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 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.

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.