Clinical Trials Impacted by the COVID-19 Pandemic: Adaptive Designs to the Rescue?

Design changes Heterogeneity Interim analysis SARS-CoV-2

Journal

Statistics in biopharmaceutical research
ISSN: 1946-6315
Titre abrégé: Stat Biopharm Res
Pays: United States
ID NLM: 101507745

Informations de publication

Date de publication:
19 Aug 2020
Historique:
entrez: 30 6 2021
pubmed: 1 7 2021
medline: 1 7 2021
Statut: epublish

Résumé

Very recently the new pathogen severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was identified and the coronavirus disease 2019 (COVID-19) declared a pandemic by the World Health Organization. The pandemic has a number of consequences for ongoing clinical trials in non-COVID-19 conditions. Motivated by four current clinical trials in a variety of disease areas we illustrate the challenges faced by the pandemic and sketch out possible solutions including adaptive designs. Guidance is provided on (i) where blinded adaptations can help; (ii) how to achieve Type I error rate control, if required; (iii) how to deal with potential treatment effect heterogeneity; (iv) how to use early read-outs; and (v) how to use Bayesian techniques. In more detail approaches to resizing a trial affected by the pandemic are developed including considerations to stop a trial early, the use of group-sequential designs or sample size adjustment. All methods considered are implemented in a freely available R shiny app. Furthermore, regulatory and operational issues including the role of data monitoring committees are discussed.

Identifiants

pubmed: 34191979
doi: 10.1080/19466315.2020.1799857
pii: 1799857
pmc: PMC8011492
doi:

Types de publication

Journal Article

Langues

eng

Pagination

461-477

Informations de copyright

© 2020 American Statistical Association.

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Auteurs

Cornelia Ursula Kunz (CU)

Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach, Germany.

Silke Jörgens (S)

Janssen-Cilag GmbH, Neuss, Germany.

Frank Bretz (F)

Novartis Pharma AG, Basel, Switzerland.
Section for Medical Statistics, Medical University of Vienna, Vienna, Austria.

Nigel Stallard (N)

Division of Health Sciences, Warwick Medical School, The University of Warwick, Coventry, UK.

Kelly Van Lancker (K)

Department of Applied Mathematics, Computer Science and Statistics, Ghent University, Ghent, Belgium.

Dong Xi (D)

Novartis Pharmaceuticals, East Hanover, NJ.

Sarah Zohar (S)

INSERM, Centre de Recherche des Cordeliers, Sorbonne Université, Université de Paris, Paris, France.

Christoph Gerlinger (C)

Statistics and Data Insights, Bayer AG, Berlin, Germany.
Department of Gynecology, Obstetrics and Reproductive Medicine, University Medical School of Saarland, Homburg/Saar, Germany.

Tim Friede (T)

Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany.
DZHK (German Center for Cardiovascular Research), Partner Site Göttingen, Göttingen, Germany.

Classifications MeSH