The approval of eflornithine (brand name Iwilfin) for pediatric high-risk neuroblastoma offers an interesting example of how the FDA evaluates single-arm trials supported by external control arms (ECAs). External controls are increasingly common in oncology and rare disease drug development, but they remain a source of careful regulatory scrutiny. In this case, the FDA’s assessment… Continue reading Regulatory Insights from FDA Review of the External Control Arm in a Pediatric Rare Disease Trial
Author: Nadia Barozzi
Passionate about data-driven insights and the advancement of Real World Evidence research, drug safety and pharmacovigilance.
The Expanding Role of Epidemiology in Modern Real-World Data Research
When observational retrospective research began to be used more systematically more than twenty years ago, the real-world data (RWD) landscape was relatively simpler. Not necessarily easier, but simpler. A limited number of large administrative and clinical databases dominated the field. Claims databases, national registries and a handful of electronic medical record (EMR) sources were directing… Continue reading The Expanding Role of Epidemiology in Modern Real-World Data Research
Digital Biomarkers vs. Digital Phenotypes: Signals, Context, and Clinical Insight
The definitions digital biomarker and digital phenotype are part of the everyday vocabulary of digital health. They refer to two methodological approaches, regulatory pathways, and levels of clinical inference that are gaining attention in Real-World Data (RWD) research. As digital signals move closer to informing clinical decision-making, it is essential to clearly understand what these… Continue reading Digital Biomarkers vs. Digital Phenotypes: Signals, Context, and Clinical Insight
The Hidden Architecture of RWE: When Complete Data Stop Being True Data
Real-world evidence (RWE) is built on the premise that data generated during routine clinical care can reveal how medicines and interventions actually perform outside the environment of controlled trials. Electronic health records (EHRs) and disease registries are often treated as the foundational infrastructure of this approach. To improve data quality, many systems increasingly rely on… Continue reading The Hidden Architecture of RWE: When Complete Data Stop Being True Data
The RWE Paradox: Plenty of Data, Fragile Structure
For more than a decade, we have framed real-world evidence (RWE) challenges as a data problem. Not enough access. Not enough scale. Not enough linkage. Not enough quality. However, this narrative has become increasingly difficult to sustain. Across major markets, data availability has expanded, platforms have matured, and analytical methods have become more standardized and… Continue reading The RWE Paradox: Plenty of Data, Fragile Structure