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
Category: Clinical trials
Can Real-World Data Redefine Clinical Trial Protocol Design?
A System Built Backwards? Clinical trials are the gold standard for evaluating new treatments, but they are often built on idealized assumptions that poorly reflect the complexity of real-world clinical care. Protocols are crafted around rigid eligibility criteria, theoretical endpoints, and often over-optimized designs that filter out the majority of patients seen in actual clinical… Continue reading Can Real-World Data Redefine Clinical Trial Protocol Design?
Label Expansion and the Role of RWD
In the lifecycle of a pharmaceutical product, initial regulatory approval is often just the beginning. A newly authorized drug typically enters the market with a carefully defined label specifying the disease, population, dose, and setting for its use based on data from randomized controlled trials (RCTs). But often label specifications evolve. Real world clinical needs… Continue reading Label Expansion and the Role of RWD
Bridging the Gap: Key Data Elements in Oncology RCTs vs. RWD
Conducting Real-World Evidence (RWE) research in oncology requires access to diverse data sources, such as patient registries, electronic health records (EHR), claims data, and cancer-specific databases. These data sources often vary in completeness and quality limiting their suitability. Additionally, the rapid advancement of cancer therapies, including immunotherapies, targeted therapies, and combination treatments, complicates the ability… Continue reading Bridging the Gap: Key Data Elements in Oncology RCTs vs. RWD
Digital Biomarkers: Transforming Healthcare Through Continuous and Real-World Monitoring
Digital biomarkers are revolutionizing the way healthcare professionals and researchers monitor, diagnose, and treat diseases. Enabled by digital technologies, these biomarkers provide objective, quantifiable physiological and behavioral data collected through connected devices such as smartphones, wearables, and sensors. Unlike traditional biomarkers, which rely on laboratory tests or imaging, digital biomarkers enable continuous, real-world monitoring, offering… Continue reading Digital Biomarkers: Transforming Healthcare Through Continuous and Real-World Monitoring