Leveraging external data in the design and analysis of clinical trials in neuro-oncology - 28/09/21

Doi : 10.1016/S1470-2045(21)00488-5

Rifaquat Rahman, MD ^a,⁎ , Steffen Ventz, PhD ^b, Jon McDunn, PhD ^d, Bill Louv, PhD ^d, Irmarie Reyes-Rivera, PhD ^e, Mei-Yin C Polley, PhD ^f, Fahar Merchant, PhD ^g, Lauren E Abrey, MD ^h, Joshua E Allen, PhD ⁱ, Laura K Aguilar, MD ^j, Estuardo Aguilar-Cordova, MD ^j, David Arons, JD ^k, Kirk Tanner, PhD ^k, Stephen Bagley, MD ^l, Mustafa Khasraw, MD ^m, Timothy Cloughesy, ProfMD ⁿ, Patrick Y Wen, ProfMD ^c, Brian M Alexander, MD ^a,^o,^{*
Co-senior authors}, Lorenzo Trippa, PhD ^b,^{*
Co-senior authors}

^a Department of Radiation Oncology, Dana-Farber/Brigham and Women’s Cancer Center, Harvard Medical School, Boston, MA, USA

^b Department of Data Sciences, Dana-Farber Cancer Institute, Harvard T H Chan School of Public Health, Boston, MA, USA

^c Center for Neuro-Oncology, Dana-Farber Cancer Institute, Harvard T H Chan School of Public Health, Boston, MA, USA

^d Project Data Sphere, Morrisville, NC, USA

^e F Hoffmann-La Roche, Basel, Switzerland

^f Department of Public Health Sciences, University of Chicago, Chicago, IL, USA

^g Medicenna Therapeutics, Toronto, ON, Canada

^h Novartis, Basel, Switzerland

ⁱ Chimerix, Durham, NC, USA

^j Candel Therapeutics, Needham, MA, USA

^k National Brain Tumor Society, Newton, MA, USA

^l Division of Hematology and Oncology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

^m Department of Neurosurgery, Preston Robert Tisch Brain Tumor Center, Duke University Medical Center, Durham, NC, USA

ⁿ Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA, USA

^o Foundation Medicine, Cambridge, MA, USA

^* Correspondence to: Dr Rifaquat Rahman, Department of Radiation Oncology, Dana-Farber/Brigham and Women’s Cancer Center, Harvard Medical School, Boston, MA 02215, USA Department of Radiation Oncology Dana-Farber/Brigham and Women’s Cancer Center Harvard Medical School Boston MA 02215 USA

Summary

Integration of external control data, with patient-level information, in clinical trials has the potential to accelerate the development of new treatments in neuro-oncology by contextualising single-arm studies and improving decision making (eg, early stopping decisions). Based on a series of presentations at the 2020 Clinical Trials Think Tank hosted by the Society of Neuro-Oncology, we provide an overview on the use of external control data representative of the standard of care in the design and analysis of clinical trials. High-quality patient-level records, rigorous methods, and validation analyses are necessary to effectively leverage external data. We review study designs, statistical methods, risks, and potential distortions in using external data from completed trials and real-world data, as well as data sources, data sharing models, ongoing work, and applications in glioblastoma.

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Plan

Introduction

Early phase trial designs

Overview of trial designs that include external data

Externally augmented clinical trial designs

External data sources

External control groups beyond neuro-oncology

Considerations and implications for regulatory decision making

Data sharing models

Conclusion

Search strategy and selection criteria

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Vol 22 - N° 10

P. e456-e465 - octobre 2021 Retour au numéro

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Leveraging external data in the design and analysis of clinical trials in neuro-oncology - 28/09/21

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