Cancer as a pathfinder
The burden of cancer is increasing in the EU. The estimated number of new cancers diagnosed in 2022 was 2.7 million, and 1.3 million people lost their lives to itiAn overview of the cancer disease burden in the EU is available in the European Cancer Information System. Cancer is the second leading cause of mortality after cardiovascular disease. However, there still remains an area of high unmet medical need in Europe. In 2023, EMA launched a new initiative to help develop cancer medicines and build up valuable expertise in line with the aims of the Europe’s Beating Cancer Plan.
For many years now, oncology has been the therapeutic area that has had the highest number of new approvals. In 2023 alone, the CHMP gave positive opinions to 25 new cancer medicines, which represents one third of all the new medicines reviewed positively by EMA. But there are still many unmet medical needs to address.
In the last few years, significant breakthroughs such as targeted therapies and immunotherapies have transformed the treatment of many types of cancer, including more common cancers like breast cancer, lung cancer and less frequent cancers like leukaemias.
In 2023, EMA launched a new initiative, called ‘Cancer Medicines Pathfinder’, to further support the development and approval of cancer medicines which could have a meaningful impact on transforming patient care.
“The Cancer Medicines Pathfinder builds strategically on the EU network’s experience during the pandemic and leverages available tools and processes to proactively help advance and refine cancer treatments.”
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Francesco Pignatti presents the Cancer Medicines Pathfinder initiative at EMA’s press briefing on human medicines highlights.
This new initiative explores how EMA can improve medicine evaluation overall, including by applying some of the learnings from the COVID-19 pandemic. Cancer was chosen for this initiative because it is a therapeutic area with a high rate of innovation and scientific progress, but also a high unmet medical need. Ultimately, this work will pave the way for other therapeutic areas, to support patients’ swift access to the most innovative therapies.
The Cancer Medicines Pathfinder rests on three pillars: accelerating assessments of medicines, strengthening dialogue with stakeholders and communicating benefits and risks.
Accelerating assessment of medicines critical to patient care
In 2023, EMA explored how it can further expedite and refine its processes in the evaluation of cancer medicines which have a significant potential to address unmet medical needs, focussing on those designated as PRIME (PRIority MEdicines) products. The objective is to maximise efficiency in the assessment of medicines critical to patient care, while keeping the high-quality standards applied to all the medicines approved. EMA took further steps to guide developers in their submissions and build capacity within the multinational assessment framework. It has started to analyse challenges in the development and submissions to inform new developments, focussing on conditional approval and real-world data as an external comparator.
Building oncology expertise across Europe
EMA has re-shaped its expert communities through the Oncology European Specialised Expert Community (ESEC), a platform to promote information sharing among European experts on scientific and regulatory topics.
The ESEC is composed of over 150 experts: assessors working for a National Competent Authority (NCA), members of EMA working parties and advisory groups, and European experts with a special interest or expertise in oncology. Its tasks include mapping competences across the European network of experts, providing training and upskilling to the European medicines regulatory network and connecting experts from multidisciplinary teams and across topics of expertise. In March 2023, EMA organised a dedicated webinar for ESEC experts on challenges in drug development, regulation and clinical practice in Acute Myeloid Leukaemia.
In addition, EMA took steps to further enable clinical-oncology scientists to participate in medicines regulation. In May 2023, EMA launched a pilot educational program in oncology which consists of live and recorded webinars that provide an overview of the regulatory requirements for medicines’ evaluation, with a focus on oncology. The general training curriculum was delivered in 2023 and a specific curriculum for oncology is planned for 2024. One of the goals of this pilot is to encourage scientists to contribute to regulatory activities, should the opportunity arise and, more generally, to increase collaboration between regulatory authorities and stakeholders in healthcare and academia.
Strengthening multi-stakeholder dialogue and international collaboration
EMA has a long track record of multi-stakeholder dialogue in cancer, involving researchers, developers, payers, patients, and other regulators. These partnerships were further strengthened in 2023 to enhance research and access to innovative treatments.
EMA continued its work through the Cancer Medicines Forum, established in 2022 in collaboration with the European Organisation for Research and Treatment of Cancer (EORTC). In the Forum, academics and regulators can discuss specific challenges around research into optimising cancer treatments. The Forum met three times in 2023. Topics discussed included experience with pragmatic trials and real-world data, which treatment optimisation questions should be prioritised, and how EMA can support efforts to optimise treatments with oncology medicines.
Addressing unmet medical needs often requires a global effort. EMA has a lot of experience with international collaboration in oncology, e.g. through the ‘cluster’ meetings with SwissMedic, the US FDA, Health Canada, the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), the Australian Therapeutic Goods Administration (TGA), which are organised each month to share clinical review information. In 2023, EMA reinforced global collaboration with the view to further expand the level of interactions earlier in the assessment.
Conversations on Cancer: hearing the patient’s voice
EMA works closely with patients to ensure their views and preferences are understood and taken on board in the evaluation of medicines. In October 2023, EMA joined for the first time as a co-organiser the FDA’s Conversations on Cancer, a public panel discussion. For the first edition of this new joint initiative, a range of speakers from the US and the EU highlighted the day-to-day, year-to-year experience of patients living with metastatic breast cancer.
Communicating benefits and risks with patients
Patients and healthcare professionals need access to clear information to make informed decisions. In 2023, EMA continued to explore ways to refine its approaches to ensure the benefits and risks of cancer medicines are not only better assessed, but also better explained and communicated. This goes hand in hand with efforts to improve evidence generation in the regulatory process.
Actions include structured templates for benefit risk communication, advancing endpoint methodology to measure the benefits of cancer drugs using different sources of data, exploring the role of patient preference studies to inform regulatory decisions, and new international guidance on patient preference studies. Work in these areas is expected to continue during 2024.
Perception of progression-free survival as a clinical endpoint
In December 2023, academics and EMA experts published an article in the European Journal of Cancer which describes the attitudes of healthcare professionals and drug regulators to progression-free survival (PFS) as an efficacy endpoint in clinical trials with patients with advanced cancer. It also explores to what extent these attitudes influence the willingness to trade between PFS and toxicity. The study highlighted the need to improve communication to patients about meaning, strengths and limitations of improvements in PFS.