EMA, EORTC, and SIOG discussed drug development challenges for older adults, Amsterdam on 9 April 2025 (18/04/2025)

EMA, EORTC, and SIOG discussed drug development challenges for older adults, Amsterdam on 9 April 2025. The focus was on supporting drug development and optimisation activities to better meet the needs of this key population. Read the editorial.

CHMP highlights (oncology) April 2025: Summary table (01/04/2025)

CHMP highlights (oncology) April 2025: Summary table

Ziihera (zanidatamab), applicant: Jazz Pharmaceuticals Ireland Limited (03/03/2025)

Ziihera (zanidatamab), applicant: Jazz Pharmaceuticals Ireland Limited

Dazublys (trastuzumab), applicant: CuraTeQ Biologics s.r.o (30/04/2025)

Dazublys (trastuzumab), applicant: CuraTeQ Biologics s.r.o

Denbrayce (denosumab), applicant: Mabxience Research SL (30/04/2025)

Denbrayce (denosumab), applicant: Mabxience Research SL

Enwylma (denosumab), applicant: Zentiva k.s. (30/04/2025)

Enwylma (denosumab), applicant: Zentiva k.s.

Vevzuo (denosumab), applicant: Biosimilar Collaborations Ireland Limited (30/04/2025)

Vevzuo (denosumab), applicant: Biosimilar Collaborations Ireland Limited

Yaxwer (denosumab), applicant: Gedeon Richter Plc. (30/04/2025)

Yaxwer (denosumab), applicant: Gedeon Richter Plc.

Adcetris (brentuximab vedotin), MAH: Takeda Pharma A/S (30/04/2025)

Adcetris (brentuximab vedotin), MAH: Takeda Pharma A/S

Calquence (acalabrutinib), MAH: AstraZeneca AB (30/04/2025)

Calquence (acalabrutinib), MAH: AstraZeneca AB

How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions? (02/05/2025)

Read the editorial about the recent publications on strategies for measuring patient's quality of life and for ensuring the patient voice is incorporated in the decision making process.

Concept paper on the revision of the guideline on the evaluation of anticancer medicinal products and its appendices (18/04/2025)

The Revision 7 aims to keep the guideline aligned with evolving regulatory and scientific developments and to implement the estimands framework introduced by ICH E9(R1) addendum. Other changes include the addition of sections dedicated to haematological cancers, a revision of the guidance on single-arm trials, and on clinical trials in adjuvant, neoadjuvant and perioperative settings.

Tackling vulnerabilities in the supply chain of radiopharmaceuticals in the EU (18/04/2025)

EMA and the Heads of Medicines Agencies have issued recommendations to address vulnerabilities in the supply chain of radiopharmaceuticals. The use of these radioisotopes, used to diagnose or treat medical conditions such as cancer is steadily increasing, while manufacturing capacity in Europe is limited.

HMA-EMA Catalogues of real-world data sources and studies (18/04/2025)

The HMA-EMA RWD Catalogues are repositories of metadata collected from real-world data (RWD) sources and RWD studies. They are intended to help regulators, pharmaceutical companies and researchers to identify and use such data when investigating the use, safety and effectiveness of medicines.

Second EMA / Alliance for Regenerative Medicine bilateral meeting - 8 May 2025 (08/05/2025)

This meeting between European Medicines Agency (EMA) and Alliance for Regenerative Medicine (ARM) is organised in the context of the EMA annual bilateral meetings with industry stakeholder organisations. The purpose of the meeting is to exchange views and promote dialogue on topics of common interest.

Mandatory use of ISO/ICH E2B(R3) individual case safety reporting in the EU: hands-on training course using the EudraVigilance system - 6 June 2025 (06/06/2025)

Training on EudraVigilance which is the EU's system for managing and analysing information on adverse reactions to medicines which have been authorised or are being studied in clinical trials in the EEA and supports the reporting and analysis of suspected adverse reactions originating from clinical trials and the post-authorisation phase of medicinal products.

Cancer Medicines Forum (CMF) - Three years' review (01/04/2025)

Setting up in March 2022, the CMF was created by EMA in collaboration with the European Organisation for Research and Treatment of Cancer (EORTC) with the overall goal to optimise cancer treatments. Treatment optimisations’ proposals by the EHA, ESMO, and EORTC, include both general study ideas and specific study concepts.

CHMP highlights (oncology) March 2025: Summary table (01/04/2025)

CHMP highlights (oncology) March 2025: Summary table

Jubereq (denosumab), applicant: Accord Healthcare S.L.U. (01/04/2025)

Jubereq (denosumab), applicant: Accord Healthcare S.L.U.

Bosulif (bosutinib), MAH: Pfizer Europe Ma EEIG (01/04/2025)

Bosulif (bosutinib), MAH: Pfizer Europe Ma EEIG

Calquence (acalabrutinib), MAH: AstraZeneca AB (01/04/2025)

Calquence (acalabrutinib), MAH: AstraZeneca AB

Opdivo (nivolumab), MAH: Bristol-Myers Squibb Pharma EEIG (01/04/2025)

Opdivo (nivolumab), MAH: Bristol-Myers Squibb Pharma EEIG

Tevimbra (tislelizumab), MAH: BeiGene Ireland Ltd (01/04/2025)

Tevimbra (tislelizumab), MAH: BeiGene Ireland Ltd

Pemazyre (pemigatinib), MAH: Incyte Biosciences Distribution B.V. (01/04/2025)

Pemazyre (pemigatinib), MAH: Incyte Biosciences Distribution B.V.

Biomarkers and Companion Diagnostics in Oncology (04/04/2025)

The landscape of in vitro diagnostics in the European Union has undergone a significant transformation with the implementation of the In Vitro Diagnostic Regulation (IVDR), which came into full effect in May 2022. More stringent requirements have been introduced to ensure the safety and performance of IVDs, including companion diagnostics.

Electronic product information (ePI) (03/04/2025)

A draft reflection paper on patient access to electronic product information (ePI) is available for public consultation. It outlines a proposal for accessing the ePI of a medicine by using a smart phone to scan a two-dimensional code printed on medicine packages to access up-to-date information on medicines safety, benefits and conditions of use, in their preferred language where available.

Streamlining development and assessment of biosimilar medicines (02/04/2025)

This reflection paper suggests that demonstrated structural and functional comparability, together with comparative data on how the body interacts with the medicines (pharmacokinetic data), may be sufficient to demonstrate similarity with the reference medicine waiving, potentially reducing the need for extensive clinical efficacy studies.

ICH M11 guideline, clinical study protocol template and technical specifications - Scientific guideline (01/04/2025)

The purpose of this new harmonised guideline is to introduce the clinical protocol template and the technical specification to ensure that protocols are prepared in a consistent fashion and provided in a harmonised data exchange format acceptable to the regulatory authorities.

Insights into new approach methodology innovation: an EMA perspective (01/04/2025)

New approach methodologies (NAMs) in the context of medicines development may be incorporated into the assessment of the safety, efficacy and quality of a medicinal product to replace, reduce or refine (3Rs) the use of animals.

EMA approvals of new medicinal products and new biosimilar products in January and February 2025 (01/04/2025)

In January and February 2025, the EMA Committee for Medicinal Products for Human Use (CHMP) recommended the granting of an EU-wide marketing authorisation for seven new medicines and three new biosimilar products. The CHMP also recommended two new generic medicines and extensions of the indication for 24 medicinal products already authorised in the EU.

Joint strategy sets direction of EMA and EU medicines regulatory agencies to 2028 (01/04/2025)

EMA and the Heads of Medicines Agencies (HMA) have published their joint EU medicines agencies’ network strategy to 2028 (EMANS), following its recent adoption by the HMA and the EMA Management Board. The strategy, titled ‘Seizing opportunities in a changing medicines landscape’, is a comprehensive update of the five-year strategy which was developed to cover the period 2021 to 2025 (EMANS 2025).

Unregulated advanced therapy medicinal products pose serious risks to health (01/04/2025)

EMA and the Heads of Medicines Agencies (HMA) are warning the public about the dangers of unregulated advanced therapy medicinal products (ATMPs) offered to patients in the European Union in particular dendritic cell cancer therapies.

New Approach Methodologies EU-IN Horizon Scanning Report - February 2025 (01/04/2025)

Horizon scanning is the systematic examination of information to detect early signs of scientific and technological developments with previously unknown regulatory challenges or public health opportunities. This HMA/EMA report include recommendations to adapt the European Medicines Regulatory Network (EMRN) to minimise regulatory bottlenecks, to support developers and to facilitate innovation reaching patients.

Survey of academic and not-for-profit developers: share your needs and views on scientific and regulatory support (01/04/2025)

EMA invites developers of medicines and tools for medicine development from academic and not-for-profit organisations to participate in a short survey which aims to map academic developments and better understand the needs and experiences of academic developers. It will be used to inform EMA’s activities in supporting academic and not-for-profit developers.

Joint HTAb-regulatory perspectives on understanding evidence challenges, managing uncertainties and exploring potential solutions (03/04/2025)

Outcome of a workshop series between HTA bodies and regulators using three case studies: i) Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy, ii) Rybrevant (amivantamab), for the treatment of locally advanced or metastatic non-small cell lung cancer, iii) Yescarta (axicabtagene ciloleucel), for the treatment of adult patients with diffuse large B-cell lymphoma.

High-risk medical devices: consultation procedures and advice (01/04/2025)

EMA published a report on the pilot on the Advice from the Expert Panels to Manufacturers of High-Risk Medical Devices which include: i) Class III implantable devices (e.g. breast implants) and ii) Class IIb active devices that are intended to administer or remove medicinal products from the body.

Regulation on the European Health Data Space published (01/04/2025)

The Regulation on the European Health Data Space (EHDS) has been published in the Official Journal of the EU. The Regulation is an important milestone in the EU’s efforts to build a secure and efficient digital health ecosystem as part of the European data strategy. The EHDS will provide a comprehensive framework for the access to and use of electronic health data across Member States.

European Organisation for Research and Treatment of Cancer (EORTC) bilateral on cancer treatments for older adults meeting 9 April 2025 (01/04/2025)

EMA is hosting a closed meeting with the EORTC with the support of the International Society of Geriatric Oncology (SIOG) to exchange views and promote an open and direct dialogue on cancer treatment for older adults.

EMA workshop on the challenges in drug development, regulation and clinical practice for immunoglobulins 5 March 2025 (03/04/2025)

Immunoglobulins can be given in various indications but one of them is the treatment of secondary immunodeficiencies which cover treatment of side effects in oncology (e.g. hypogammaglobulinemia). EMA held a workshop to get clinicians’ / healthcare professionals’ perspective on the use of immunoglobulins.

Meeting of the Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) – 1 April 2025 (01/04/2025)

The Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) was established under Regulation (EC) 2022/123 on a reinforced role for the EMA in crisis preparedness and management for medicinal products and medical devices. The MSSG discussed the vulnerabilities in the supply chain of radiopharmaceuticals.

The work plan of the Oncology working party 2025 to 2027 has been published (28/02/2025)

The Oncology working party has just published its consolidated 3 years work plan until December 2027 which includes short term as well as long-term goals.

CHMP highlights (oncology) February 2025: Summary table (04/03/2025)

CHMP highlights (oncology) February 2025: Summary table

Lynozyfic (linvoseltamab), applicant: Regeneron Ireland Designated Activity Company (03/03/2025)

Lynozyfic (linvoseltamab), applicant: Regeneron Ireland Designated Activity Company

Trabectedin Accord (trabectedin), applicant: Accord Healthcare S.L.U. (03/03/2025)

Trabectedin Accord (trabectedin), applicant: Accord Healthcare S.L.U.

Calquence (acalabrutinib), MAH: AstraZeneca AB (03/03/2025)

Calquence (acalabrutinib), MAH: AstraZeneca AB

Columvi (glofitamab), MAH: Roche Registration GmbH (04/03/2025)

Columvi (glofitamab), MAH: Roche Registration GmbH

Darzalex (daratumumab), MAH: Janssen-Cilag International N.V. (04/03/2025)

Darzalex (daratumumab), MAH: Janssen-Cilag International N.V.

Enhertu (trastuzumab deruxtecan), MAH: Daiichi Sankyo Europe GmbH (04/03/2025)

Enhertu (trastuzumab deruxtecan), MAH: Daiichi Sankyo Europe GmbH

Imfinzi (durvalumab), MAH: AstraZeneca AB (04/03/2025)

Imfinzi (durvalumab), MAH: AstraZeneca AB

Jaypirca (pirtobrutinib), MAH: Eli Lilly Nederland B.V. (04/03/2025)

Jaypirca (pirtobrutinib), MAH: Eli Lilly Nederland B.V.

Keytruda (pembrolizumab), MAH: Merck Sharp & Dohme B.V. (04/03/2025)

Keytruda (pembrolizumab), MAH: Merck Sharp & Dohme B.V.

Kizfizo (temozolomide), applicant: Orphelia Pharma (04/03/2025)

Kizfizo (temozolomide), MAH: Orphelia Pharma

Orphan medicines in the EU (28/02/2025)

28 February marks the rare disease day. Finding effective treatment for patients with rare diseases can be very difficult. Since the EU orphan regulations entered into force in 2000, it has played a central role in facilitating the development and authorisation of medicines for rare diseases. Over 260 orphan medicines have been authorised in the EU since 2000.

Challenges and ongoing initiatives towards better integrated EU scientific advice (04/03/2025)

Co-authored by EMA colleagues, this article explored how the new EU pharmaceutical legislation is expected to solidify advice mechanisms and creates new avenues for enhanced integration of development support. Development support and guidance for scientific evidence generation need to evolve with more intense collaboration between medicines regulators and HTA bodies to improve patient access to new medicines.

Cardiovascular Safety in Oncology Clinical Trials (04/03/2025)

Co-authored by an oncology working party member, this article highlights that the development of novel treatments has improved cancer outcomes but may result in cardiovascular toxicities. The aim of this collaborative effort is to fulfill the need for clear description of cancer treatment benefits alongside quantified cardiovascular adverse event.

Clinical Evidence 2030 (04/03/2025)

Authored by EMA colleagues, this article highlights that, by 2030, clinical evidence generation will be further guided by the patient voice and informed by existing data and knowledge; study design will be driven by research questions to be addressed; clinical trials will be more efficient and impactful; real-world evidence will be enabled and fully established; and trust will be built through transparency.

Article on the new European platform for advancing regulatory science research (28/02/2025)

Launching in 2025, the European Platform for Regulatory Science Research will bring together academia, regulators and other stakeholders to accelerate collaborative regulatory science research solutions. The platform will launch with a one-year pilot phase to discuss selected questions, case studies, research gaps, and priority topics.

Finding clinical trials with the ACT EU Trial Map (28/02/2025)

A new clinical trial map is now accessible from the public website of the Clinical Trials Information System. Users can look for ongoing trials by geographic area and medical condition. On 7 March 2025 (13.30-14.30 CET), a live demonstration of the a map will be given and the event will be broadcast live.

How does DARWIN EU make health data count? - Video Explainer (04/03/2025)

How does DARWIN EU make health data count? - Video Explainer

EMA establishes regular procedure for scientific advice on certain high-risk medical devices (28/02/2025)

EMA, in close collaboration with the European Commission, has established a standard procedure free of charge for manufacturers of certain high-risk medical devices to request scientific advice on their intended clinical development strategy and proposals for clinical investigation.

Guideline on assessment and reporting of mechanistic models used in the context of model informed drug development (07/03/2025)

A concept paper has been published to provide guidance on the assessment and reporting of mechanistic models such as physiologically based pharmacokinetic, physiologically based biopharmaceutics and quantitative systems pharmacology models. Comments should be provided by 31 May 2025.

Joint EC/HMA/EMA multi-stakeholder workshop on pharmacogenomics 24 September 2024 (04/03/2025)

The meeting report provides recommendations on pharmacogenomics in medicines regulation and on implementation into clinical practice. Recommendations include: priority areas for additional regulatory action, facilitating the uptake of genomics by national healthcare systems, leverage genomic data linked to real-world data sources and increase the impact of EU-funded projects.

European medicines agencies network strategy (EMANS) to 2028 webinar on 13 February 2025 (03/03/2025)

The EU Polish Presidency, the Heads of Medicines Agencies (HMA) and the EMA jointly organised a webinar to present how the feedback received during the public consultation on the European Medicines Agencies Network Strategy (EMANS) to 2028 has been incorporated into the draft EMANS and to clarify further input before final adoption.

World Cancer Day – 4 February (04/02/2025)

'Supporting the 'big leaps' is fundamental. And so is every little step we make along the way. Every advancement could help one patient.' Emer Cooke, EMA's Executive Director.

EMA Pathfinder: Enhancing Efficiency in the Centralised Procedure (05/02/2025)

The EMA under its Cancer Medicines Pathfinder initiative is launching a focused dialogue with industry to explore ways to improve the efficiency of the centralised procedure. A key starting point is communication ensuring clarity, consistency, and timeliness throughout the process. By bringing stakeholders together in a dedicated focus group, EMA aims to identify practical solutions that streamline interactions, reduce procedural uncertainties, and enhance efficiency for regulators and applicants. Stay tuned for insights from this initiative as discussions progress.

CHMP highlights (oncology) January 2025: Summary table (05/02/2025)

CHMP highlights (oncology) January 2025: Summary table

Datroway (datopotamab deruxtecan), applicant: Daiichi Sankyo Europe GmbH (04/02/2025)

Datroway (datopotamab deruxtecan), applicant: Daiichi Sankyo Europe GmbH

Tivdak (tisotumab vedotin), applicant: Pfizer Europe MA EEIG (04/02/2025)

Tivdak (tisotumab vedotin), applicant: Pfizer Europe MA EEIG

Breyanzi (lisocabtagene maraleucel), MAH: Bristol-Myers Squibb Pharma EEIG (04/02/2025)

Breyanzi (lisocabtagene maraleucel), MAH: Bristol-Myers Squibb Pharma EEIG

Imfinzi (durvalumab), MAH: AstraZeneca AB (04/02/2025)

Imfinzi (durvalumab), MAH: AstraZeneca AB

Opdivo (nivolumab), MAH: Bristol-Myers Squibb Pharma EEIG (04/02/2025)

Opdivo (nivolumab), MAH: Bristol-Myers Squibb Pharma EEIG

Yervoy (ipilimumab), MAH: Bristol-Myers Squibb Pharma EEIG (04/02/2025)

Yervoy (ipilimumab), MAH: Bristol-Myers Squibb Pharma EEIG

Datopotamab deruxtecan Daiichi Sankyo (datopotamab deruxtecan), Daiichi Sankyo Europe GmbH (04/02/2025)

Datopotamab deruxtecan Daiichi Sankyo (datopotamab deruxtecan), Daiichi Sankyo Europe GmbH

Recommendations on eligibility to PRIME scheme - December 2024 (10/12/2024)

Published PRIME outcome from December 2024 CHMP meeting, the CHMP reviewed 5 recommendations for eligibility to PRIME: 2 were granted and 3 were denied.

New EU rules for health technology assessments (HTA) become effective for oncology medicines as of January 2025 (04/02/2025)

The regulation recognises the value of cooperation between decision-makers, namely regulators who evaluate the benefits and the risks of medicines and HTA bodies who then assess their effectiveness compared to existing products. It builds on the longstanding cooperation between EMA and HTA bodies, developed with the European Network for Health Technology Assessment (EUnetHTA) until September 2023.

Evidence generation throughout paediatric medicines life cycle: findings from collaborative work between European Medicines Agency (EMA) and EUnetHTA on use of extrapolation (04/02/2025)

Co-authored by EMA colleagues, this article explored collaboratively the unique specific challenges relating to paediatric drug development, ethically and in its ability to leverage prior knowledge, as exemplified using extrapolation. Learnings from these offer opportunities to further develop methodology on how to leverage uncertainties across a product's life cycle for small populations generally.

Paediatric strategy forum for medicinal product development in diffuse midline gliomas (DMG) in children and adolescents ACCELERATE in collaboration with the EMA with participation of the FDA (07/02/2025)

Fewer than 10 % of children with DMG survive 2 years from diagnosis. Radiation therapy remains the cornerstone of treatment. Capturing and utilising data from each child receiving DMG therapy is essential to inform the collective knowledge for the DMG community to work together, swiftly and strategically to coordinate an international, randomised, platform, complex innovative design trial.

Which clinical trial designs and statistical approaches have been used in assessments of orphan maintenance by the European Medicines Agency between 2012 and 2022? A cross-sectional study (04/02/2025)

The use of indirect treatment comparisons in EMA orphan drug assessment is increasing. This article shows that indirect comparisons are the most common approach for comparing new orphan drugs to relevant comparator drugs (44% of comparisons). Differences were seen between oncology and non-oncology orphan maintenance procedures with regards to study design and the type of indirect comparisons most frequently used.

Demonstration of Major Therapeutic Advantage From a Review of EU Conditional Marketing Authorizations in Oncology and Hematology (04/02/2025)

Conditional marketing authorisation is a pragmatic tool available in the EU to enable an early approval of a medicine that fulfils an unmet medical need while comprehensive data continue to be generated. In that context, a scientific review by EMA colleagues explored how a major therapeutic advantage against existing options was justified for oncology and haematology products receiving CMAs between 2006 and 2023.

New Insights on External Comparators with Real-World Data (05/02/2025)

This collaborative study with EMA, EHA and ESMO is exploring the role of external comparators using real-world data to supplement non-randomised studies. The study surveyed the attitudes of assessors and clinicians from regulatory agencies and medical organisations worldwide. The final results will be presented at the upcoming ESMO Sarcoma and Rare Cancers Congress 2025 in Lugano.

EMA celebrates 30 years of progress in science and medicines in the European Union (04/02/2025)

EMA is celebrating 30 years of operations marking its evolving role at the centre of the European Union (EU) assessing and authorising safe, high-quality medicines for 450 million people and countless animals in Europe. EMA was set up in 1995 to manage the centralised procedure for the authorisation of medicines and to harmonise the work of existing national medicine regulatory bodies.

Clinical Trials Regulation becomes fully applicable (04/02/2025)

From January 2025, all clinical trials in the EU, including ongoing trials that were approved under the Clinical Trials Directive, are governed by the Clinical Trials Regulation. Clinical Trials Information System (CTIS) includes a public searchable database for healthcare professionals, patients and the general public to deliver the high level of transparency foreseen by the regulation.

ACT EU workplan for 2025-2026 (04/02/2025)

The revised workplan highlights ACT EU’s areas of focus over the next two years, reflecting feedback received through the multi-stakeholder platform advisory group and other channels in 2023 and 2024.

Revised rules on handling of competing interests published (04/02/2025)

EMA has published the revised version of its policies on the handling of competing interests of scientific committee members and experts (‘Policy 0044’) and of the EMA Management Board members (‘Policy 0058’). These policies set out the ground rules on which EMA involves them in its work.

European medicines agencies network strategy (EMANS) to 2028 webinar organised on 13 February 2025 (04/02/2025)

The EU Polish Presidency, the Heads of Medicines Agencies and the EMA are jointly organising a webinar to present how the feedback received during the public consultation on the European Medicines Agencies Network Strategy (EMANS) to 2028 has been incorporated into the draft EMANS and to clarify further input before final adoption.

Twelfth Industry Standing Group (ISG) meeting (05/02/2025)

EMA is holding its twelfth quarterly meeting of the Industry Standing Group (ISG) to discuss the EMA Mandate extension (Regulation (EU) 2023/123) and the HTA Regulation (Regulation (EU) 2021/2282).

ACT EU workshop on ICH E6 R3 Good Clinical Practice (GCP) modernisation (principles and Annex 1) (19/02/2025)

This workshop on 19-20 February will provide an overview of the major changes in the guideline, key concepts for adaptation to recent developments in trial design, organisation and technology, and enable discussion with stakeholders. The event will be broadcast live.

Qualification of non-mutagenic impurities - EMA scientific guideline (05/02/2025)

This reflection paper considers the safety evaluation of non-mutagenic impurities (NMI) in chemically synthesised pharmaceuticals and how to qualify novel impurities. A webinar on the reflection paper on the qualification of non-mutagenic impurities will take place on 6 March 2025.

Joint EMA/FDA Conversations on Cancer - Pediatric Cancers: Navigating the Challenges Together (06/02/2025)

This Conversations on Cancer discussed on 19 November 2024 the complex and challenging decisions related to clinical care, research, and regulatory decision making in pediatric oncology.

Joint EMA/FDA Conversations on Cancer: Cervical Cancer Treatment Innovation—A Collaborative Discussion (04/02/2025)

Commemorating cervical cancer awareness month, the EMA and the FDA discussed on 14 January 2025 the historical path that has led us to the current treatment landscape, the impact of new therapies on individuals with cervical cancer and their families, areas of ongoing high impact research, and what the future might bring.

Two years' review of the Oncology European Specialised Expert Community (ESEC) (19/12/2024)

Established in May 2022, the oncology European Specialised Expert Community (ESEC) is a community of around 160 of European experts and EMA staff with expertise in oncology. It provides a platform of information sharing and communication in oncology.

CHMP highlights (oncology) December 2024: summary table (10/12/2024)

CHMP highlights (oncology) December 2024: summary table

Rytelo (imetelstat), applicant: Geron Netherlands B.V. (18/12/2024)

Rytelo (imetelstat), applicant: Geron Netherlands B.V.

Welireg (belzutifan), applicant: Merck Sharp & Dohme B.V. (13/12/2024)

Welireg (belzutifan), applicant: Merck Sharp & Dohme B.V.

Osenvelt (denosumab), applicant: Celltrion Healthcare Hungary Kft. (13/12/2024)

Osenvelt (denosumab), MAH: Celltrion Healthcare Hungary Kft.

Blincyto (blinatumomab), MAH: Amgen Europe B.V. (13/12/2024)

Blincyto (blinatumomab), MAH: Amgen Europe B.V.

Jemperli (dostarlimab), MAH: GlaxoSmithKline (Ireland) Limited (13/12/2024)

Jemperli (dostarlimab), MAH: GlaxoSmithKline (Ireland) Limited

Kizfizo (temozolomide), MAH: Orphelia Pharma (13/12/2024)

Kizfizo (temozolomide), MAH: Orphelia Pharma

Keytruda (pembrolizumab), MAH: Merck Sharp & Dohme B.V. (13/12/2024)

Keytruda (pembrolizumab), MAH: Merck Sharp & Dohme B.V.

Recommendations on eligibility to PRIME scheme - November 2024 (10/12/2024)

Published PRIME outcome from November 2024 CHMP meeting, the CHMP reviewed 9 recommendations for eligibility to PRIME: 1 was granted and 8 were denied.

Article on uncertainties about the benefit-risk balance of oncology medicines assessed by the European Medicines Agency (19/12/2024)

Co-authored by EMA oncology staff, this article aimed to evaluate the type and number of uncertainties described in the benefit-risk assessment for initial marketing authorisations of oncology medicines assessed by the EMA in the view to develop a systematic classification of uncertainties to contribute to improved communication about uncertainties.

Call for experts to join scientific advisory groups at EMA (19/12/2024)

Experts can express their interest in becoming Scientific Advisory Groups (SAGs) members, as part of the upcoming mandate renewal. Candidates need to provide a motivation letter specifying the group the applicant is interested in joining, a curriculum vitae (CV) including a list of publications; a completed declaration of interest form and one letter of recommendation. Deadline to apply is 6 January 2025, 23:59 CET.

European Medicines Agency mid-year report 2024 (January-June 2024) (10/12/2024)

In the first half of 2024, the EMA made substantial progress in its overall strategic priorities. The Agency has been using haematology and oncology products as pathfinders to enhance the assessment of key medicines, strengthen collaboration with stakeholders and improve communication on benefits and risks.

16th annual report of the Good Clinical Practice (GCP) Inspectors' Working Group (IWG) 2023 - 26 November 2024 (10/12/2024)

This group was established in 1997 under the scope of Article 51(e) of Council Regulation (EEC) No. 2309/93, subsequently amended as Article 57(1)(i) of Regulation (EC5 No. 726/2004. The report provides an overview of inspections carried out in 2023, their geographical distribution and categorisation findings and the new procedures and guidance documents which have been published.

Public information on medicine shortages (10/12/2024)

EMA publishes information on medicine shortages that affect or are likely to affect more than one European Union (EU) country, where it has assessed the shortage and provided recommendations to patients and healthcare professionals. Added on 9 December 2024: 'Communication to healthcare professionals' section.

New dedicated cancer webpages on EMA website (10/12/2024)

A new cancer webpage has been created on the EMA website where information can be found on the Cancer Medicines Pathfinder initiative, the Cancer Medicines Forum, the educational projects on cancer medicine regulation as well as on patient engagement in oncology. In addition, the links to the previous EU Regulatory Oncology Newsletters are also published there.

Article on evidence generation throughout paediatric medicines life cycle: findings from collaborative work between European Medicines Agency (EMA) and EUnetHTA on use of extrapolation (10/12/2024)

Co-authored by EMA paediatric office staff, an article titled on the Evidence generation throughout paediatric medicines lifecycle: findings from collaborative work between the EMA and European Network for Health Technology Assessment (EUnetHTA) on use of extrapolation.

Conversations on cancer: Cervical Cancer Treatment Innovation—A Collaborative Discussion - 14 January 2024 (19/12/2024)

The European Medicines Agency (EMA) in collaboration with the FDA Oncology of Excellence (OCE) presents a Conversations on Cancer program, January 14, 2025, 10:00-11:00 AM ET/16:00-17:00 PM CET. The session, titled Cervical Cancer Treatment Innovation: A Collaborative Discussion, will commemorate Cervical Cancer Awareness Month.

Cancer Medicines Forum - 1 October 2024 (10/12/2024)

The EMA set up the Cancer Medicines Forum in 2022 to help advance research into optimising cancer treatments and foster high standards in cancer care in the European Union (EU). This forum brings together representatives of academic organisations from EMA’s Healthcare Professionals Working Party and the European medicines regulatory network.

19th Industry Stakeholder Platform – Operation of EU Pharmacovigilance - 15 November 2024 (10/12/2024)

The meeting focused on: DARWIN EU and real world evidence studies in medicines regulation, PSUR updates, non-fixed drug combinations & pharmacovigilance, update on the pregnancy labelling guideline, the Good Pharmacovigilance Practices, AI in pharmacovigilance, eudravigilance and an update about the implementing Regulation (EU) No. 520/2012 and Regulation (EU) 2024/568.

13th Industry stakeholder platform on research and development support - 2 December 2024 (10/12/2024)

The meeting covered the following topics: the experience with Portfolio and Technology meetings (PTMs), the development support offering for programme-specific evidence planning, the support of paediatric developments, focus group to explore opportunities for the use of Real-World Data (RWD) and the generation of Real-World Evidence (RWE) and an operational update.

World Children’s Day - 20 November 2024 (20/11/2024)

Building a brighter future for children battling cancer.

Augtyro (repotrectinib), Bristol-Myers Squibb Pharma EEIG (19/11/2024)

Augtyro (repotrectinib), Bristol-Myers Squibb Pharma EEIG

Lazcluze (lazertinib), Janssen Cilag International (19/11/2024)

Lazcluze received a positive opinion for the first‑line treatment in combination with amivantamab of adult patients with advanced non‑small cell lung cancer (NSCLC) with EGFR exon 19 deletions or exon 21 L858R substitution mutations.

Rybrevant (amivantamab), Janssen-Cilag International NV (19/11/2024)

The CHMP adopted a new indication to extend the use of Rybrveant to the first‑line treatment of adult patients with advanced non‑small cell lung cancer (NSCLC) with EGFR Exon 19 deletions or Exon 21 L858R substitution mutations, in combination with lazertinib

Jakavi (ruxolitinib), Novartis Europharm Limited (22/11/2024)

The CHMP adopted a new indication for the treatment of paediatric patients with acute and chronic graft versus host disease (GvHD) together with a new pharmaceutical form, Jakavi 5 mg/mL oral solution. The oral solution is to be used by patients who cannot swallow tablets.

Opdivo (nivolumab), Bristol-Myers Squibb Pharma EEIG (19/11/2024)

The CHMP adopted a new indication to extend the use of Opdivo in combination with Yervoy to patients with Mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) colorectal cancer (CRC)

Yervoy (ipilimumab), Bristol-Myers Squibb Pharma EEIG (19/11/2024)

The CHMP adopted a new indication to extend the use of Yervoy in combination with nivolumab for the treatment of adults patients with mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer

Keytruda (pembrolizumab), Merck Sharp & Dohme B.V. (19/11/2024)

The CHMP adopted a new indication to extend the use of Keytruda to patients with Malignant pleural mesothelioma (MPM).

Sarclisa (isatuximab), Sanofi Winthrop Industrie (19/11/2024)

The CHMP adopted a new indication to extend the use of Sarclisa in combination with bortezomib, lenalidomide, and dexamethasone, for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant.

Tagrisso (osimertinib), AstraZeneca AB (19/11/2024)

The CHMP adopted a new indication to extend the use of Tagrisso as monotherapy for the treatment of adult patients with locally advanced, unresectable (stage III) NSCLC whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations and whose disease has not progressed during or following platinum based chemoradiation therapy

Kizfizo (temozolomide), Orphelia Pharma (19/11/2024)

The European Medicines Agency has recommended the refusal of the marketing authorisation for Kizfizo, a medicine intended for the treatment of children with neuroblastoma, a cancer of nerve cells. The Agency issued its opinion on 14 November 2024. The company that applied for authorisation, Orphelia Pharma, may ask for re-examination of the opinion within 15 days of receiving the opinion.

Inaqovi (cedazuridine / decitabine) (19/11/2024)

Otsuka Pharmaceutical Netherlands B.V. withdrew its application for the use of Inaqovi in the treatment of adult patients with myelodysplastic syndromes (conditions where the bone marrow does not make enough healthy blood cells or platelets) or chronic myelomonocytic leukaemia (another type of cancer of the white blood cells). The company withdrew the application for CMML on 5 August 2024 and for MDS on 6 November 2024.

Advancing regulatory science research (19/11/2024)

Comments should be provided until 31 December 2024 on the draft updated Regulatory science research needs and on the concept for a European platform for Regulatory science research. These were discussed among academic researchers, regulators and stakeholders, including from oncology, at the recent public event Advancing regulatory science research.

Draft European Medicines Agencies Network Data Strategy (20/11/2024)

Comments should be provided until 31 December 2024.

Fifth EMA/HMA Big Data Stakeholder Forum (19/11/2024)

The fifth annual Big Data multi-stakeholder forum will take place on 28 November 2024 at the EMA building in Amsterdam.

ACT EU workshop on ICH E6 R3 (principles and Annex 1) (19/11/2024)

ACT EU Priority Action – GCP Modernisation is conducting a Workshop on ICH E6 R3 on 19 and 20 February 2025.

Foreword (17/10/2024)

We are pleased to introduce our first European Regulatory Oncology Newsletter. This publication is designed for anyone with an interest in cancer medicines. Each month, we will dive into the latest scientific opinions on oncology medicines in the EU, and share news about events, initiatives and activities from EU regulators in the field.

Korjuny (catumaxomab), Lindis Biotech GmbH (17/10/2024)

Korjuny received a positive opinion for the intraperitoneal treatment of malignant ascites in adults with epithelial cellular adhesion molecule (EpCAM)-positive carcinomas, who are not eligible for further systemic anticancer therapy

Tevimbra (tislelizumab), BeiGene Ireland Ltd II-03 and II- 06 (21/10/2024)

Tevimbra received a positive opinion to extend the use of Tevimbra to patients with previously untreated oesophageal squamous cell carcinoma (OSCC).

Cancer medicines pathfinder, an initiative to accelerate the development and assessment of cancer medicines (28/10/2024)

Launched in 2023, the cancer medicines pathfinder explores how EMA can improve the evaluation of medicines. It is based on three pillars: accelerating the assessment of cancer medicines, strengthening multi-stakeholder dialogue and international collaboration, and improving communication on benefits and risks of cancer medicines.

Public consultation on Oncology Working Party on assessment of cardiovascular safety of oncology medicinal products (12/09/2024)

The purpose of the reflection paper on assessment of cardiovascular safety (CV) of oncology medicinal products is to address the CV safety concerns in drug development in oncology specifically the lack of uniformity in CV toxicity endpoints, characterisation of the baseline CV risk, monitoring, assessment and follow-up in oncology studies to date. Comments can be provided by 31 October 2024.

Public consultation open on the concept paper on clinical evaluation of therapeutic radiopharmaceuticals in oncology (17/10/2024)

The objective of this concept paper is to consider the impact and role of implementing patient-specific dosimetry in clinical trials and in clinical practice with the purpose of optimising safety and efficacy of the therapeutic radiopharmaceuticals treatments. Comments are open until 31 January 2025.

Reflection paper on establishing efficacy based on single-arm trials (SAT) submitted as pivotal evidence in a marketing authorisation application (23/09/2024)

This reflection paper outlines perspectives on SAT that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications. It concludes that unbiased estimates are difficult to obtain from SATs and how multiple potential sources of bias need to be addressed throughout the design, conduct, analysis and reporting of results from a SAT.

ICH guideline E11A on pediatric extrapolation - Scientific guideline (23/09/2024)

This guideline recommends approaches to assess factors that influence the determination of similarity of disease, drug pharmacology, and response to treatment between a reference and pediatric target population and how the characteristics of the disease, drug pharmacology and response to treatment may influence this determination.

Election of new CHMP vice-chair (21/10/2024)

Following the appointment of Bruno Sepodes as chair of the Committee for Human Medicinal Products (CHMP) on 21 September 2024, the CHMP elected Outi Mäki-Ikola as its new vice-chair for a three-year mandate, starting on 15 October 2024.

European Medicines Agencies network strategy to 2028 – launch of public consultation (21/10/2024)

The draft joint EU network strategy to 2028 focus on accessibility, leveraging data, digitalisation and artificial intelligence, regulatory science, innovation and competitiveness, antimicrobial resistance and other health threats, availability and supply, sustainability of the network. Comments are open until 30 November 2024.

SNE/Scientific Specialist (Clinical specialist in oncology/haematology) (23/10/2024)

The EMA has published a vacancy to join the oncology office and advanced therapies and haemato-oncology office at EMA as a Seconded National Expert for 2 years. We are looking for clinical expertise in the diagnosis and treatment of cancer and/or haematological diseases, in particular solid tumours, haematological malignancies, and/or non-malignant haematological diseases. Deadline for application is 18th November.

Translating innovation into access for ATMPs: 3rd EU-Innovation network (EU-IN) multi-stakeholder meeting (15/11/2024)

The EU-Innovation Network (EU-IN) and the Italian Medicines Agency (AIFA) are organising a multi-stakeholder meeting on 15 November 2024 to discuss how innovation in the development of Advanced Therapy Medicinal Products (ATMPs) can be translated into effective and safe therapies at the point of care in Europe. CAR-T cells as a specific case study will be discussed.

Conversations on cancer – Paediatric Cancers: Navigating the Challenges Together (19/11/2024)

EMA in conjunction with FDA Oncology Center of Excellence (OCE) presents a one-hour Conversations on Cancer public panel discussion on November 19, 2024 to foster a better understanding of how decisions made by patients and their parents impact their day-to-day lives and how regulators approach difficult regulatory decisions.

Thirteenth meeting of the industry stakeholder platform on the operation of the centralised procedure for human medicines (22/11/2024)

EMA is hosting the thirteenth industry stakeholder platform on 22 November 2024 on the operation of the centralised procedure for human medicines. One of the topic to be discussed will be on cancer medicines pathfinder regulatory interactions.

Training session for patients, consumers and healthcare professionals involved in medicine regulatory activities (05/12/2024)

This virtual training for representatives of patients, consumers and healthcare professionals' organisations aims at presenting an overview of medicines regulatory activities. Using a combination of presentations, plenary discussions and breakout sessions, participants will learn more about how they can be involved and contribute to medicines regulatory activities.

14th Paediatric Strategy Forum on novel ways to target and enhance efficiency of anti-GD2 therapies (24/10/2024)

Anti-GD2 therapies are part of standard of care in neuroblastoma. Jointly organised by Accelerate and EMA, this forum discussed novel ways to target GD2 and explore the use of monoclonal antibodies, CAR T-cells, antibody–drug conjugates, radiolabeled antibodies, T-cell engaging bispecific antibodies and vaccines to identify and prioritise their most effective clinical settings.

Meeting report published from EMA and EORTC workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions? (23/10/2024)

The EMA and EORTC jointly organised on 29 February 2024 a workshop on how patient-reported outcomes (PRO) as well as health-related quality of life (HRQoL) data can inform regulatory decisions. The meeting report has just been published.

Meeting report published from joint HMA/EMA multi-stakeholder workshop on submission predictability (21/10/2024)

EMA and the European medicines regulatory network are working to accelerate the availability of safe and effective treatments for patients. The final report of the focus group on submission predictability which was held on 25 September 2024 highlight the importance to promptly and transparently communicate any changes from the submission plan of the Marketing Authorisation Application.