External Control Arms in Oncology: Current Use and Future Directions

FDA Approval Summary: Selumetinib for Plexiform Neurofibroma

(2021) Denise Casey et al.   CLINICAL CANCER RESEARCH

FDA Approval Summary: Pembrolizumab plus Lenvatinib for Endometrial Carcinoma, a Collaborative International Review under Project Orbis

(2020) Shaily Arora et al.   CLINICAL CANCER RESEARCH

Real‐world evidence to support regulatory decision‐making for medicines: Considerations for external control arms

(2020) Mehmet Burcu et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Synthetic and External Controls in Clinical Trials – A Primer for Researchers

(2020) Kristian Thorlund et al.   Clinical Epidemiology

Regulatory Considerations for Contribution of Effect of Drugs used in Combination Regimens: Renal Cell Cancer Case Studies

(2020) Jamie R Brewer et al.   CLINICAL CANCER RESEARCH

A Bayesian approach in design and analysis of pediatric cancer clinical trials

(2020) Jingjing Ye et al.   PHARMACEUTICAL STATISTICS

A Food and Drug Administration analysis of survival outcomes comparing the Adjuvant Paclitaxel and Trastuzumab trial with an external control from historical clinical trials

(2020) L. Amiri-Kordestani et al.   ANNALS OF ONCOLOGY

Estimation of historical control rate for a single arm de-escalation study – Application to the POSITIVE trial

(2020) Zhuoxin Sun et al.   BREAST

Single-arm Trials With External Comparators and Confounder Misclassification

(2020) Michael Webster-Clark et al.   MEDICAL CARE

Methods for external control groups for single arm trials or long‐term uncontrolled extensions to randomized clinical trials

(2020) John D. Seeger et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Design and Evaluation of an External Control Arm Using Prior Clinical Trials and Real-World Data

(2019) Steffen Ventz et al.   CLINICAL CANCER RESEARCH

Using Electronic Health Records to Derive Control Arms for Early Phase Single‐Arm Lung Cancer Trials: Proof‐of‐Concept in Randomized Controlled Trials

(2019) Gillis Carrigan et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1

(2018) Andrea M Gross et al.   NEURO-ONCOLOGY

Minimizing Patient Burden Through the Use of Historical Subject-Level Data in Innovative Confirmatory Clinical Trials

(2018) Jessica Lim et al.   Therapeutic Innovation & Regulatory Science

FDA Approval: Blinatumomab for Patients with B-cell Precursor Acute Lymphoblastic Leukemia in Morphologic Remission with Minimal Residual Disease

(2018) Emily Y. Jen et al.   CLINICAL CANCER RESEARCH

Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas

(2014) B. C. Widemann et al.   NEURO-ONCOLOGY

Adaptive adjustment of the randomization ratio using historical control data

(2013) Brian P Hobbs et al.   Clinical Trials

Prognostic score–based balance measures can be a useful diagnostic for propensity score methods in comparative effectiveness research

(2013) Elizabeth A. Stuart et al.   JOURNAL OF CLINICAL EPIDEMIOLOGY

An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies

(2011) Peter C. Austin   MULTIVARIATE BEHAVIORAL RESEARCH

Assessment of benign tumor burden by whole-body MRI in patients with neurofibromatosis 1

(2008) Victor-F. Mautner et al.   NEURO-ONCOLOGY