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Lookup NU author(s): Professor Ruth PlummerORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2025 The Authors. Rare cancers account for a quarter of cancer diagnoses in Europe yet clinical research, diagnosis, treatment access, and survival outcomes lag significantly behind common cancers. Despite the potential of precision oncology, the consistent implementation of comprehensive genomic profiling in routine clinical practice and robust evidence-generation remains a challenge in this population, compounded by regulatory hurdles and a lack of investment in drug development. A concerted effort across all stakeholders is required to optimise diagnostics, including access to molecular profiling, to expedite clinical trials and treatment access, and to gather high-quality data, including patient-reported outcomes, in rare cancers. Some initiatives are already showing promise including the establishment of national expert reference centres and European Reference Networks such as EURACAN. However, further collaboration is required to speed up the diagnostic trajectory so that rare cancer patients present with less late-stage disease, and to facilitate clinical trials leading to wider access to precision oncology drugs shown to be safe and effective. In the context of so many hurdles (diagnosis, treatment, research, development and regulatory), there is an even greater role for patient and clinical trial organisations and funders to help fill the aforementioned gaps. Innovative solutions are urgently required to address the high unmet medical need for patients with rare cancers.
Author(s): van der Graaf WTA, Heiss NS, Hynes CL, Keller SP, Weinman A, Blay J-Y, Franco P, Giles RH, Lacombe D, Schlatter P, Thomas DM, van Waalwijk van Doorn-Khosrovani SB, Williamson M, Plummer R
Publication type: Review
Publication status: Published
Journal: Cancer Treatment Reviews
Year: 2025
Volume: 140
Print publication date: 01/11/2025
Online publication date: 14/08/2025
Acceptance date: 13/08/2025
ISSN (print): 0305-7372
ISSN (electronic): 1532-1967
Publisher: W.B. Saunders Ltd
URL: https://doi.org/10.1016/j.ctrv.2025.103013
DOI: 10.1016/j.ctrv.2025.103013
