Leveraging external data in the design and analysis of clinical trials in neuro-oncology

R Rahman, S Ventz, J McDunn, B Louv… - The Lancet …, 2021 - thelancet.com
Integration of external control data, with patient-level information, in clinical trials has the
potential to accelerate the development of new treatments in neuro-oncology by …

[HTML][HTML] World on data perspective

MKM Nasution - World, 2022 - mdpi.com
It is not simple to consider the world from only one side, but analyzing all sides can cloud
comprehension without reaching deep insight found at the core. In a word as a whole, there …

For insights into the real world, consider real-world data

S Raoof, R Kurzrock - Science Translational Medicine, 2022 - science.org
Randomized control trials (RCTs) are required before drug and device approvals and have
contributed to patient safety, but they have also increased the cost and time of regulatory …

Do SGLT2 inhibitors and GLP-1 receptor agonists modulate differently the risk of stroke? Discordance between randomised controlled trials and observational studies

AJ Scheen - Diabetes & Metabolism, 2023 - Elsevier
Stroke represents a major burden in patients with type 2 diabetes, yet this cerebrovascular
complication has been less carefully investigated than the risk of cardiovascular mortality …

Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real‐life experience from a national reference network

H Mosbah, MC Vantyghem, E Nobécourt… - Diabetes, Obesity …, 2022 - Wiley Online Library
Aim To describe baseline characteristics and follow‐up data in patients with lipodystrophy
syndromes treated with metreleptin in a national reference network, in a real‐life setting …

[HTML][HTML] Comparative effectiveness and safety of pharmaceuticals assessed in observational studies compared with randomized controlled trials

YD Hong, JP Jansen, J Guerino, ML Berger, W Crown… - BMC medicine, 2021 - Springer
Background There have been ongoing efforts to understand when and how data from
observational studies can be applied to clinical and regulatory decision making. The …

[HTML][HTML] Using public clinical trial reports to probe non-experimental causal inference methods

E Steinberg, N Ignatiadis, S Yadlowsky, Y Xu… - BMC Medical Research …, 2023 - Springer
Background Non-experimental studies (also known as observational studies) are valuable
for estimating the effects of various medical interventions, but are notoriously difficult to …

Comparing Survival Outcomes between Hemodialysis and Hemodiafiltration Using Real-World Data from Brazil

EP da Rocha, CA Kojima… - Journal of Clinical …, 2024 - mdpi.com
The CONVINCE trial demonstrates that high-dose hemodiafiltration offers a survival
advantage for patients in the high-flux hemodiafiltration group compared to hemodialysis …

[HTML][HTML] Generating real-world evidence compatible with evidence from randomized controlled trials: a novel observational study design applicable to surgical …

X Yu, Z Wang, L Wang, Y Huang, Y Wang, S Xin… - BMC Medical Research …, 2022 - Springer
Background Numerous observational studies have revealed an increased risk of death and
complications with transfusion, but this observation has not been confirmed in randomized …

How real can we get in generating real world evidence? Exploring the opportunities of routinely collected administrative data for evaluation of medical devices

B Pongiglione, A Torbica - Health Economics, 2022 - Wiley Online Library
Real‐world data are considered a potentially valuable source of evidence for assessing
medical technologies in clinical practice, but their widespread use is hampered by …