Volume 15•Issue 4

In this update we cover the US–UK Economic Prosperity Deal announced in December 2025, which includes NICE's first major cost-effectiveness threshold increase in over two decades. We also analyze the latest developments in US pharmaceutical pricing ...

What is this summary about? This article describes results from Study 041. Study 041 was a clinical study of ataluren, a treatment for people living with nonsense mutation Duchenne muscular dystrophy (nmDMD for short). Over time, people living with ...

Randomized controlled trials (RCTs) are the gold standard generating evidence owing to their rigorous methodology. However, their logistical, financial and ethical limitations highlight the need for alternative approaches using real-world data. Target ...

Aim: Mendelian randomization (MR) is an instrumental variable (IV) method that utilizes genetic variants to establish causality between risk factors and outcomes in observational studies. These methods were primarily developed under assumptions ...

Aim: Diabetic macular edema (DME) is one of the leading causes of vision impairment in diabetic patients. The aim of this study was to evaluate the cost-effectiveness of faricimab (Vabysmo^®), the first bispecific antibody targeting VEGF-A and ANG-2, for ...

Aim: Psoriasis severely affects quality of life, particularly in patients with moderate to severe psoriasis. This study aims to estimate health utility by Psoriasis Area and Severity Index (PASI) response after biologic induction therapy in Chinese ...

Background: Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce. Aim: To describe and compare patients' characteristics, treatment patterns, healthcare resource ...

Aim: New IDSA guidelines define uncomplicated UTI (uUTI) as infection limited to the bladder in both men and women. This study compared the effectiveness of β-lactams, nitrofurantoin and trimethoprim/sulfamethoxazole (TMP/SMX) to fluoroquinolones for ...

Aim: In oncology trials, blinded independent central review (BICR) is the standard for treatment response classification. Real-world data methodologies that align with BICR may reduce misclassification in real-world evidence (RWE) studies and enhance ...

Aim: To evaluate the performance of four stent types (BMS, bare metal stents; Eluvia, a polymer-based paclitaxel-eluting stent; Viabahn, a covered stent; and Zilver PTX, a polymer-free paclitaxel-coated stent) in femoropopliteal lesions at 12 and 24 ...

This study conducted an indirect treatment comparison (ITC) of iptacopan, the first oral monotherapy targeting factor B, versus pegcetacoplan, a subcutaneous infusion targeting complement component 3, in patients with paroxysmal nocturnal hemoglobinuria (...

Aim: Community-acquired bacterial pneumonia (CABP) remains a major cause of morbidity and mortality worldwide, particularly among elderly and susceptible populations. Escalating antimicrobial resistance among prevalent CABP pathogens in China, combined ...

More interventions that better manage cardiovascular disease are urgently needed in the Middle East. To discuss this issue, we held a symposium at the Heart Masters Middle East 2023 congress (Dubai, UAE; May 2023) on personalized management of angina and ...