Volume 14•Issue 10

In this update, we examine Spain’s comprehensive pharmaceutical legislation reform; France’s refined health economic evaluation approach; and the US’ proposed Most Favored Nation pricing mechanism.

Composite endpoints amalgamate multiple clinical outcomes into a single measure, offering efficiency gains in clinical trials through increased event rates and reduced sample sizes, thus accelerating clinical development and regulatory approval. However, ...

The first paper of this two-part series critically examined the role of composite endpoints in health technology assessments (HTAs) and outlined strategies for determining whether to employ the composite estimate of treatment effect or disaggregate into ...

Aim: Stent-assisted endovascular coiling is a safe and effective treatment for unruptured intracranial aneurysms (UIAs). This study compared 180-day inpatient readmission and cost among patients with UIA who underwent stent-assisted coiling (SAC) using ...

Aim: Surgical site infections pose a significant challenge in postoperative care, traditionally managed by keeping wounds dry. However, recent studies indicate that early postoperative showering may not increase infection risk. This study aimed to assess ...

Aim: To report the efficacy and safety of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the phase III, 72-week, placebo-controlled period of Study 041. Materials & methods: Inclusion criteria: boys with nmDMD aged ≥...

Aim: This study compares the efficacy of diroximel fumarate (DRF) with ocrelizumab (OCR) and interferon beta-1a (IFNβ-1a) for treating relapsing multiple sclerosis (MS) through matching-adjusted indirect comparisons (MAICs). Materials & methods: We used ...

Aim: We aimed to identify variables that affect the prognosis and treatment effect in multiple myeloma (MM). Materials & methods: Published literature of randomized controlled trials (RCTs) and population-adjusted indirect comparisons (PAICs) in newly ...

Aim: Ribociclib + nonsteroidal aromatase inhibitor (NSAI) and abemaciclib + endocrine therapy (ET) are approved for high-risk hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer based on data from ...

Aim: Persistently active noninfectious keratitis can lead to permanent corneal damage and loss of vision. Given the limited treatment options, Acthar Gel, a US FDA-approved treatment for ocular inflammatory conditions, can be a potential therapy. This ...

Real-world evidence (RWE) from outside Canada or the UK is sometimes included in submissions to health technology assessments by Canada’s Drug Agency/L'Agence des médicaments du Canada (CDA-AMC) and National Institute for Health and Care Excellence when ...