Volume 15•Issue 1

In this update, we explore a review on transportability methods to enable the use of cross-jurisdictional evidence when local data are limited, a review of clinical trials that use pragmatic elements and finally, we discuss a study highlighting the ...

What is this summary about? This plain language summary is based on an article about the APHENITY trial that was published in The Lancet journal in October 2024. The APHENITY trial was a phase 3 clinical trial to find out whether sepiapterin helped ...

Aim: Fractional Polynomial (FP) models are widely used in survival analysis for health technology assessment and network meta-analysis (NMA). However, current implementations rely on a fixed set of pre-specified powers, which may constrain model ...

Aim: This study assessed the acceptability of external control-arm (ECA) use in nononcology health technology assessment (HTA) submissions in Europe. Materials & methods: We conducted a sequential mixed method study to investigate the study objective. ...

Aim: To assess patient characteristics, burden of disease, healthcare resource utilization and costs of acute myeloid leukemia (AML) by treatment intensity in German claims data. Materials & methods: In this retrospective cohort study using claims data ...

Aim: Lorlatinib demonstrated superior efficacy over alectinib as a first-line treatment for ALK-positive (ALK+) advanced/metastatic non-small cell lung cancer in a matching-adjusted indirect comparison (MAIC) using 3-year follow-up data from CROWN (...

Aim: The aim of the study is to understand the impact of lymph node dissection (LND) on overall survival (OS) and cancer-specific survival (CSS) in elderly patients with early-stage non-small cell lung cancer (NSCLC), and to find the best characteristics ...

Aim: The use of amyloid-beta (Aβ) clearance to support regulatory approvals of drugs in Alzheimer’s disease (AD) remains controversial. We evaluate Aβ as a potential trial-level surrogate endpoint for clinical function in AD. Materials & methods: Data on ...

Aim: Stroke is a clinically defined syndrome characterized by an acute, focal neurological deficit due to vascular injury. Strokes can result in various disabilities, including sensory and motor abnormalities, leading to difficulties with motor control, ...

Background: The impact of significant tricuspid regurgitation (sTR) on healthcare costs and utilization in real-world populations remains understudied. Aim: Describe healthcare costs and utilization before and after development of sTR and describe ...

Background: The impact of race and ethnicity on prognosis and clinical outcomes in patients with significant tricuspid regurgitation (sTR) is not well understood. Aim: Describe healthcare utilization trends preceding the development of sTR and assess ...

Aim: Two fixed-dose combination triple therapies, budesonide/glycopyrronium/formoterol fumarate dihydrate (BGF) 320/14.4/10 μg and fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) 100/62.5/25 μg, have been shown to reduce all-cause mortality in ...

Aim: Anticholinergic burden (ACB) is associated with profound clinical and economic burden; however, anticholinergic medications are often prescribed for overactive bladder (OAB). This analysis assessed risk of adverse health outcomes and costs ...

Aim: Polycythemia vera (PV), a rare, chronic myeloproliferative neoplasm, that negatively impacts patient outcomes, and optimal therapy remains unclear due to a lack of head-to-head trials. A targeted literature review and feasibility assessment for an ...