ESMO 2023 – Personal takeaways

For the last few days, I’ve been reflecting on my experience at this year’s ESMO conference, and here I am sharing some of the thoughts and insights that have had the most impact on me.

Aside from the important findings from various clinical trials that will lead to changes in how cancer patients are treated, I was particularly inspired by the presentations of patient advocates, policy experts, and clinicians who addressed some of the current challenges and opportunities related to wider access to innovative molecular approaches and therapies.

Investing in Learning Health Care Systems

It is crucial that we invest more in the development and promotion of patient-centered learning health care systems. These systems integrate scientific and technological advancements, policy, legal, social, and ethical aspects into a dynamic ecosystem (1). In this regard, comprehensive and standardized collection of high-quality real-world data (RWD) is a key, as RWD can then be utilized for education, digitalization, scientific collaboration and other beneficial purposes. Good quality RWD can also contribute to clinical drug development by serving as a control arm in trials using single-arm approaches, as well as during regulatory submission-decision making by FDA and EMA. However, as it has been presented during this year’s ESMO, currently available real-world evidence (RWE) is heterogenous and of insufficient quality (2). Therefore, the harmonization of clinical and genomic data collection efforts is warranted (3) and this is why strategic initiatives such as ESMO Guidance for Reporting Oncology Real-World evidence (GROW) will be a key for a systematic collection of RWD.

Addressing Clinical Trial Design Challenges and Opportunities

We must ensure that the development of clinical trials is driven by the unmet needs of patients rather than commercial interests (4-7). We have been reminded that less than 20% of randomized clinical trials (RCTs) for major solid tumors meet the threshold for clinically meaningful benefit and only about 50% of FDA and EMA approved drugs have been shown to improve patients’ overall survival (OS) or quality of life. This signifies the need for stricter guidelines when designing future RCTs. With that in mind, trials like Optimus (8) and DESTINY-Lung02, which focus on optimizing drug dosages in oncology, are crucial in this regard. By testing minimal effective doses (MED) as opposed to maximally tolerated doses (MTD), these trials aim to improve patients’ quality of life while also enhancing drug development programs. The results of these trials and their impact on patient toxicity, tolerability, and overall survival will be eagerly anticipated.

When discussing side effects, we must also consider their impact on mental health and fertility, particularly for young adult cancer patients. Further research in these areas is greatly needed, and it is encouraging to know that the EU has initiated several grants to address these concerns.

We need to do more about the awareness

Despite the staggering number of new cancer cases reported in 2020 (19 million), which means that one in five individuals will face cancer at some point in their lives, there is still a significant need for increasing awareness about this devastating disease. The COVID-19 pandemic served as a reminder of the importance of public health crises, leading to the rapid development of vaccines and other innovations. We should strive for the same level of awareness and urgency in the fight against cancer by promoting screening, early detection, and prevention initiatives. Currently, only 16% of countries meet the WHO guidelines for cancer screening programs, emphasizing the necessity for education and awareness campaigns. Improving socioeconomic aspects such as facilitating access and infrastructure for these tests is also crucial. Additonally, early detection not only improves survival rates but also reduces the economic burden (between 2 to 4-fold) associated with advanced-stage cancer treatment (9).

Controversies and Opportunities in Clinical Trial Design

During the discussions at ESMO, traditional endpoints like progression-free survival (PFS) were called into question by some presenters. While PFS is often used as a primary endpoint to assess the efficacy of a drug, its significance depends on weighing the improved or delayed symptoms against any added toxicity. The patient advocates rightly raised concerns about the tolerability of certain drugs. What about drugs that showed improvement in PFS but failed to impact overall survival? What about the costs of these drugs? Some presenters suggested that overall survival (OS) and quality of life (QoL) should be prioritized as primary endpoints in clinical trials. For slower-progressing tumors, early changes in PFS can still be valuable. However, FDA and EMA approvals should require a larger difference in PFS and reconsider the approval if there are no subsequent improvements in OS or QoL. Evaluating the clinical value of a drug can be aided by utilizing the ESMO magnitude of clinical benefit scale (MCBS).

Few speakers discussed whether new endpoints should be considered in clinical trials such as ctDNA clearence in advanced setting, stage shift in early detection and in general should toxicity levels be replaced by tolerability? All interesting areas which we will see how they evolve over coming months.

Patient Perspectives

Patients desire easily accessible and understandable information. This necessitates simplifying informed consent and using patient-friendly terminology throughout clinical trial processes. Patient preferences should be taken into account before and during the study. Digitalization and new technologies have shown promise in predicting toxicity, recurrence, and prognosis as presented by a Swedish nurse. These advancements can also support healthcare providers, particularly in the face of workforce reductions in nursing. Quality of life, including drug tolerability, remains a fundamental consideration for patients. The concept of toxicity should be re-evaluated and possibly redefined to better address patient-specific needs.

Biomarker Access

In terms of genomic testing, there is still a significant variation in approaches and turnaround times, even for the testing of mandatory biomarkers like EGFR in non-small-cell lung cancer (10). For instance, genomic aberrations including ROS1, that should be evaluated in NSCLC patients, is tested in 67% and 56% of cancer patients in France and Spain respectively, showing still a significant portion of patients not being tested for this biomarker that could potentially help in a treatment decision making (11).

Additionally, there has been shown a poor uptake of the genomic scales for tiering of genomic variants according to their actionability such as ESCAT, highlighting a need for further education amongst oncologists around this topic (12). This recently published study by ESMO confirmed that classical single-gene techniques are widely available across Europe, whereas access to advanced biomolecular technologies such as next-generation sequencing (NGS) panels and testing of more complex biomarker is highly heterogeneous. The biggest barriers to adoption of multigene testing approaches are the reimbursement of the test and the availability of a suitable treatments.

Oncologists would benefit from further education on the importance of multigene testing approaches using NGS panels and tiered genomic variant testing. Reimbursement and treatment availability are additional important barriers to the widespread adoption of these advanced biomolecular technologies.



1.       Menear, M. et al. A framework for value-creating learning health systems. Health Res Policy Syst. 2019 Aug 9;17(1):79.

2.       Boyle, J.M. et al. Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study. Eur J Cancer. 2021 Sep;155:136-144.

3.       A. Pellat et al. Comprehensive mapping review of real-world evidence publications focusing on targeted therapies in solid tumors: A collaborative work from ESMO real-world data and Digital Health Working Group. Abstract 1689O ESMO 2023.

4.       Christopher M.Booth, Ajay Aggarwal and Richard Sullivan. Commercial Determinants of Cancer Medicine. Eurohealth 2022; 28(2).

5.       Richard Sullivan, Christopher M.Booth and Ajay Aggarwal Non-pharmaceutical technologies in cancer care: For profit or for patients? Eurohealth 2022; 28(2).

6.       Henderson, R.H. et al. Delivering the precision oncology paradigm: reduced R&D costs and greater return on investment through a companion diagnostic informed precision oncology medicines approach. J of Pharm Policy and Pract 16, 84 (2023).

7.       Del Paggio J.C. et al. Do Contemporary Randomized Controlled Trials Meet ESMO Thresholds for Meaningful Clinical Benefit? Ann Oncol. 2017 Jan 1;28(1):157-162.

8.       Araujo, D. et al. Oncology phase I trial design and conduct: time for a change – MDICT Guidelines 2022. Ann Oncol. 2023 Jan;34(1):48-60.


10.   Hofman P. et al. Real-world EGFR testing practices for non-small-cell lung cancer by thoracic pathology laboratories across Europe. ESMO Open. 2023 Oct;8(5):101628.

11.   Salas C. et al. Real-world biomarker testing rate and positivity rate in NSCLC in Spain: Prospective Central Lung Cancer Biomarker Testing Registry (LungPath) from the Spanish Society of Pathology (SEAP). J Clin Pathol. 2022 Mar;75(3):193-200.

12.   Bayle A. et al. ESMO study on the availability and accessibility of biomolecular technologies in oncology in Europe. Ann Oncol. 2023 Oct;34(10):934-945.

Disclaimer: The information in this blog is not intended or implied to be a substitute for professional medical advice, diagnosis or treatment. All content available here is for general information purposes only.