Clinical trial design in the era of precision medicine

E Fountzilas, AM Tsimberidou, HH Vo, R Kurzrock - Genome medicine, 2022 - Springer
Recent rapid biotechnological breakthroughs have led to the identification of complex and
unique molecular features that drive malignancies. Precision medicine has exploited next …

Real-world data: bridging the gap between clinical trials and practice

BE Wilson, CM Booth - EClinicalMedicine, 2024 - thelancet.com
Real-world data (RWD) are rapidly emerging sources of information for patients, clinicians
and regulators. While randomized controlled trials (RCTs) reduce bias and confounding …

Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy?

B Wieseler, M Neyt, T Kaiser, F Hulstaert, J Windeler - bmj, 2023 - bmj.com
Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy? |
The BMJ Skip to main content Intended for healthcare professionals Access provided by Google …

Prevalence of missing data in the national cancer database and association with overall survival

DX Yang, R Khera, JA Miccio, V Jairam… - JAMA network …, 2021 - jamanetwork.com
Importance Cancer registries are important real-world data sources consisting of data
abstraction from the medical record; however, patients with unknown or missing data are …

Evaluating generalizability of oncology trial results to real-world patients using machine learning-based trial emulations

X Orcutt, K Chen, R Mamtani, Q Long, RB Parikh - Nature Medicine, 2025 - nature.com
Randomized controlled trials (RCTs) evaluating anti-cancer agents often lack
generalizability to real-world oncology patients. Although restrictive eligibility criteria …

Reexamining time from breast cancer diagnosis to primary breast surgery

AA Wiener, BM Hanlon, JR Schumacher… - JAMA …, 2023 - jamanetwork.com
Importance Although longer times from breast cancer diagnosis to primary surgery have
been associated with worse survival outcomes, the specific time point after which it is …

Improved postoperative outcomes after prehabilitation for colorectal cancer surgery in older patients: an emulated target trial

TC Heil, EGG Verdaasdonk, HAAM Maas… - Annals of surgical …, 2023 - Springer
Background The aim of this study was to assess the effect of a multimodal prehabilitation
program on perioperative outcomes in colorectal cancer patients with a higher postoperative …

Design differences and variation in results between randomised trials and non-randomised emulations: meta-analysis of RCT-DUPLICATE data

R Heyard, L Held, S Schneeweiss, SV Wang - BMJ medicine, 2024 - pmc.ncbi.nlm.nih.gov
Objective To explore how design emulation and population differences relate to variation in
results between randomised controlled trials (RCT) and non-randomised real world …

Practice patterns for organ preservation in US patients with rectal cancer, 2006-2020

A Loria, MA Tejani, LK Temple, CF Justiniano… - JAMA …, 2024 - jamanetwork.com
Importance In March 2023, the National Comprehensive Cancer Network endorsed watch
and wait for those with complete clinical response to total neoadjuvant therapy. Neoadjuvant …

Novel prognostic staging system for patients with de novo metastatic breast cancer

JK Plichta, SM Thomas, DF Hayes… - Journal of Clinical …, 2023 - ascopubs.org
PURPOSE Given the heterogeneity and improvement in outcomes for metastatic breast
cancer (MBC), we developed a staging system that refines prognostic estimates for patients …