Rare diseases represent a highly heterogeneous group of disorders with high phenotypic
and genotypic diversity within individual conditions. Due to the small numbers of people …

Background A systematic review and meta-analysis was conducted to assess breast cancer
(BC) outcomes among patients with early-stage hormone receptor positive (HR+), human …

Background There is a growing trend to include non-randomised studies of interventions
(NRSIs) in rare events meta-analyses of randomised controlled trials (RCTs) to complement …

Abstract Real-world data (RWD), such as electronic health records, reimbursement requests
as adjudicated by health insurance companies, and health survey data as collected by …

Background: Many rare events meta-analyses of randomized controlled trials (RCTs) have
lower statistical power, and real-world evidence (RWE) is becoming widely recognized as a …

Objective An important consideration when combining RCTs and NRSIs is how to address
their potential biases in the pooled estimates. This study aimed to propose a Bayesian bias …

Background When designing studies it is common to search the literature to investigate
variability estimates to use in sample size calculations. Proprietary data of previously …

Abstract Effect modification (EM) may cause bias in network meta‐analysis (NMA). Existing
population adjustment NMA methods use individual patient data to adjust for EM but …

Objective Traditionally, validation of surrogate endpoints has been carried out using
randomized controlled trial (RCT) data. However, RCT data may be too limited to validate …

In network meta‐analysis (NMA), we synthesize all relevant evidence about health
outcomes with competing treatments. The evidence may come from randomized clinical …