Realising precision oncology through shared real-world data infrastructure
DOI:
https://doi.org/10.2340/1651-226X.2026.45074Keywords:
Precision medicine, Secondary Data Analysis, Costs and Cost Analysis, Health data governance, Public-private partnership, Real-world data, Outcomes-based pricing negotiationsDownloads
References
Rulten SL, Grose RP, Gatz SA, Jones JL, Cameron AJM. The future of precision oncology. Int J Mol Sci. 2023;24(16):12613.
https://doi.org/10.3390/ijms241612613 DOI: https://doi.org/10.3390/ijms241612613
European Medicines Agency. Guideline on the clinical evaluation of anticancer medicinal products [Internet]. 2023 Nov. Report No.: EMA/CHMP/205/95 Rev.6. [Cited date: 04-11-2025] Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-evaluation-anticancer-medicinal-products-revision-6_en.pdf?utm_source=chatgpt.com#page=4.05
Lozano-Ortega G, Hodgson M, Csintalan F, Zerbini C, Johnston K, Nam J. PPM11 tumour-specific randomized controlled trials in rare oncogene-driven cancers: asking for the impossible? Value Health. 2019;22:S838–9.
https://doi.org/10.1016/j.jval.2019.09.2328 DOI: https://doi.org/10.1016/j.jval.2019.09.2328
Birk HO, Vrangelbæk K, Rudkjøbing A, Krasnik A, Eriksen A, Richardson E, et al. Denmark: health system review. Copenhagen Ø, Denmark: WHO Regional Office for Europe; 2024. 151 p. (Health systems in transition).
Sherman RE, Anderson SA, Dal Pan GJ, Gray GW, Gross T, Hunter NL, et al. Real-world evidence – what is it and what can it tell us? N Engl J Med. 2016;375(23):2293–7.
https://doi.org/10.1056/NEJMsb1609216 DOI: https://doi.org/10.1056/NEJMsb1609216
Derksen JWG, Martins-Branco D, Valachis A, Pellat A, Van Nassau SCMW, Aggarwal A, et al. Real-world evidence reported for clinical efficacy evaluation in European Public Assessment Reports of authorised targeted therapies for solid malignancies: a comprehensive review (2018–2022). ESMO Real World Data Digit Oncol. 2024;4:100039.
https://doi.org/10.1016/j.esmorw.2024.100039 DOI: https://doi.org/10.1016/j.esmorw.2024.100039
Riskin DJ, Monda KL, Gagne JJ, Reynolds R, Garan AR, Dreyer N, et al. Implementing accuracy, completeness, and traceability for data reliability. JAMA Netw Open. 2025;8(3):e250128.
https://doi.org/10.1001/jamanetworkopen.2025.0128 DOI: https://doi.org/10.1001/jamanetworkopen.2025.0128
Haj Mohammad SF, Timmer HJL, Zeverijn LJ, Geurts BS, Spiekman IAC, Verkerk K, et al. The evolution of precision oncology: the ongoing impact of the Drug Rediscovery Protocol (DRUP). Acta Oncol. 2024;63:368–72.
https://doi.org/10.2340/1651-226X.2024.34885 DOI: https://doi.org/10.2340/1651-226X.2024.34885
Puco K, Fagereng GL, Brabrand S, Niehusmann P, Støre Blix E, Samdal Steinskog ES, et al. IMPRESS-Norway: improving public cancer care by implementing precision medicine in Norway; inclusion rates and preliminary results. Acta Oncol. 2024;63:379–84.
https://doi.org/10.2340/1651-226X.2024.28322 DOI: https://doi.org/10.2340/1651-226X.2024.28322
Lassen U. ProTarget – a Danish nationwide clinical trial on targeted cancer treatment based on genomic profiling [Internet]. clinicaltrials.gov; 2025 [cited 2026 Jan 7]. Report No.: NCT04341181. Available from: https://clinicaltrials.gov/study/NCT04341181
Taskén K, Haj Mohammad SF, Fagereng GL, Sørum Falk R, Helland Å, Barjesteh Van Waalwijk Van Doorn-Khosrovani S, et al. PCM4EU and PRIME-ROSE: collaboration for implementation of precision cancer medicine in Europe. Acta Oncol. 2024;63:385–91.
https://doi.org/10.2340/1651-226X.2024.34791 DOI: https://doi.org/10.2340/1651-226X.2024.34791
Dickson D, Johnson J, Bergan R, Owens R, Subbiah V, Kurzrock R. The master observational trial: a new class of master protocol to advance precision medicine. Cell. 2020;180(1):9–14.
https://doi.org/10.1016/j.cell.2019.12.009 DOI: https://doi.org/10.1016/j.cell.2019.12.009
Bjerrum A, Lassen U. Putting data to work for precision medicine. Cell Rep Med. 2023;4(7):101090.
https://doi.org/10.1016/j.xcrm.2023.101090 DOI: https://doi.org/10.1016/j.xcrm.2023.101090
Pizzamiglio C, Vernon HJ, Hanna MG, Pitceathly RDS. Designing clinical trials for rare diseases: unique challenges and opportunities. Nat Rev Methods Primer. 2022;2(1):13.
https://doi.org/10.1038/s43586-022-00100-2 DOI: https://doi.org/10.1038/s43586-022-00100-2
Mathoulin-Pélissier S, Pritchard-Jones K. Evidence-based data and rare cancers: the need for a new methodological approach in research and investigation. Eur J Surg Oncol. 2019;45(1):22–30.
https://doi.org/10.1016/j.ejso.2018.02.015 DOI: https://doi.org/10.1016/j.ejso.2018.02.015
OHDSI. Observational Medical Outcomes Partnership (OMOP) Common Data Model (CDM) [Internet]. 2023. [Cited date: 04-11-2025] Available from: https://www.ohdsi.org/data-standardization/
European Commission. European Health Data Space Regulation (EHDS) [Internet]. [cited 2025 Nov 10]. Available from: https://health.ec.europa.eu/ehealth-digital-health-and-care/european-
health-data-space-regulation-ehds_en
The Finnish Innovation Fund (SITRA). VALO – Value from Nordic health data [Internet]. [cited 2025 Nov 10]. Available from: https://www.sitra.fi/en/projects/valo-value-from-nordic-health-data/
Verbiest ATL, Theophanous S, Ilonen I, Golozar A, Carus J, Cheese E, et al. 1867P FALCON-Lung: Evolving global ICI treatment patterns and outcomes in the era of personalized mNSCLC treatment, Annals Oncology 2025;36;S1004.
https://doi.org/10.1016/j.annonc.2025.08.2491 DOI: https://doi.org/10.1016/j.annonc.2025.08.2491
Published
How to Cite
Issue
Section
Categories
License
Copyright (c) 2026 Andreas Bjerrum, Andreas Fanø, Ulrik Lassen

This work is licensed under a Creative Commons Attribution 4.0 International License.
