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European Organisation for Research and Treatment of Cancer

EORTC

The European Organisation for Research and Treatment of Cancer (EORTC) has delivered investigator driven, changing practice clinical trials since 1962.

Lobbying Activity

Meeting with Maya Matthews (Head of Unit Health and Food Safety)

6 Nov 2025 · Involvement of clinical experts taking part in joint clinical assessments and scientific consultations under the EU Health Technology Assessment (HTA) Regulation

Meeting with Tilly Metz (Member of the European Parliament)

26 Mar 2025 · Cancer, public health research

Meeting with Olivier Chastel (Member of the European Parliament)

6 Mar 2025 · Politique de santé de l'Union européenne - Optimisation des traitements

Response to Health technology assessment – Cooperation with the European Medicines Agency

15 Jul 2024

1. Introduction: The EORTCs position The European Organisation for the Research and Treatment of Cancer (EORTC) is pleased to be able to contribute to the implementation of the European HTA Regulation (HTAR), working towards the successful implementation of a regulation that improves the availability and funding of innovative health care technologies and streamlines how the value of medicines is assessed. We welcome the opportunity to provide feedback on this draft implementing act on cooperation with the European Medicines Agency (EMA) under the Health Technology Assessment (HTA) Regulation. The EORTC supports the goals of the Commission and is committed to the exchange of information between the Coordination Group on Health Technology Assessment (Member States), the Commission, and the EMA. We believe, however, that it is crucial to establish an inclusive and active stakeholder network for the collection and integration of involved stakeholders input under the HTAR for the successful planning and forecast of the joint clinical assessments and joint scientific consultations as well as for the facilitation of effective engagement with various stakeholders. Such a network would guarantee adherence to the HTA by providing a platform for diverse perspectives, improving the quality and relevance of the HTA through inclusive decision-making and thorough input. By enabling structured dialogue and efficient feedback mechanisms, the network would build consensus and address concerns, leading to broader acceptance and support for HTA findings. 2. Basis for successful implementation A. Truly multistakeholder effort needed Since HTA bodies use clinical information to estimate what health outcomes patients might experience when given a new medicine, patient-centred approaches and the use of high-quality data is crucial for the joint assessment process. Continuous feedback and knowledge-sharing would drive innovation and advancement in methodologies, promoting best practices. Such an involved network would also facilitate market access and uptake of new health technologies by understanding and addressing potential barriers, making the HTA process more effective and applicable to real-world circumstances. Full caution should be paid to ensure that a multidisciplinary panel of experts will be available to deliver meaningful assessment. Experts in the relevant disease and health technology(ies) such as clinicians, epidemiologists and health economists should be involved. The Commission and HTA coordination group should collaborate with reputable scientific organisations that can propose relevant experts. B. Empowered with a clear mandate The stakeholder network will operate under a clear and well-defined mandate that coordinates its role in the joint scientific consultations (JSCs) and joint clinical assessments (JCAs) within the HTA framework. Their contributions to these processes in cooperation with the EMA and the HTA bodies will extend beyond mere consultation and observer roles. Their function will be key to understanding and emphasizing public health considerations. This decisive involvement ensures that health technologies are evaluated effectively and efficiently, reflecting the most pressing public health needs. The mechanisms for coordinating the stakeholder group by an independent panel and the procedural rules for voting internally would need to be determined by the Commission. The formation of separate stakeholder subgroups that are disease-specific would also be possible. The involvement of clinical experts from the stakeholder network in JCAs throughout the scoping process, in addition to the scientific consultations, would also be possible. Our legal proposal is available in the attached file.
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Meeting with Stella Kyriakides (Commissioner) and

11 Jun 2024 · Meeting on Europe’s Beating Cancer Plan and EU Policy on cancer; research and treatment optimisation

Meeting with Catherine Amalric (Member of the European Parliament, Shadow rapporteur)

20 Feb 2024 · Révision de la législation pharmaceutique

Response to Evaluation and revision of the general pharmaceutical legislation

30 Oct 2023

The European Organisation for Research and Treatment of Cancer (EORTC), with a mission to design, coordinate, and conduct multidisciplinary clinical and translational trials to improve the standard of cancer treatment for patients, welcomes the proposal to review the EU general pharmaceutical legislation. Many drugs enter the market without evidence of their true clinical benefit for patients. Regulatory clinical trials aimed at identifying and qualifying new drugs should be complemented with independent clinical research, studying the optimal way to use the drugs. Treatment optimisation (TO) studies are clinical trials that address questions (dosage, treatment duration, treatment combinations, etc.) that are not usually investigated in industry led clinical research. Optimising the use of therapies in clinical practice should produce similar therapeutic benefits but fewer toxicities for patients, while also generating significant cost savings for healthcare systems. To achieve this purpose, the EORTC proposes the amendment of the provision under Article 12 Committee Opinion, part 4(h): 4. If an opinion is favourable to the granting of the relevant marketing authorisation, the following documents shall be annexed to the opinion: (h) where appropriate, details of any recommended obligation to conduct any other post-authorisation studies post-authorisation treatment optimisation studies to improve the safe and effective use of the medicinal product. Such an obligation to conduct such studies shall take into account the scientific guidance referred to in Article 123 of [revised Directive 2001/83/EC];
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Response to Evaluation and revision of the general pharmaceutical legislation

25 May 2023

The EORTC believes the revision of the general pharmaceutical legislation provides with an opportunity to introduce the concept of treatment optimisation into the legislation. What is treatment optimisation? Many drugs enter the market without evidence of their true clinical benefit for patients. Regulatory clinical trials aimed at identifying and qualifying new drugs should be complemented with independent clinical research, studying the optimal way to use the drugs. Treatment optimisation (TO) studies are clinical trials that address questions (dosage, treatment duration, treatment combinations, etc.) that are not usually investigated in industry led clinical research. Optimising the use of therapies in clinical practice should produce similar therapeutic benefits but fewer toxicities for patients, while also generating significant cost savings for healthcare systems. How to implement TO research into the framework for developing new treatments? In 2019, the EORTC carried out a study for the Scientific Foresight and Innovation Panel of the European Parliament. Three policy options were identified through which TO research could be implemented. Today, the EORTC is proposing to implement the following policy option in the proposal for a pharmaceutical regulation. Policy option: Implementing TO research post-marketing authorisation The revised pharmaceutical legislation should incorporate the following provisions possibly in Article 20: Imposed post-authorisation studies: 1) The obligation for the marketing authorisation holder to submit TO data to the EMA within certain time limit. 2) The obligation for the marketing authorisation holder to perform a TO clinical trial in collaboration with an independent not-for-profit research organisation able to develop and peer-review the clinical trial protocol, and to collect and analyse independently the clinical trial data. The drug owner will have the obligation to provide all relevant information regarding the drug and financial support to the independent non-profit research organisation. 3) The EMA will have the responsibility to develop an appropriate guideline regarding the design, conduct, and analysis of TO clinical trials. A major advantage of this strategy is that it does not impede patient and clinician access to new therapies. Once the initial marketing authorisation has been awarded, and the national reimbursement application process has been successfully completed, the treatment will be available for use in clinical practice in that particular country. In the meantime, the manufacturer could set up partnerships with independent research institutions for the conduct of TO studies. Additionally, to counter the argument of increased development costs, these TO trials could be funded by the revenue generated by the company from the sale of their product during this period and/or through public funding when treatment optimisation studies generate savings for healthcare systems. Moreover, since the original decision to approve the therapy is revisited after a certain amount of time, the results of these studies would have a direct impact on regulatory decision-making. This strategy is also in line with the adaptive pathways model designed by EMA.
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Meeting with Véronique Trillet-Lenoir (Member of the European Parliament)

14 Nov 2022 · Right to be forgotten

Meeting with Véronique Trillet-Lenoir (Member of the European Parliament)

10 May 2022 · Health Data and research

Meeting with Tove Ernst (Cabinet of Commissioner Stella Kyriakides)

11 Mar 2022 · Discussion on Europe's Beating Cancer Plan and breast cancer

Response to Evaluation and revision of the general pharmaceutical legislation

14 Apr 2021

EORTC would like to stress several aspects that would deserve specific actions: Access to medicines is definitively a key aspect. The affordability of medicines has implications for both public and household finances. EORTC fully endorses the Commission statement that minimising waste and optimising the value of spending on medicines are also critical to achieving efficient and sustainable health systems. Many drugs enter the market without evidence of their true clinical benefit for patients. There is a risk of exposing patients to poorly documented and expensive treatments. Regulatory clinical trials aimed at identifying and qualifying new drugs should be complemented with independent clinical research, studying the optimal way to use the drugs: such as dosage, treatment duration… Improved knowledge on the use of multidisciplinary treatment strategies is a healthcare priority. Europe should foster the integration of clinical research, free of commercial interest, in the process of access to treatments to inform healthcare systems. EORTC suggests a transparent and independent multi-stakeholder process through which public health priorities are assessed as well as the needs for treatment optimisation studies taking into account the public health priorities, the profile of the drugs and their impact on public resources. EORTC is very interested by the Commission proposal to revise the pharmaceutical legislation to encompass new methods of evidence generation and assessment, such as analysis of big and real world data to support the development, authorisations and use of medicines. EORTC welcomes the statement clarifying that the main source of evidence for the authorisations of innovative medicines should remain robust clinical trials with suitable comparators reflecting the standard of care in the EU. It is critical to realize there is a continuum of data that should be used for appropriate purpose in full understanding of limitation of the different types of data. Real-world data can definitively support investigation on treatment toxicity or pattern of use but real-world-data can’t inform about the efficacy of a therapy. Furthermore, it makes no sense to oppose clinical trials with other sources of real-world data such patient files and registries. Indeed, pragmatic clinical trials where the treatment is prescribed and used as in normal daily practice have to be considered as generating real world data. Treatment optimization study should be considered as pragmatic trial. As pointed out by the Commission, commercial interest is often weaker for treatment optimization studies, so they are mainly organized by academia, where the price of trial medication and insufficient regulatory knowledge can be bottle-necks. EORTC believes that the proposed action is not well designed since it is limited to support and training in regulatory science. What is needed is rather public financial support for performing the needed treatment optimization studies without compromising independency. Moreover, many academia and not-for-profit organisations do have the needed expertise in regulatory science. Horizon Europe, Cancer Mission and Public-Private-Partnership such the Innovative Health Initiative could support international independent research consortia conducting treatment optimization studies. EORTC learned with great interest the Commission’s proposal to revise legislation to give regulatory authorities more power to adapt on their own initiative the terms of marketing authorisations on the basis of scientific evidence. After EMA approval, national authorities decide on how medicines are introduced in their health systems, so an effective mechanism must induce both the EU and national mechanisms to take account of treatment optimization studies. Furthermore, treatment optimization study should be compulsory and fully part of the path to the patient access to treatment.
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Meeting with Stella Kyriakides (Commissioner) and

24 Feb 2021 · Exchange of views on Europe’s Beating Cancer Plan, the EU Pharmaceutical Strategy and the availability of medicines.

Response to A European Health Data Space

26 Jan 2021

Generally speaking, creating an EU environment where data are available, accessible and interoperable can only be welcome. Data are complex and need to be informative on the disease, the host and their respective evolution overtime. Data are not static must should be seen as dynamic set of varying inter-dependent information. The multidimensionality of health data (clinical, biological, medical imaging) related to the disease but also the host leads to different types of challenges at any time-point where data could be looked at. This multidimensionality of data should also be taken in its longitudinal dimension as the disease evolves and the host ages. Central to therapeutic progress and care is to understand the curability of diseases and/or their natural evolution over time, but also how treatments can influence favorably of unfavorably the evolution of diseases. This can only be achieved by science going back to patients and hypothesis generating and data are the vehicle of this process. Any initiative addressing data for health should therefore be patient centric and intended to push the boundaries of care and treatment options, not only for individual patients but also for collective decisions on access to therapeutic options. This impacts substantially on how data should be structured and how knowledge may be returned to patients, this beyond the simple data portability for isolated decision but further towards the capacity to generate easily meaningful datasets providing evidence for the majority. This patient centric vision and ultimate objective including clinical validation of findings is crucially missing in this document. Developing new solutions alongside the development of AI, development of digital health services and products will be needed but they should remain at the level of tools and vehicles and not be an objective per se. Therefore, it is unclear how this will be developed to serve the needs of patients in health care systems. For example, “re-use” of data is mentioned several times in the document and is actually intimately linked to the longitudinal dimension and versatility of data over time so critical to researchers to help patients as the disease evolve. In such case, matching the data, specially the biological data, as they were at a previous state of the disease and compare to a future state of the disease may help finding correlation in patterns of relapse and/or some biological features which may indicate how treatments influence the patterns of disease evolution. In such case, the interplay with GDPR, which is indicated in several places, is crucial to be tackled as it implies that pseudo-anonymization should apply and not full anonymization. Thus, the ultimate application of the EHDS should drive the process of building it. Statements like “optimising treatment options”; “increasing the capability of researchers”; “support research on effective treatments” can only come true if this objectives and their challenges are well identified, then this can helps transforming the regulatory environment to enable gathering efficiently meaningful datasets. It should lead to re-engineering the full chain of the process from patient consent at initial diagnosis and throughout the patient lifetime . This is specifically relevant for chronic diseases. Therefore, reading that the commission will continue to assess the horizontal framework for AI and may consider for a specific sectoral policy initiative appears not only as an under estimated statement but should actually be considered as part of the initial diagnosis and needs to be addressed for any future EHDS. The objectives of EHDS remain unclear: is it just to facilitate exchange of datasets or is it for researchers to optimise use of data to develop new knowledge. Ways to reach to these capabilities through EHDS may have been underestimated.
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Meeting with Tove Ernst (Cabinet of Commissioner Stella Kyriakides)

3 Nov 2020 · Exchange of views on Europe’s Beating Cancer Plan

Response to Pharmaceutical Strategy - Timely patient access to affordable medicines

29 Jun 2020

The EORTC welcomes the roadmap for a pharmaceutical strategy for Europe. The development of better treatments is critical for European citizens. The development of new molecules supporting precision medicine should be fully aligned with societal needs. Patients should be at the center. Market access should be supported by robust evidence about the clinical utility of the new drugs. Furthermore, the use of new drugs should be optimized by filling the knowledge gaps in the understanding on how to use optimally new drugs in health care systems through independent and well-designed research. Several academic and HTA papers have highlight concerns that approved expensive new drugs especially in oncology may not provide substantial added benefits to patients. Today there is no efficient process in Europe to recognize truly innovative treatments and bring them efficiently to patients. This can be partially explained by the gap between regulatory clinical trials for the approval of drugs and their use in the clinic. Addressing treatment duration, dosage and combination of therapies through independent trials designed to optimize the use of treatments in health care systems is a critical missing link to bring proportionate, risks based affordable treatments to all. Using efficiently public resources is key for ensuring access to innovation for European citizens. Regulatory clinical trials aimed at identifying and qualifying new agents are needed but not sufficient to inform health care systems. They should be complemented with independent clinical research studying the optimal way to use the drugs. Improved knowledge on the use of multidisciplinary treatment strategies is a health care priority. Optimisation clinical studies addressing the use of new drugs should already be underway at the time of market approval. Continuity from drug registration into health care research should be mandatory. To achieve this, Europe should be equipped with a multidisciplinary, patient centered, free of commercial interest, evaluation process identifying key questions to validate innovation and its use in health care systems based on robust and meaningful clinical endpoints. This was highlighted in the EORTC manifesto widely supported by stakeholders (https://www.eortc.org/app/uploads/2020/05/Manifesto-29052020.pdf). Key opinion leader clinicians, epidemiologists, patients, scientific societies and other stakeholders such HTA and payers should define, independently from the industry, what are the public health priorities and which drugs need to be optimised. Industry should contribute to the efforts once the priorities are defined. International collaboration is key since public resources are scarce and should be pooled to efficiently address common public health issues. EORTC suggests a transparent and independent multistakeholder process (State of Science in Care https://cancerworld.net/voices/eortc-and-ecco-push-for-the-state-of-science-in-care/) through which public health priorities are assessed as well as the needs for treatment optimisation research taking into account the public health priorities, the profile of the drugs and their impact on public resources. Such multistakeholder process should be part of the pharmaceutical strategy for Europe. The European Organisation for Research and Treatment of Cancer (EORTC) is the largest independent cancer clinical research organization. The EORTC has delivered investigator driven, changing practice clinical trials since 1962. Its mission is to improve survival and quality of life of cancer patients. The EORTC is a not-for-profit independent organisation performing multidisciplinary clinical research activities across tumor types. EORTC’s activities involve on a voluntary basis more than 5300 doctors and scientists from 930 university hospitals in 36 countries. EORTC clinical trials are involving several thousands of patients on a yearly basis. More information at https://www.eortc.org
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Response to Europe’s Beating Cancer Plan

21 Feb 2020

The EORTC welcomes the roadmap high-level objectives of improving the quality and accessibility of cancer treatments. Collaborative clinical research to maximise impact and knowledge translation into new therapies and clinical practice is a key European bottleneck for access to all. It is central to EORTC contribution here in. Filling the knowledge gaps in the understanding of cancer and optimized treatments in health care systems through independent and well-designed research is critical for access to treatments for all citizens and should be supported by the Cancer Mission. Several academic and HTA papers have highlight concerns that approved expensive new drugs may not provide substantial added benefits to patients. Today there is no efficient process in Europe to recognize truly innovative treatments and bring them efficiently to patients. This can be partially explained by the gap between regulatory clinical trials for the approval of drugs and their use in the clinic. Addressing treatment duration, dosage and combination of therapies through independent trials designed to optimize the use of treatments in health care systems is a critical missing link to bring proportionate, risks based affordable treatments to all. For example, today, despite the innovative use of Immuno-oncology , duration of treatment is not addressed despite its high costs and potential side effects. Direct and indirect savings which could be made by shorter treatments could help a more rationale access to innovative drugs. Using efficiently public resources is key for ensuring access to innovation for European citizens. Currently, drug owners are not incentivised to perform the needed post authorisation research and investigating the optimal use of their products. This will require concertation for independent research in health care systems across Member States. Regulatory clinical trials aimed at identifying and qualifying new anti-cancer agents are needed but not sufficient to inform health care systems. They should be complemented with independent clinical research studying the optimal way to use the drugs. Improved knowledge on the use of multidisciplinary treatment strategies is a health care priority. Optimisation of clinical studies addressing the use of new treatments should already be underway at the time of market approval. Continuity from drug registration into health care research should be mandatory. To achieve this, Europe should be equipped with a multidisciplinary, patient centered, free of commercial interest, evaluation process identifying key questions to validate innovation and its use in health care systems based on robust and meaningful clinical endpoints. This was highlighted in the EORTC manifesto widely supported by stakeholders (www.eortc.org/manifesto). Key opinion leader clinicians, epidemiologists, patients, scientific societies and other stakeholders such HTA and payers should define, independently from the industry, what are the public health priorities and which treatments need to be optimised. Industry should contribute to the efforts once the priorities are defined. International collaboration is key since public resources are scarce and should be pooled to efficiently address common public health issues. EORTC suggests a transparent and independent multistakeholder process (state of science of cancer treatment optimisation) through which public health priorities are assessed as well as the needs for treatment optimisation research taking into account the public health priorities, the profile of the drugs and their impact on public resources. The EORTC is the largest not-for-profit independent cancer clinical research organization. Since 1962, its mission is to improve survival and quality of life of cancer patients. The EORTC is performing multidisciplinary clinical research across tumor types. EORTC’s activities involve 5300 doctors and scientists from 930 university hospitals in 36 countries.
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Meeting with Anne Bucher (Director-General Health and Food Safety)

19 Feb 2020 · challenges of documenting robust evidence for the use of cancer treatments

Meeting with Stella Kyriakides (Commissioner) and

5 Feb 2020 · Discussion on Cancer and Europe's Beating Cancer Plan

Response to European Partnership for innovative health

27 Aug 2019

Several authors have demonstrated that the majority of the new cancer agents do not translate into a clinically meaningful benefit for patients such as survival or quality of life. Nevertheless, the vast majority of these agents do get on the market while limited information is known on such agents including the safety profile. Once on the market, the commercial sector may not actively follow up with clinically robust studies which inform how to integrate new agents in existing therapeutic strategies, how to sequence, combine, and document the optimal duration of treatment. Documentation of the exact dose, or even of the ultimate patient population to benefit often need optimisation studies as well. Earlier access to market in absence of optimisation of new agents contributes to feed the gap efficacy-effectiveness. Caution is needed to claim innovation too early exposing patients and society to poorly documented and expensive treatments. EORTC advocates for a continuum of structured clinical research from drug development into health care. Access to affordable and appropriately documented treatments is the challenge for which new parameters have to be defined, based on patient and society centricity. New approaches sequencing drug development, treatment optimisation and real world monitoring need to be set up through the chain of involved stakeholders. In that respect, EORTC has initiated a European discussion through the manifesto, presented at the EU parliament and endorsed by many organisations. A major transformation of clinical research building on the strengths and complementarity of stakeholders working alongside new business models must be tackled. a) Patients triage (molecular screening) and trial access lead by the non-commercial sector: academia in partnership with pharma, biotech, diagnostics etc. b) Drug development lead by the commercial sector: industry in partnership with academia. c) Therapeutic optimization lead by the non-commercial sector: academia in partnership with HTA and payers. d) Real life implementation and long term monitoring of treatments led by the non-commercial sector: academia in partnership with registries, HTA and payers. What could the European Partnership for Innovative Health do? The new partnership should support the reengineering of medicine development: - The partnership should fund more projects involving patient screening platform aiming at in-depth understanding of disease biology and the mechanisms of drug action but also improved patient access to the right clinical trials according to patient biology. Such platform should incorporate massive molecular screening efforts, enabling high quality samples and clinical data collection, bioinformatics and knowledge distribution coupled with long-term longitudinal clinical follow-up of patients. - The partnership should enable treatment optimisation studies at large scale involving the industry, academia, patients, HTA and payers. Treatment optimisation studies are pragmatic prospective clinical trials randomized as much as possible in unselected patient population. Such studies aim to generate the evidence on the optimal way to use the drug in real-life i.e. treatment duration, dosage and combination with other treatment modalities. Optimisation studies complement drug development regulatory clinical trials and bridge the so-called efficacy- effectiveness gap. Treatment optimisation studies should attempt as much as possible to generate level 1 evidence that will help clinicians to update treatment guidelines, HTA to assess the value of the drug and payers to decide the reimbursement.
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Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis)

3 May 2018 · HTA

Meeting with Xavier Prats Monné (Director-General Health and Food Safety)

16 Apr 2018 · Discussion Cancer Research

Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis)

5 May 2017 · Clinical research