External comparator arms should be used when investigating novel therapies for gastrointestinal stromal tumor and other rare tumors to facilitate drug testing and regulatory approvals.
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Acknowledgements
This work was funded in part by the FERO Foundation and the Asociación Española Contra el Cáncer (AECC CLSEN20004SERR), both through grants to C.S. The general work of M.C.H. has been supported by grants from the US Department of Veterans Affairs (1 I01 BX005358-01A1) and National Institutes of Health/National Cancer Institute (NIH/NCI; 1 R21 CA263400-01) and by philanthropic donations from the GIST Cancer Research Fund and the Jonathan David Foundation. The work of J.K.S. was supported by NIH/NCI R01 CA226803-01A1. We thank J. Carmona (scientific strategy office, VHIO) for critical review of this manuscript.
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C.S. has received research funding (institution) from IDRX, Blueprint, Karyopharm, Pfizer, Deciphera and Bayer; consulting fees (advisory role) from Newbay, Cogent, Immunicum AB, Deciphera and Blueprint; payment for lectures from Roche, PharmaMar, Deciphera, Bayer and Blueprint; and travel grants from Gilead, PharmaMar, Pfizer and Bayer AG. M.C.H. reports consulting roles for Blueprint, Deciphera, C Stone Pharmaceuticals, Zai Labs, Cogent and Theseus Pharmaceuticals, and patents, royalties and other intellectual property regarding a patent on treatment of GIST-licensed to Novartis. G.V. has received speaker’s fees from MSD, GSK, Pfizer and Pierre Fabre, and has held an advisory role with AstraZeneca. S.G. has received funding (institution) from IDRX, Theseus, Blueprint, Deciphera, BioAtla, Springworks, Merck, Eisai and Daiichi Sankyo; consulting fees from Immunicum, Deciphera, Blueprint and Kayothera; equity in Abbott Labs; and other fees from WCG (Ayala-DSMB). J.-Y.B. receives research support (institution) from Deciphera, Eisai, Roche, Pharmamar, Bayer, GSK and MSD. J.K.S. receives consultant fees from Deciphera, Aadi and Grand Rounds; serves as a consultant for CureMatch; has received speaker’s fees from Deciphera, Hoffman-La Roche, Foundation Medicine, Merck, QED and Daiichi Sankyo; and owns stock in Personalis. R.L.J. reports consulting or advisory roles for Lilly, Immune Design, Merck Serono, Adaptimmune, Daiichi Sankyo, Eisai, Morphotek, TRACON Pharma, Immodulon Therapeutics, Deciphera, PharmaMar, Blueprint Medicines Corporation, Clinigen Group, Epizyme, Boehringer Ingelheim, Bayer, Karma Oncology and UpToDate; research funding from GlaxoSmithKline; and reimbursement of travel and accommodation expenses from PharmaMar. P.R. has received honoraria for lectures and Advisory Board meetings from MSD, BMS, Novartis, Pierre Fabre, Sanofi, Merck, Philogen and Astra Zeneca outside of the scope of this study. N.S. has received consulting fees (advisory role) from Deciphera, Bayer, Boehringer-Ingelheim, Epizyme and Aadi Biosciences. J.C.T. consulted for Blueprint, Deciphera, Cogent, Daiichi-Sankyo, Foghorn, Bayer and Adcendo. Life Raft Group, where S.R. and D.E. are employed, has received program-related grants from Blueprint, Cogent Biosciences, Daichii Sankyo, Deciphera, Genentech, IDRx, Novartis, Pfizer and Theseus. The remaining authors declare no conflicts of interest.
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Serrano, C., Rothschild, S., Villacampa, G. et al. Rethinking placebos: embracing synthetic control arms in clinical trials for rare tumors. Nat Med 29, 2689–2692 (2023). https://doi.org/10.1038/s41591-023-02578-z
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DOI: https://doi.org/10.1038/s41591-023-02578-z
