Real‐world experience in treating pediatric relapsed/refractory or therapy‐related myeloid malignancies with decitabine, vorinostat, and FLAG therapy based on a …

ES Schafer, K Chao, AM Stevens, E Jo… - Pediatric Blood & …, 2022 - Wiley Online Library
Current therapies for relapsed/refractory (R/R) pediatric myeloid neoplasms are
inadequately effective. Real‐world data (RWD) can improve care by augmenting traditional …

[HTML][HTML] Survival outcomes of patients with metastatic non-small cell lung cancer receiving chemotherapy or immunotherapy as first-line in a real-life setting

Y Belaroussi, F Bouteiller, C Bellera, D Pasquier… - Scientific Reports, 2023 - nature.com
Abstract Treatment of metastatic non-small cell lung cancer (mNSCLC) has been modified
due to the development of immunotherapy. We assessed survival outcomes (overall [OS] …

The challenges and opportunities in using real-world data to drive advances in healthcare in East Asia: expert panel recommendations

G Crane, JCW Lim, CS Gau, J Xie… - Current Medical Research …, 2022 - Taylor & Francis
Objective To provide recommendations for overcoming the challenges associated with the
generation and use of real-world evidence (RWE) in regulatory approvals, health technology …

[HTML][HTML] Digital health and big data analytics: implications of real-world evidence for clinicians and policymakers

T Magalhães, RJ Dinis-Oliveira… - International Journal of …, 2022 - mdpi.com
Real world data (RWD) and real-world evidence (RWE) plays an increasingly important role
in clinical research since scientific knowledge is obtained during routine clinical large-scale …

Evolving use of real-world evidence in the regulatory process: a focus on immuno-oncology treatment and outcomes

JC O'Donnell, TK Le, R Dobrin, M Higashi… - Future …, 2021 - Future Medicine
In recent years, regulatory bodies have increasingly recognized the utility of real-world
evidence (RWE) for supplementing and supporting clinical trial data in new drug …

Using bayesian evidence synthesis methods to incorporate real-world evidence in surrogate endpoint evaluation

L Wheaton, A Papanikos, A Thomas… - Medical Decision …, 2023 - journals.sagepub.com
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 …

Interpretable AI in healthcare: Enhancing fairness, safety, and trust

S MacDonald, K Steven, M Trzaskowski - Artificial Intelligence in Medicine …, 2022 - Springer
The value and future potentials of AI in healthcare are becoming self-evident, presenting an
escalating body of evidence. However, the adoption into clinical practice is still significantly …

Clouds across the new dawn for clinical, diagnostic and biological data: accelerating the development, delivery and uptake of personalized medicine

D Horgan, E Capoluongo, F Dube, D Trapani… - Diagnosis, 2023 - degruyter.com
Growing awareness of the genetic basis of disease is transforming the opportunities for
improving patient care by accelerating the development, delivery and uptake of …

Regulatory and HTA Considerations for Development of Real‐World Data Derived External Controls

LH Curtis, O Sola‐Morales, J Heidt… - Clinical …, 2023 - Wiley Online Library
Regulators and Health Technology Assessment (HTA) bodies are increasingly familiar with,
and publishing guidance on, external controls derived from real‐world data (RWD) to …

Real-World Efficacy and Safety of Extended-Release Methylphenidate (PRC-063) in the Treatment of ADHD in Pediatric and Adult Subjects: Results of a Phase IV …

J van Stralen, G Parhar, A Parhar… - Journal of Attention …, 2023 - journals.sagepub.com
Objective: To evaluate the real-world efficacy, safety, and functional outcomes of PRC-063
(multilayer-release methylphenidate) versus lisdexamfetamine (LDX) in ADHD subjects in a …