The assessment of technology in hospital settings is a crucial step towards ensuring the delivery of efficient, effective, and safe healthcare.

This study conducts a Hospital-Based Health Technology Assessment to evaluate the efficacy of a screening rapid test for mild Traumatic Brain Injury (mild TBI) utilizing blood biomarkers, specifically Glial Fibrillary Acidic Protein (GFAP) and Ubiquitin C-terminal Hydrolase L1 (UCH-L1). The assessment focuses on the clinical utility and performance characteristics of the proposed rapid test within a hospital setting.

The screening model was meticulously examined for its ability to accurately detect mild TBI, considering the sensitivity and specificity of GFAP and UCH-L1 as blood biomarkers. The study involved a thorough evaluation of the test’s diagnostic accuracy, comparing its outcomes with established standards for mild TBI diagnosis.

Results from the Hospital-Based Health Technology Assessment highlight the potential of the GFAP and UCH-L1 blood biomarker-based rapid test as an efficient screening tool for mild TBI within a hospital environment. The evidence results show that the test is highly sensitive (91 percent to 100 percent) for the prediction of acute traumatic intracranial lesions, which helps rule out injury when the result is negative. When used within 12 hours of injury in adult patients with mild TBI, this test holds promise in reducing the utilization of CT.

The findings contribute valuable insights into the feasibility and reliability of implementing this technology for timely and accurate identification of mild TBI, enhancing clinical decision making and patient care in hospital settings.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.

A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).

Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were “relevance of outcomes for a rare disease,” “impact on patient’s quality of life,” “price negotiation,” and “adjusted cost-effectiveness threshold.” On the other hand, “budget impact threshold,” “innovative nature of treatment,” and “willingness to accept greater uncertainty in clinical evidence” received negative evaluations and were excluded from the final framework.

A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.

Left ventricular assist devices (LVAD) are a therapeutic option in patients with advanced heart failure (HF) not a candidate for cardiac transplant as destination therapy (DT). However, important uncertainties remain regarding the use of LVAD in the long-term in real practice settings. When planning registries, it is important to choose the appropriate outcomes that ensure comparability and reduce the possibility of bias.

The purpose of this study was to establish a minimum dataset (MDS) that should be collected in all LVAD for DT registries to meet the needs and demands of Health Technology Assessment (HTA) doers and health professionals.

To design the MDS for LVAD, a preliminary list of outcome domains and data items were developed attending to the gaps and research needs derived from existing evidence coming from HTA carried out at the European Network of Health Technology Assessment (EUnetHTA) level. The list of data items and domains was agreed upon by all involved HTA organizations and a three-round Delphi was conducted among an experienced panel of cardiologists to rate the importance of the items for measuring uncertainty gaps.

After the three-round Delphi process, the expert panel reached a consensus regarding eighteen outcomes and forty-seven variables divided into seven main domains (safety, effectiveness, patient acceptability, satisfaction, healthcare system impact, pharmaceutical management, and technique-related factors).

The MDS of outcomes and measures, developed based on research gaps and needs, can allow for standardizing data collection and improving the quality of data for decision making and practice.

The EUnetHTA Core Model® is well-established in the HTA community. Some recommendations of corresponding guidance documents leave room for alternative methodological choices. Considering the new HTA regulation (HTAR), we aimed to identify needs for concretization (NCs) in EUnetHTA guidance and provide indicative methodological options.

We carried out a qualitative document analysis and structured group discussion. Twenty-two EUnetHTA documents were screened using transparent criteria. Identified NCs were classified into topics according to the PRISMA statement and presented to Austrian HTA practitioners (n = 11) during a structured group discussion. Participants rated NC’s importance. To identify potential solutions, selected key handbooks for generic (Cochrane) and HTA-specific (IQWIG/NICE) evidence synthesis were systematically reviewed and matching content was charted against the NCs.

Thirty-two topics with varying numbers of NCs were identified, twenty-six during the screening process, and six from the group discussion. Most of the topics related to evidence synthesis methods (nine topics), evidence eligibility criteria (nine topics), risk of bias (three topics), and certainty assessment (three topics). Other topics related to information sources, search strategy, data collection process, data items, effect measures, and reporting bias. One or more methodological approaches and recommendations could be identified for each identified topic from the included methodological handbooks.

Our analysis identified a need for concretization in some EUnetHTA guidelines. The structured overview of methodological options may support HTA doers in adapting and applying the guidelines to the national and local practical context.

Following acquired brain injury (ABI), individuals often experience anxiety and/or depressive symptoms. BrainACT is an adapted form of Acceptance and Commitment Therapy (ACT) tailored to this target group. The current study is a trial-based health-economic evaluation comparing BrainACT to a psychoeducation and relaxation control treatment.

An economic evaluation from a societal perspective was conducted in the Netherlands alongside a multicenter randomized controlled two-armed parallel trial including 72 participants. A cost-utility and cost-effectiveness analysis was conducted where incremental costs, quality-adjusted life-years (QALYs), and anxiety/depression (Hospital Anxiety and Depression Scale (HADS) score) were collected and presented over a 1-year follow-up period. Bootstrapping, scenario, and subgroup analyses were performed to test the robustness of the results.

The BrainACT arm reported non-significant lower total costs (incremental difference of €−4,881; bootstrap interval €−12,139 to €2,330) combined with significantly decreased anxiety/depression (HADS) (3.2; bootstrap intervals 0.7–5.7). However, the total QALYs were non-significantly lower (−0.008; bootstrap interval −0.060 to 0.042) for BrainACT. The probability of the intervention being cost-effective was 86 percent at a willingness-to-accept threshold of €50,000/QALY. The scenario and subgroup analyses confirmed the robustness of the results.

BrainACT may be a more cost-effective alternative to a psychoeducation and relaxation intervention for anxiety and/or depressive symptoms following ABI. Despite limitations, BrainACT appears to be a promising addition to treatment options in the Netherlands. Further research is needed to validate these findings, and consideration should be given to implementing BrainACT in Dutch clinical settings with ongoing monitoring.

Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in the use of surrogate endpoints has been accompanied by literature describing the frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales.

This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.

Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.

This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals, which can lead to uncertainty in decision making.