Real-world evidence in support of precision medicine: clinico-genomic cancer data as a case study

V Agarwala, S Khozin, G Singal, C O'Connell, D Kuk… - Health …, 2018 - healthaffairs.org
The majority of US adult cancer patients today are diagnosed and treated outside the
context of any clinical trial (that is, in the real world). Although these patients are not part of a …

Model-assisted cohort selection with bias analysis for generating large-scale cohorts from the EHR for oncology research

B Birnbaum, N Nussbaum, K Seidl-Rathkopf… - arXiv preprint arXiv …, 2020 - arxiv.org
Objective Electronic health records (EHRs) are a promising source of data for health
outcomes research in oncology. A challenge in using EHR data is that selecting cohorts of …

The opportunities and shortcomings of using big data and national databases for sarcoma research

HG Lyu, AH Haider, AB Landman, CP Raut - Cancer, 2019 - Wiley Online Library
The rarity and heterogeneity of sarcomas make performing appropriately powered studies
challenging and magnify the significance of large databases in sarcoma research …

Harnessing the power of real‐world evidence (RWE): a checklist to ensure regulatory‐grade data quality

RA Miksad, AP Abernethy - Clinical Pharmacology & …, 2018 - Wiley Online Library
The role of real‐world evidence (RWE) in regulatory, drug development, and healthcare
decision‐making is rapidly expanding. Recent advances have increased the complexity of …

[HTML][HTML] Generating real-world tumor burden endpoints from electronic health record data: comparison of RECIST, radiology-anchored, and clinician-anchored …

SD Griffith, M Tucker, B Bowser, G Calkins, C Chang… - Advances in …, 2019 - Springer
Introduction Real-world evidence derived from electronic health records (EHRs) is
increasingly recognized as a supplement to evidence generated from traditional clinical …

Speed of adoption of immune checkpoint inhibitors of programmed cell death 1 protein and comparison of patient ages in clinical practice vs pivotal clinical trials

JM O'Connor, KL Fessele, J Steiner… - JAMA …, 2018 - jamanetwork.com
Importance The US Food and Drug Administration (FDA) is increasing its pace of approvals
for novel cancer therapeutics, including for immune checkpoint inhibitors of programmed cell …

[HTML][HTML] Machine learning methods in health economics and outcomes research—the PALISADE checklist: a good practices report of an ISPOR task force

WV Padula, N Kreif, DJ Vanness, B Adamson… - Value in health, 2022 - Elsevier
Advances in machine learning (ML) and artificial intelligence offer tremendous potential
benefits to patients. Predictive analytics using ML are already widely used in healthcare …

[HTML][HTML] Real-world outcomes of first-line pembrolizumab plus pemetrexed-carboplatin for metastatic nonsquamous NSCLC at US oncology practices

V Velcheti, X Hu, B Piperdi, T Burke - Scientific reports, 2021 - nature.com
Evidence from real-world clinical settings is lacking with regard to first-line immunotherapy
plus chemotherapy for the treatment of non-small cell lung cancer (NSCLC). Our aim was to …

Characteristics of real‐world metastatic non‐small cell lung cancer patients treated with nivolumab and pembrolizumab during the year following approval

S Khozin, AP Abernethy, NC Nussbaum, J Zhi… - The …, 2018 - academic.oup.com
Background Evidence from cancer clinical trials can be difficult to generalize to real‐world
patient populations, but can be complemented by real‐world evidence to optimize …

Real‐world outcomes of patients with metastatic non‐small cell lung cancer treated with programmed cell death protein 1 inhibitors in the year following US regulatory …

S Khozin, KR Carson, J Zhi, M Tucker, SE Lee… - The …, 2019 - academic.oup.com
Background Evidence from cancer clinical trials has strong internal validity but can be
difficult to generalize to real‐world patient populations. Here we analyzed real‐world …