare NFC and DRI. Results indicate that clinical efficacy and cost-effectiveness are the most critical factors, with NFC showing potential but not significantly outperforming DRI. Sensitivity analyses highlight the need for further research to validate NFC as an effective diabetes screening tool.

Early high-dose cryoprecipitate to reduce mortality in adult patients with traumatic haemorrhage: the CRYOSTAT-2 RCT with cost-effectiveness analysis.

Traumatic haemorrhage is common after severe injury, leading to disability and death. Cryoprecipitate, a source of fibrinogen, may improve outcomes for patients with traumatic haemorrhage. To investigate the effects of early fibrinogen supplementation in the form of 3 pools (15 units, approximately 6g of fibrinogen) of cryoprecipitate on 28-day mortality. A randomised, parallel-group, unblinded, multicentre, international trial and economic evaluation. Patients were randomised to either the intervention (early cryoprecipitate) or the comparator (standard major haemorrhage protocol) arm via opaque, sealed envelopes in the emergency department or the transfusion laboratory/blood bank. All analyses were performed on an intention-to-treat basis. A cost-effectiveness analysis was undertaken. Twenty-five major trauma centres in the UK and one level 1 trauma centre in the USA. Adults who had traumatic haemorrhage following severe injury requiring activation of the major haemorrhage protocol and had received a blood transfusion. Early cryoprecipitate - 3 pools (equivalent to 15 single units of cryoprecipitate or 6 g of fibrinogen supplementation), infused as rapidly as possible, within 90 minutes of arrival at hospital in addition to standard major haemorrhage protocol or standard major haemorrhage protocol only. The primary outcome was all-cause mortality at 28 days. The secondary outcomes were all-cause mortality at 6 hours, 24 hours, 6 months and 12 months from admission; death from bleeding at 6 hours and 24 hours; transfusion requirements at 24 hours from admission; destination of participant at discharge; quality-of-life measurements (EuroQol-5 Dimensions, five-level version and Glasgow Outcome Scale) at discharge/day 28 and 6 months after injury; and hospital resource use up to discharge or day 28 (including ventilator-days, hours spent in critical care and inpatient stays). Eight hundred and five patients were randomised to receive the standard major haemorrhage protocol (control arm). Seven hundred and ninety-nine patients were randomised to receive an additional three pools of cryoprecipitate in addition to standard care (intervention arm). Baseline characteristics appeared well matched. Patients had a median age of 39 (interquartile range 26-55) years, and the majority (79%) were male. All-cause 28-day mortality (n=1531 patients; intention to treat) was 25.3% in the intervention arm compared with 26.1% in the control arm (odds ratio 0.96; p=0.74). There was variability in the timing of cryoprecipitate administration, with overlap between the treatment arms, limiting the degree of intervention separation. There was no evidence that early empiric administration of high-dose cryoprecipitate reduced the risk of death in unselected patients with traumatic haemorrhage. There was also no difference in adverse events. The cost-effectiveness of the intervention was similar to that of standard care. Research to evaluate if fibrinogen replacement is more beneficial for selected patients, for example those with low fibrinogen blood levels, is needed, as is further exploration of whether there is a difference in outcome according to mechanism of injury. This trial is registered as ISRCTN14998314. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 15/57/02) and is published in full in Health Technology Assessment; Vol. 28, No. 76. See the NIHR Funding and Awards website for further award information.