Leveraging external data in the design and analysis of clinical trials in neuro-oncology.

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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Declaration of interests RR received research support from the Project Data Sphere, outside of submitted work. IR-R reports employment and owns stocks of Roche and Genentech. FM reports employment at Medicenna Therapeutics. LEA reports employment and owns stocks of Novartis. JEA reports employment and owns stocks of Chimerix. LKA and EA-C report employment at Candel Therapuetics. SB reports grants and personal fees from Novocure; grants from Incyte, GSK, and Eli Lilly; and personal fees from Bayer and Sumitomo Dainippon. MK reports personal fees from Ipsen, Pfizer, Roche, and Jackson Laboratory for Genomic Medicine and research funding paid to his institution from Specialised Therapeutics. TC reports personal fees from Roche, Trizel, Medscape, Bayer, Amgen, Odonate Therapeutics, Pascal Biosciences, DelMar Pharmaceuticals, Tocagen, Karyopharm, GW Pharmaceuticals, Kiyatec, AbbVie, Boehinger Ingelheim, VBI Vaccines, Dicephera, VBL Therapeutics, Agios, Merck, Genocea, Puma, Lilly, Bristol Myers Squibb, Cortice, Wellcome Trust; and stock options from Notable Labs. TC has a patent (62/819,322) with royalties paid to Katmai and is a board member for the 501c3 Global Coalition for Adaptive Research. PYW reports personal fees from Abbvie, Agios, AstraZeneca, Blue Earth Diagnostics, Eli Lilly, Genentech, Roche, Immunomic Therapeutics, Kadmon, Kiyatec, Merck, Puma, Vascular Biogenics, Taiho, Tocagen, Deciphera, and VBI Vaccines; and research support from Agios, AstraZeneca, Beigene, Eli Lily, Genentech, Roche, Karyopharm, Kazia, MediciNova, Merck, Novartis, Oncoceutics, Sanofi-Aventis, and VBI Vaccines. BMA reports employment at Foundation Medicine; personal fees from AbbVie, Bristol Myers Squibb, Precision Health Economics, and Schlesinger Associates; and research support from Puma, Eli Lilly, Celgene. SV, JM, BL, M-YCP, DA, KT, and LT declare no competing interests.