Volume 14•Issue 11

In this update, we explore the vocal adoption of the target trial emulation framework by the US FDA, the FRAME methodology for systematically evaluating the use and impact of RWE in health technology assessment and regulatory submissions, and the ...

In this update, we examine the US’ Food and Drug Administration (FDA) Commissioner’s National Priority Voucher pilot program; The Netherlands’ Zorginstituut Nederland (ZIN) implementation framework for the EU HTA Regulation; and Italy’s Agenzia Italiana ...

Randomized controlled trials (RCTs) are regarded as the highest level of evidence in medical research, but RCTs also have their drawbacks. Over the years, several alternative study designs have been introduced to address these problems. However, many of ...

Aim: The eligibility criteria of randomized controlled trials can exclude groups of patients indicated for the therapy post-approval and the trial population may not be generalizable to routine care. We attempted to emulate the MONALEESA-2 trial of ...

Aim: Current Parkinson’s disease (PD) research evaluates patients with early and severe PD, but often overlooks patients with intermediate PD. This study aims to quantify unmet treatment needs, disease burden and healthcare resource utilization among ...

Aim: Clinical trial data have demonstrated that direct oral anticoagulants (DOACs) are both noninferior to and safer than conventional therapy for the treatment of venous thromboembolism (VTE). This study aimed to compare the effectiveness and safety of ...

Aim: Sinonasal inverted papilloma is a benign yet locally aggressive tumor with potential for recurrence. Surgical resection is the primary treatment, and both endoscopic sinus surgery (ESS) and external approaches are employed depending on tumor ...

Aim: While only recently approved for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), many patients with MASH have taken glucagon-like peptide-1 receptor agonists (GLP-1 RAs) for the treatment of comorbid Type 2 diabetes (T2D) ...

Aim: Bruton’s tyrosine kinase inhibitors (BTKis), including ibrutinib and acalabrutinib, transformed the treatment landscape of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) by improving outcomes compared with ...

Aim: Continuous glucose monitoring (CGM) supports glycemic control and reduces diabetes complications. CGM systems include intermittently scanned CGM (is-CGM) and real-time CGM (rt-CGM). While rt-CGM may provide better outcomes than is-CGM, it costs more ...

Aim: Ischemic stroke continues to be a significant contributor to mortality and disability on a global scale. The blood neurofilament light chain (bNfL) as a prognostic indicator for stroke functional outcomes is a topic of ongoing debate. Thus, the ...

Analysis of single arm trials complemented with external comparator arms (ECAs) may be used to support evidence of effectiveness of novel therapies in oncology research when a randomized trial is unavailable or unfeasible. However, the intention-to-treat ...