External control arms in oncology: current use and future directions.

Humans Medical Oncology Neoplasms / drug therapy Randomized Controlled Trials as Topic Research Design United States United States Food and Drug Administration
Food and Drug Administration external controls real-word data

Journal

Annals of oncology : official journal of the European Society for Medical Oncology
ISSN: 1569-8041
Titre abrégé: Ann Oncol
Pays: England
ID NLM: 9007735

Informations de publication

Date de publication:
04 2022
Historique:
received: 16 04 2021
revised: 21 12 2021
accepted: 27 12 2021
pubmed: 14 1 2022
medline: 6 5 2022
entrez: 13 1 2022
Statut: ppublish

Résumé

Although randomized control trials allow for a comparison of treatment arms with minimal concern for confounding by known and unknown factors, a randomized study is not feasible in certain disease settings. When a randomized design is not possible, incorporating external control data into the study design can be an effective way to expand the interpretability of the results of an experimental arm by introducing the ability to carry out a formal or an informal comparative analysis. This paper provides an introduction to the concepts of external controls in oncology trials, followed by a review of relevant and current research on this topic. The paper also focuses on general considerations for designing a trial that may incorporate external control data, followed by case studies of the marketing applications submitted to the Food and Drug Administration that included external control data.

Identifiants

DOI: 10.1016/j.annonc.2021.12.015 PMID: 35026413
pubmed: 35026413
pii: S0923-7534(22)00006-0
doi: 10.1016/j.annonc.2021.12.015
pii:
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

376-383

Informations de copyright

Published by Elsevier Ltd.

Déclaration de conflit d'intérêts

Disclosure The authors have declared no conflicts of interest.

Auteurs

P S Mishra-Kalyani (PS)

Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA. Electronic address: pallavi.mishra-kalyani@fda.hhs.gov.

L Amiri Kordestani (L)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.

D R Rivera (DR)

Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

H Singh (H)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA; Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

A Ibrahim (A)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.

R A DeClaro (RA)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA; Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

Y Shen (Y)

Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.

S Tang (S)

Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.

R Sridhara (R)

Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

P G Kluetz (PG)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA; Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

J Concato (J)

Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.

R Pazdur (R)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA; Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

J A Beaver (JA)

Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA; Oncology Center of Excellence, U.S, Food and Drug Administration, Silver Spring, USA.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › External control arms in oncology: current use...
questionsmedicales.fr › Professions de santé › Médecine › Médecine interne › Oncologie médicale › External control arms in oncology: current use...
questionsmedicales.fr › Tumeurs › External control arms in oncology: current use...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Essais cliniques contrôlés comme sujet › Essais contrôlés randomisés comme sujet › External control arms in oncology: current use...
questionsmedicales.fr › Disciplines des sciences naturelles › Science › Recherche › Plan de recherche › External control arms in oncology: current use...
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