How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers.

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Title: How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers.
Authors: Stacchiotti S; Department of Medical Oncology, Adult Mesenchymal and Rare Tumours Unit Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy. Electronic address: silvia.stacchiotti@istitutotumori.mi.it., Pantziarka P; Anticancer Fund, Meise, Belgium; George Pantziarka TP53 Trust, London, United Kingdom., Leonard H; The EHE Rare Cancer Charity, Kingston-Upon-Thames, United Kingdom., Voltz C; European Medicines Agency, Amsterdam, The Netherlands., Abatedaga L; Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy; TU.TO.R. Tumori Toracici Rari, Patient Advocacy, Milan, Italy., Bouche G; Anticancer Fund, Meise, Belgium., Bouygues C; Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Bovee JVMG; Department of Pathology, Leiden University Medical Center (LUMC), Leiden, The Netherlands., Van der Graaf WTA; Department of Medical Oncology, Netherlands Cancer Institute, Amsterdam, The Netherlands; Department of Medical Oncology Erasmus MC Cancer Institute, Erasmus Medical Center, Rotterdam, The Netherlands., Rojas T; Arztpraxis Mylife Fulenbach, Fulenbach, Switzerland., D'Ambrosio L; Department of Medical Oncology, University of Turin, Turin, Italy., Donoghue M; U.S. Food and Drug Administration, Silver Spring, MD, United States., Enzmann H; European Medicines Agency, Amsterdam, The Netherlands., Feeney G; Sarcoma Patient Advocacy Global Network, Wolfersheim, Germany., Foggi P; Agenzia Italiana del Farmaco, Rome, Italy., Frezza AM; Department of Medical Oncology, Adult Mesenchymal and Rare Tumours Unit Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Herold R; European Medicines Agency, Amsterdam, The Netherlands., Jones RL; Sarcoma Unit, The Royal Marsden NHS and Institute of Cancer Research, London, United Kingdom., Kasper B; Sarcoma Unit, Mannheim Cancer Center, Mannheim University Medical Center, University of Heidelberg, Mannheim, Germany., Roes K; Radboud University Medical Center, Department IQ Health, Biostatistics, Nijmegen, The Netherlands., Marreaud S; European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium., Miceli R; Biostatistics for Clinical Research Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Robinson D; The EHE Foundation, Hobart, WI, United States., Sommer J; The Chordoma Foundation, Durham, NC, United States., Tap WD; Department of Medicine, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, United States., Tydings C; Sarcoma Patient Advocacy Global Network, Wolfersheim, Germany., Trama A; Department of Epidemiology and Data Science, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Houdt WV; Netherland Cancer Institute, Amsterdam, The Netherlands., van Oortmerssen G; Sarcoma Patient Advocacy Global Network, Wolfersheim, Germany., Wagner AJ; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, United States., Widemann B; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD, United States., Gelderblom H; Department of Medical Oncology , Leiden University Medical Center, Leiden, The Netherlands., Gronchi A; Department of Surgery, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Napolitano A; Sarcoma Unit, The Royal Marsden NHS and Institute of Cancer Research, London, United Kingdom., Dufresne A; Department of Medical Oncology, Centre Leon Berard, Lyon, France., Fumagalli ER; Department of Medical Oncology, Adult Mesenchymal and Rare Tumours Unit Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy., Baldi G; Department of Oncology, Hospital of Prato, Azienda USL Toscana Centro, Prato, Italy., Marquina G; Department of Medical Oncology, Hospital Clínico San Carlos, Madrid, Spain., Koseła-Paterczyk H; Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland., Martin Javier JB; Medical Oncology Department, Fundación Jimenez Diaz University Hospital, Madrid, Spain., Junker N; Department of Oncology, Sarcoma Unit, Herlev and Gentofte Hospital, Copenhagen, Denmark., Rutkowski P; Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland., Oyen WJG; Department of Radiology and Nuclear Medicine, Radboud University Medical Centre, Nijmegen, The Netherlands., Lacombe D; European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium., Pignatti F; European Medicines Agency, Amsterdam, The Netherlands., Demolis P; Icelandic Medicines Agency, Iceland.
Source: Cancer treatment reviews [Cancer Treat Rev] 2025 Nov; Vol. 140, pp. 103003. Date of Electronic Publication: 2025 Aug 06.
Publication Type: Journal Article; Review
Journal Info: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 7502030 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-1967 (Electronic) Linking ISSN: 03057372 NLM ISO Abbreviation: Cancer Treat Rev Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1532-1967
DOI:10.1016/j.ctrv.2025.103003