Realising the potential of tumour-agnostic therapies in Europe
The next evolution in precision oncology isn’t about where cancer starts – it’s about its molecular identity.
This white paper brings together expert recommendations to guide the future of oncology in Europe.
The white paper was funded by AstraZeneca (Major Funder), Bayer AG (Intermediate Funder), MSD (Intermediate Funder) and Daiichi Sankyo (Minor Funder). The white paper was developed with input from an expert working group; members contributed voluntarily and without compensation. The project to convene the expert working group was initiated by AstraZeneca. The Secretariat was provided by Syneos Health Communications. The findings and views expressed herein are based on the insights of the working group and do not necessarily reflect the views of the Funders. AstraZeneca has reviewed the white paper for scientific and legal accuracy.
Realising the potential of tumour-agnostic therapies in Europe
The next evolution in precision oncology isn’t about where cancer starts – it’s about its molecular identity.
This white paper brings together expert recommendations to guide the future of oncology in Europe.
The white paper was funded by AstraZeneca (Major Funder), Bayer AG (Intermediate Funder), MSD (Intermediate Funder) and Daiichi Sankyo (Minor Funder). The white paper was developed with input from an expert working group; members contributed voluntarily and without compensation. The project to convene the expert working group was initiated by AstraZeneca. The Secretariat was provided by Syneos Health Communications. The findings and views expressed herein are based on the insights of the working group and do not necessarily reflect the views of the Funders. AstraZeneca has reviewed the white paper for scientific and legal accuracy.
Tumour-agnostic therapies target specific molecular alterations regardless of cancer location or origin, making them a key treatment innovation for patients.1
The science is moving fast, and we must make sure Europe’s systems keep pace and advance evaluation, access and adoption of innovative medicines such as tumour-agnostic therapies.
Driven by a shared sense of urgency, leading experts from across Europe and beyond joined forces to align on where we are today and chart the path forward.
This white paper lays out the key challenges and the system-level changes needed to unlock the full potential of tumour-agnostic therapies, and how to deliver them to all eligible patients across Europe faster.
Over the last decades, Europe has seen steady improvements in survival rates for people with cancer.2 Advances in diagnosis, care and treatment – including precision medicines – have significantly contributed to this.3,4
By targeting specific molecular alterations regardless of cancer location or origin, tumour-agnostic therapies act on the scientific identity of cancer cells, including their basis for development and growth. They have the potential to extend the benefits of precision medicine to areas of high unmet need.1
Tumour-agnostic therapies are the next step in the fight against cancer.
However, the characteristics of tumour-agnostic therapies present specific challenges to existing evidence, access and clinical practice frameworks.5 Without system-level change, health systems risk failing patients by delaying the delivery of innovative treatments or denying access to them entirely.
The number of tumour-agnostic therapies is increasing,6,7 and Europe faces an opportunity for action to ensure that cancer patients can benefit from the latest scientific innovations in the fight against cancer.
How do we create an environment that accelerates access to tumour-agnostic therapies for patients?
Recommendations
Regulators and Health Technology Assessment (HTA) bodies should publish guidance outlining requirements for the acceptance of alternative trial designs and real-world evidence in the assessment of tumour-agnostic therapies.
Structured engagement is needed on evidence requirements, conditional reimbursement and real-world data collection to enable timely access to tumour-agnostic therapies, involving patients, regulators, HTA bodies, healthcare professionals and developers.
Regulators and HTA bodies should be ready to evolve guidance, policy and practice in response to new insights from pilot programmes, regulatory sandboxes and real-world experience.
Healthcare decision-makers must ensure that clinical practice keeps pace with tumour-agnostic therapies through guidelines, workflows and testing infrastructure.
The expert contributors
Sahar Barjesteh van Waalwijk van Doorn-Khosrovani, Prime-Rose Consortium and Leiden University Medical Centre, Netherlands
Jean-Yves Blay, Centre Léon Bérard, France
Yvonne-Beatrice Böhler, TH Köln, University of Applied Sciences, Germany
Antonella Cardone, Cancer Patients Europe, Belgium
Ilaria Colombo, Oncology Institute of Southern Switzerland, Switzerland
Jan Geissler, Patvocates, Germany
Viktor Grünwald, University Hospital Essen, Germany
Ebba Hallersjö Hult, Vision Zero Cancer, Stockholm School of Economics, Sweden
Garbiñe Lizeaga, Hospital Universitario Donostia, Spain
Domenica Lorusso, Humanitas University and Humanitas San Pio X Hospital, Italy
Lavinia Magee, Lung Cancer Europe
Warnyta Minnaard, World CUP Alliance, Netherlands
Frédérique Penault-Llorca, University of Clermont-Auvergne, France
Russell Petty, University of Dundee, United Kingdom
Javier Puente, Hospital Clinico San Carlos, Madrid, Spain
Jade Putzolu, Neovidence, France
Naureen Starling, The Royal Marsden NHS Foundation Trust, United Kingdom
Albrecht Stenzinger, University Hospital Heidelberg, Germany
Vivek Subbiah, Sarah Cannon Research Institute, United States
Benedikt Westphalen, Comprehensive Cancer Centre, LMU University Hospital Munich, Germany
The next era of precision oncology is here. Discover the urgent steps needed to align evidence, access and care with tumour-agnostic therapies.
References:
- 1.Westphalen CB, Martins-Branco D, Beal JR, et al. The ESMO Tumour-Agnostic Classifier and Screener (ETAC-S): a tool for assessing tumour-agnostic potential of molecularly guided therapies and for steering drug development. Ann Oncol. 2024;35(11):936-953.
- 2.OECD/European Commission. EU country cancer profiles synthesis report 2025. Accessed October 2025 [https://www.oecd.org/en/publications/eu-country-cancer-profiles-synthesis-report-2025_20ef03e1-en.html].
- 3.Lichtenberg FR. The relationship between pharmaceutical innovation and cancer mortality in Spain, from 1999 to 2016. Value Health. 2023;26(12):1711–1720.
- 4.Luís AB, Seo MK. Has the development of cancer biomarkers to guide treatment improved health outcomes? Eur J of Health Eco. 2021;22(5):789–810.
- 5.Wu S, Thawani R. Tumor-agnostic therapies in practice: challenges, innovations, and future perspectives. Cancers. 2025;17(5):801.
- 6.Hierro C, Matos I, Martin-Liberal J, et al. Agnostic-Histology Approval of New Drugs in Oncology: Are We Already There? Clin Cancer Res. 2019;25:3210–3219.
- 7.Weymann D, Pollard S, Lam H, et al. Toward best practices for economic evaluations of tumor-agnostic therapies: a review of current barriers and solutions. Value Health. 2023;26(11):1608–1617.
This white paper was funded by AstraZeneca (Major Funder), Bayer AG (Intermediate Funder), MSD (Intermediate Funder) and Daiichi Sankyo (Minor Funder). The project to convene the expert working group was initiated by AstraZeneca. The Secretariat was provided by Syneos Health Communications. The findings and views expressed herein are based on the insights of the working group and do not necessarily reflect the views of the Funders. AstraZeneca has reviewed the white paper for scientific and legal accuracy.
This white paper was developed by Syneos Health Communications with contributions from an expert working group assembled specifically to advance regulatory approval, access and clinical adoption of tumour-agnostic therapies in Europe. This multidisciplinary group brought together leading experts across geographies, various disciplines (e.g. healthcare professionals, health technology assessment specialists and patient advocates) and tumour specialisms. Working group members participated in their individual capacities and received no financial compensation from the Funders or Secretariat.
Veeva number: Z3-1193 | Date of preparation: October 2025