Welcome to the April issue of Human Medicines Highlights (11/04/2025)

Welcome to the April 2025 issue of the Human Medicines Highlights newsletter, the monthly newsletter published by the European Medicines Agency specifically for patients, consumers, healthcare professionals and their representative organisations.

EMA qualifies first artificial intelligence tool to diagnose inflammatory liver disease in biopsy samples (25/03/2025)

EMA’s human medicines committee has issued the first qualification opinion on an innovative development methodology based on artificial intelligence (AI). The tool helps pathologists identify the severity of disease in clinical trials.

Streamlining development and assessment of biosimilar medicines (08/04/2025)

EMA is exploring improvements to development and evaluation of biosimilar medicines while upholding strict EU safety standards and is inviting comments on a proposed approach in a reflection paper.

Clinical Trials Information System designated as WHO primary registry (08/04/2025)

CTIS has already been a registered data provider for WHO since May 2023. Becoming a primary registry is a significant milestone in facilitating data sharing and promoting transparency and trust in clinical research.

EMA concludes review of weight management medicine Mysimba (03/04/2025)

The human medicines committee, CHMP, concluded that the benefits of Mysimba (naltrexone/bupropion), a medicine used for the management of obesity or overweight, continue to outweigh risks. New risk minimisation measures are put in place for long-term use.

Survey for healthcare professionals involved in academic medicine development open until 30 April 205 (15/04/2025)

EMA is inviting views from academic and not-for-profit developers to understand better their needs for scientific and regulatory support. The survey is open until 30 April and can be accessed via the link.

Antivirals/anti-infectives (02/04/2025)

Antivirals/anti-infectives

Cancer (02/04/2025)

Cancer

Dermatology (02/04/2025)

Dermatology

Diabetes (02/04/2025)

Diabetes

Haematology (03/04/2025)

Haematology

Immune system (02/04/2025)

Immune system

Metabolic system (03/04/2025)

Metabolic system

Musculoskeletal system (02/04/2025)

Musculoskeletal system

Nervous system (03/04/2025)

Nervous system

Ophthalmology (03/04/2025)

Ophthalmology

Rheumatology (02/04/2025)

Rheumatology

Vaccines (02/04/2025)

Vaccines

Other medicines (15/04/2025)

Other medicines

Cancer Medicines Forum: report and presentation available (16/12/2024)

The report and presentation of the Cancer Medicines Forum held in December 2024 are available on the event page.

EMA’s 30th anniversary scientific conference: Medicines, regulation and the future (25/06/2025)

The scientific conference celebrates three decades of groundbreaking achievements and advancements in medicine and regulatory science. It will bring together key partners and stakeholders to discuss latest trends, innovations, and challenges in medicines regulation, public and animal health. The event can be followed via a live broadcast without registration.

EMA multi-stakeholder workshop on reporting and qualification of mechanistic models for regulatory assessment (08/10/2025)

This multistakeholder workshop will bring together academia, regulators and industry to discuss the experience with the current regulatory landscape around the application of mechanistic models to support drug development.

Open consultations (25/03/2025)

Open consultations

Scientific committee and working party activities (25/03/2025)

Scientific committee and working party activities

EMA reflection paper on real-world evidence and roadmap for guidance development (08/04/2025)

The final EMA reflection paper on non-interventional studies using real-world data to generate evidence for regulatory purposes has now been published, along with a roadmap for producing guidance on real-world evidence to support regulatory decision-making.

EMA Management Board: highlights of March 2025 meeting (25/03/2025)

The Agency's annual report 2024, updated network strategy (EMANS 2028) and revised composition of the Emergency Task Force (ETF) were adopted during the meeting.

Pilot on advice from expert panels to manufacturers of high-risk medical devices: Interim report (03/04/2025)

This is an interim report on the experience with the pilot from February 2023 to December 2024

2024 European Medicines Agency annual report on independence (25/03/2025)

EMA published the 2024 EMA annual report on independence

From lab to patient: how EMA approves medicines (14/04/2025)

Follow the journey of a centrally authorised medicine: find out what happens at every stage and how stakeholders are involved. Interactive timeline available in English and PDF leaflet in 22 European languages.

Welcome to the March issue of Human Medicines Highlights (13/03/2025)

Welcome to the March 2025 issue of the Human Medicines Highlights newsletter, the monthly newsletter published by the European Medicines Agency specifically for patients, consumers, healthcare professionals and their representative organisations.

Joint strategy sets direction of EMA and EU medicines regulatory agencies (13/03/2025)

The update of the strategy to 2028 titled 'Seizing opportunities in a changing medicines landscape' will guide the European regulatory network over the next years to meet challenges ahead, such as public health emergencies and antimicrobial resistance.

Unregulated advanced therapy medicinal products pose serious risks to health (13/03/2025)

In a joint statement, EU authorities warn about the dangers of unregulated advanced therapy medicinal products, including dendritic cell cancer therapies, that are being advertised directly to patients on social media and other platforms.

New clinical trial map launched in the EU (13/03/2025)

The new clinical trial map provides patients and healthcare professionals with easy access to comprehensive, real-time information about clinical trials in their area, helping to increasing access to clinical research in the European Union.

EMA establishes regular procedure for scientific advice on high-risk medical devices (10/03/2025)

Manufacturers of certain devices intended to administer or remove medicines can request advice from medical device expert panels on their clinical development programmes.

EMA Management Board elects new chair (13/03/2025)

The EMA’s Management Board has elected Rui Santos Ivo of the Portuguese National Authority of Medicines and Healthcare Products as chair for a three-year period. He takes over from Lorraine Nolan, Chief Executive of the Irish Health Products Regulatory Authority.

Antivirals/anti-infectives (12/03/2025)

Antivirals/anti-infectives

Cancer (12/03/2025)

Cancer

Cardiovascular system (13/03/2025)

Cardiovascular system

Dermatology (12/03/2025)

Dermatology

Gastro-intestinal system (12/03/2025)

Gastro-intestinal system

Gynaecology & Obstetrics (12/03/2025)

Gynaecology & Obstetrics

Haematology (12/03/2025)

Haematology

Immune system (12/03/2025)

Immune system

Nephrology (12/03/2025)

Nephrology

Nervous system (18/03/2025)

Nervous system

Respiratory system (12/03/2025)

Respiratory system

Rheumatology (13/03/2025)

Rheumatology

Vaccines (12/03/2025)

Vaccines

Multistakeholder workshop on pharmacogenomics: report available (24/09/2024)

The report of the multistakeholder workshop on pharmacogenomics held in September 2024 is available on the event page, together with the presentations and recording of the event.

PCWP and HCPWP joint meeting (01/04/2025)

The Patients and Consumers' (PCWP) and Healthcare Professionals' (HCPWP) Working Parties will meet on 1-2 April 2025. The meeting is open only to members of the working parties but the meeting documents will be published on the event page.

Virtual public session of the 3Rs Working Party (02/04/2025)

As part of its April plenary meeting, the 3Rs Working Party will host a virtual public session to present its workplan and priorities for 2025-2027. The session will take place on 2 April, 09:30-10:30 CET via live broadcast and is open to all stakeholders.

Open consultations (11/03/2025)

Open consultations

New infographic and revamped web-page on orphan medicines (13/03/2025)

The EMA web-page on orphan medicines has been updated with key facts and figures and an infographic.

Horizon-scanning report: Nanotechnology-based medicinal products for human use (13/03/2025)

Nanotechnology is a rapidly evolving scientific research field with a wide applicability. The EU Innovation Network (EU-IN) has published a horizon-scanning report with an overview of the current status and key emerging trends.

From lab to patient: how EMA approves medicines (17/10/2024)

Follow the journey of a centrally authorised medicine: find out what happens at every stage and how stakeholders are involved. An interactive timeline is available in English and the PDF leaflet can be consulted in 22 European languages

Foreword Feb 2025 (06/02/2025)

Welcome to the February 2025 issue of the Human Medicines Highlights newsletter, the monthly newsletter published by the European Medicines Agency specifically for patients, consumers, healthcare professionals and their representative organisations.

EMA celebrates 30 years of progress in science and medicines in the European Union (31/01/2025)

This year, the Agency is celebrating 30 years. We bring you an overview of how its role has changed and grown since its creation in 1995 and a video with key milestones and achievements.

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

The transition period for implementation of the EU Clinical Trials Regulation ended on 31 January and the Clinical Trials Information System is now the single entry point for all clinical trials in the EU.

European Shortages Monitoring Platform fully operational for monitoring of shortages in the EU (31/01/2025)

The European Shortages Monitoring Platform (ESMP) is now live with full functionalities. ESMP facilitates monitoring and management of critical medicines during public health emergencies and major events and in the context of preparedness actions.

One Health: EU agencies unite to tackle azole fungicide resistance in Aspergillus fungi (31/01/2025)

Extensive use of azole fungicides outside human health can increase the risk of resistance. A new joint report by the EU's health and environment agencies highlights the need for cross-sectoral collaboration. A video is also available.

Joint EU campaign against fake medicines (11/02/2025)

In February, EMA and Europol are campaigning on social media about the dangers of buying fake medicines from unregistered pharmacies. Check our LinkedIn, Instagram and Bluesky accounts for the campaign.

Overview of human medicines in 2024 (20/01/2025)

EMA recommended 114 medicines for marketing authorisation in 2024, of which 46 had a new active substance never before authorised in the EU. This document gives an overview of EMA recommendations in 2024.

Antivirals/anti-infectives (20/01/2025)

Antivirals/anti-infectives

Cancer (20/01/2025)

Cancer

Dermatology (03/02/2025)

Dermatology

Gynaecology & Obstetrics (23/01/2025)

Gynaecology & Obstetrics

Haematology (03/02/2025)

Haematology

Metabolic system (31/01/2025)

Metabolic system

Nervous system (03/02/2025)

Nervous system

Ophthalmology (31/01/2025)

Ophthalmology

Respiratory system (03/02/2025)

Respiratory system

Vaccines (03/02/2025)

Vaccines

ACT EU multistakeholder platform annual meeting: report (22/10/2024)

The report of the ACT EU multistakeholder platform meeting, which took place on 22 October 2024, has been published.

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.

EMA workshop on the challenges in drug development, regulation and clinical practice for immunoglobulins (05/03/2025)

This online workshop will take place on 5 March 2025 to gain stakeholder perspectives on the use of immunoglobulins as a public consultation on a related scientific guideline is ongoing. Registration is possible by 3 March.

EMA Open Door Day (09/05/2025)

As part of its 30th anniversary, EMA will be hosting its first Open Door event on Europe day, 9 May 2025, featuring guided tours of the EMA building in Amsterdam and an exhibition of the Agency's archives. This is open to individual members of the public and free of charge.

Open consultations (20/01/2025)

Open consultations

Scientific committee and working party activities (20/01/2025)

Scientific committee and working party activities

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.

Policy on visiting and collaborating experts involved in the activities of the European Medicines Agency (20/01/2025)

EMA has published the policy on visiting and collaborating experts involved in EMA activities

How EMA engages with patients (19/01/2024)

EMA engages actively with patients and consumers since the creation of the Agency in 1995. Find out how they contribute and the added value they bring.

Welcome to the January 2025 issue (13/01/2025)

Welcome to the January 2025 issue of Human Medicines Highlights, EMA's monthly newsletter for patients, consumers, healthcare professionals and their representative organisations. I hope you have had a relaxing winter break, and this finds you in fine form to embrace 2025.

A common EU approach to data transparency in medicine regulation (10/01/2025)

EMA and HMA have updated their principles for identification and disclosure of commercially confidential information and personal data, reaffirming the commitment of EU/EEA regulatory authorities to extensive transparency when disclosing information.

New EU rules for health technology assessments become effective (13/01/2025)

EMA is ready to support the implementation of the new regulation on health technology assessment (Regulation (EU) 2021/2282) when it becomes applicable on 12 January 2025. The regulation is an important step forward in accelerating and widening access to new medicines.

Revised rules on handling of competing interests (14/01/2025)

The revised version of EMA policies on handling competing interests for scientific committee members and experts (‘Policy 0044’) and Management Board members (‘Policy 0058’) will apply as of May 2025. The revisions ensure alignment with the recent European Court of Justice findings.

Successful pilot paves the way for implementation of ePI (10/01/2025)

The report of a pilot exploring the creation and testing of electronic product information (ePI) in real regulatory procedures has been published, indicating general readiness for ePI and a need for further development.

Antivirals/anti-infectives (14/01/2025)

Antivirals/anti-infectives

Cancer (13/01/2025)

Cancer

Cardiovascular system (15/01/2025)

Cardiovascular system

Dermatology (13/01/2025)

Dermatology

Gastro-intestinal system (13/01/2025)

Gastro-intestinal system

Haematology (13/01/2025)

Haematology

HIV (13/01/2025)

HIV

Immune system (13/01/2025)

Immune system

Liver conditions (13/01/2025)

Liver conditions

Nervous system (13/01/2025)

Nervous system

Respiratory system (13/01/2025)

Respiratory system

Vaccines (14/01/2025)

Vaccines

Other medicines (15/01/2025)

Other medicines

Cancer Medicines Forum, October 2024: minutes and presentation (10/01/2025)

The minutes and presentation from The Cancer Medicines Forum held on 1 October 2024 have been published on the event page.

Fifth EMA/HMA Big Data Stakeholder Forum: video recording (28/11/2024)

A video recording of the fifth annual Big Data multi-stakeholder forum held on 28 November 2024 has been published on the event page.

Conversations on Cancer - Pediatric Cancers: video recording (19/11/2024)

The video recording of the online event Conversations on Cancer - Pediatric Cancers: Navigating the Challenges Together held on 19 November 2024 has been published on the event page.

EMA press briefing on human medicines in 2024 (16/01/2025)

On 16 January at 14:00-14:45 CET, EMA will hold its first press briefing of 2025 to discuss developments in human medicines in 2024. We will highlight the overarching trends and key advancements, with special considerations for medicines in neurology and psychiatric disorders. The briefing will be broadcast live.

ACT EU workshop on ICH E6 R3 (principles and Annex 1) (19/02/2025)

Registration for this hybrid multistakeholder workshop is no longer possible, but it can be followed through the live broadcast. For more details, please consult the events page.

Open consultations (10/01/2025)

Open consultations

Scientific committee and working party activities (10/01/2025)

Scientific committee and working party activities

CHMP Workplan 2025 (13/01/2025)

The CHMP has published its workplan for 2025 adopted in its meeting on 2 December 2024.

In this issue (12/12/2024)

Welcome to the December 2024 issue of Human Medicines Highlights, EMA's monthly newsletter for patients, consumers, healthcare professionals and their representative organisations. As we approach the end of yet another busy year, we take stock of many achievements and ongoing activities.

Medicine shortages: raising awareness and finding solutions together (10/12/2024)

Medicine shortages are a pressing issue, touching everyone in some way. From over-the-counter cough medicines to medicines for lifelong diseases, shortages can have a significant impact on the health of patients, costs and healthcare systems overall. Here we give an overview of the actions EMA is taking.

ACT EU highlights 2024 (12/12/2024)

The ACT EU (Accelerating Clinical Trials in the EU) initiative aims to enable better, faster and optimised clinical trials in multiple countries, making the EU an attractive region for clinical research that will benefit both patients and healthcare professionals.

ETF statement on the loss of activity of anti-spike protein monoclonal antibodies due to emerging SARS-CoV-2 variants: December 2024 update (16/12/2024)

EMA's Emergency Task Force (ETF) advises that monoclonal antibodies authorised to prevent and treat COVID-19 are unlikely to bring clinical benefit against the latest variants. Vaccination remains the best option to prevent disease caused by these variants.

Antivirals/anti-infectives (06/12/2024)

Antivirals/anti-infectives

Cancer (04/12/2024)

Cancer

Cardiovascular system (05/12/2024)

Cardiovascular system

Haematology (06/12/2024)

Haematology

Immune system (04/12/2024)

Immune system

Ophthalmology (04/12/2024)

Ophthalmology

Respiratory system (04/12/2024)

Respiratory system

Rheumatology (04/12/2024)

Rheumatology

Other medicines (06/12/2024)

Other medicines

Public event on advancing regulatory science research: presentations and recording (06/12/2024)

The presentations and video recording from the public event: advancing regulatory science research are now published on the EMA website.

Joint meeting of PCWP, HCPWP and eligible organisations: meeting summary and presentations (06/12/2024)

The meeting summary and presentations are now available from the annual joint meeting of the Patients' and Consumers' (PCWP) and Healthcare Professionals' (HCPWP) Working Parties meeting with all eligible organisations.

EMA workshop on hemoglobinopathies: meeting report (12/12/2024)

The meeting report of the EMA workshop on the challenges in drug development, regulation and clinical practice in hemoglobinopathies has been published on the event page.

Translating innovation into access for ATMPs: presentations and recording (12/12/2024)

Presentations and video recording are available for the event Translating innovation into access for ATMPs: third EU-Innovation network multi-stakeholder meeting, which took place on 15 November.

Enpr-EMA annual workshop: presentations (12/12/2024)

Presentations from the 2024 annual workshop of the European network of paediatric research at EMA (Enpr-EMA) are now available.

ACT EU workshop on ICH E6 R3 (principles and Annex 1) (19/02/2025)

The workshop aims to provide an overview of the major changes in the ICH E6(R3) guideline, highlight key concepts for adaptation of Good Clinical Practice to recent developments and enable discussion with stakeholders on the implementation of ICH E6(R3). It will be broadcast live.

Open consultations (06/12/2024)

Open consultations

Scientific committee and working party activities (06/12/2024)

Scientific committee and working party activities

Report of EMA's 2024 communication perception survey (12/12/2024)

The results of the fifth EMA communication perception survey, conducted in May-June 2024 are now available. The results and recommendations from this analysis will be used to establish baselines and targets to measure progress, analyse trends and improve our communication and engagement activities.

Alzheimer’s disease EU-IN Horizon Scanning Report (06/12/2024)

The EU Innovation Network works to identify emerging trends and topics in science and technology that have relevance to medical research and development. It delivers topic-specific reports that explore challenges and opportunities in these areas from a regulatory perspective.

EU NTC 10-year anniversary event report (12/12/2024)

In 2024 the EU Network Training Centre (EU NTC) celebrated its 10-year anniversary. The anniversary event highlighted past successes and outlined future plans to continue building capacity and capability across the EU for better patient and animal health outcomes; stakeholder groups also shared their experiences with similar networks, contributing valuable perspectives.

ETF statement on the use of Imvanex for the prevention of mpox in children below 12 years of age (03/12/2024)

The EMA Emergency Task Force (ETF) has considered the available evidence supporting the potential use of Imvanex in children younger than 12 years of age, in view of the need for mpox vaccines that can be used to protect children in the current public health emergency.

Take part in the MORE EUROPA survey on registries (16/12/2024)

The EU-co-funded More-EUROPA project is conducting a longitudinal survey study to identify stakeholders' perspectives on registries. Patients, consumers and healthcare professionals are invited to participate in the survey through the link by 31 January 2025.

In this issue - November 2024 (15/11/2024)

Welcome to the November 2024 issue of the Human Medicines Highlights newsletter, the monthly newsletter published by the European Medicines Agency specifically for patients, consumers, healthcare professionals and their representative organisations.

Antimicrobial resistance: fighting a silent pandemic (15/11/2024)

In Europe, more than 35,000 people die from antibiotic-resistant infections every year, with one in five bacterial infections already resistant to antibiotics. Without action, annual deaths could rise to 390,000 by 2050.

Fostering regulatory collaboration to improve access to mpox medicines (28/10/2024)

Global regulators have published a report highlighting their considerations on the development, clinical trials and availability of vaccines and therapeutics for mpox

Leqembi recommended for treatment of early Alzheimer’s disease (15/11/2024)

Following re-examination, EMA’s human medicines committee (CHMP) recommens granting a marketing authorisation to Leqembi (lecanemab) for treating early Alzheimer’s disease as its benefits outweigh risks in a restricted group of patients.

Translarna: EMA re-confirms non-renewal of authorisation of Duchenne muscular dystrophy medicine (28/10/2024)

Following a re-examination of available data, EMA’s human medicines committee (CHMP) has confirmed its previous recommendation to not renew the conditional marketing authorisation for Translarna (ataluren).

Updated advice to minimise risks of interaction between Mysimba and opioids (18/11/2024)

The weight loss medicine Mysimba (naltrexone/bupropion) must not be used in people who are dependent on opioids, people receiving treatment with opioid agonists such as methadone or buprenorphine and people going through acute opioid withdrawal.

Antivirals/anti-infectives (23/10/2024)

Antivirals/anti-infectives

Cancer (23/10/2024)

Cancer

Dermatology (23/10/2024)

Dermatology

Diabetes (22/10/2024)

Diabetes

Gastro-intestinal system (25/10/2024)

Gastro-intestinal system

Gynaecology & Obstetrics (24/10/2024)

Gynaecology & Obstetrics

Haematology (23/10/2024)

Haematology

Immune system (23/10/2024)

Immune system

Metabolic system (24/10/2024)

Metabolic system

Nervous system (23/10/2024)

Nervous system

Rheumatology (23/10/2024)

Rheumatology

Vaccines (24/10/2024)

Vaccines

Other medicines (23/10/2024)

Other medicines

Linkedin Live session: A call to action against antimicrobial resistance (06/11/2024)

On 11 November EMA organised a Linkedin live discussion with Steffen Thirstrup, Chief Medical Officer, Anastasia Pickford, project manager of EMA's Antimicrobial Sales and Use (ASU) Platform and Alessandro Faia, Press Officer. The recording can be viewed by logging in to LinkedIn.

Documents published: ATMP symposium "Contribution, evolution, revolution" (10/10/2024)

The presentations and recording from the Committee for Advanced Therapies' (CAT) symposium on advanced therapy medicinal products (ATMPs) are now published on the event page.

Report of the EMA workshop on the future of vaccine effectiveness studies in Europe (22/10/2024)

The full meeting report from this workshop has now been published on the EMA website.

Report of the joint HMA/EMA multi-stakeholder workshop on submission predictability (06/11/2024)

This multi-stakeholder workshop was organised to discuss ways to enhance submission predictability and to raise awareness of the implication and challenges that frequent and multiple delays to the submission dates pose on the Network’s resources and planning

Public event: advancing regulatory science research (18/11/2024)

This event will launch two initiatives: the 2024 draft update of the EMA Regulatory science research needs (RSRN) and the EMA/HMA European Platform for Regulatory Science Research. A recording will be made available afterwards. For more details please see the event page.

Conversations on Cancer - Pediatric Cancers: Navigating the Challenges Together (19/11/2024)

European Medicines Agency (EMA) in conjunction with FDA Oncology Center of Excellence (OCE) presents a one-hour "Conversations on Cancer" public panel discussion to examine an array of challenging decisions faced by members of the pediatric oncology community.

Joint meeting of PCWP, HCPWP and eligible organisations (20/11/2024)

The annual joint meeting of the Patients' and Consumers' (PCWP) and Healthcare Professionals' (HCPWP) Working Parties meeting with all eligible organisations will take place on 20 November 2024. The event is open to eligible organisations only but documents will be published on the events page.

Fifth EMA/HMA Big Data Stakeholder Forum (28/11/2024)

The Big Data Stakeholder Forum will reflect on the accelerating journey towards data-driven medicines regulation, consider data-enabled opportunities to improve medicines development in the EU. The event will be broadcast live and can be followed without registration.

Open consultations (17/10/2024)

Open consultations

Scientific committee and working party activities (17/10/2024)

Scientific committee and working party activities

October 2024 EMA Management Board meeting highlights (17/10/2024)

The Board elected a new vice-chair and heard updates on activities such EMANs, policy 0044 public consultation, and clinical trials. Read more in the highlights.

Expert report: Social Media Data for Real-World Evidence in Regulatory Decision Making (15/11/2024)

An expert report for the HMA/EMA Big Data Steering Group explores the potential advantages and challenges of leveraging social media data in regulatory decision-making, focusing on potential uses whilst acknowledging the challenges.

Expert report: mHealth data for real world evidence in regulatory decision-making (06/11/2024)

A report for the HMA/EMA Big Data Steering Group explores the utility of mobile health (mHealth) data from tools such as smartphones, health apps, smartwatches and other wearables for regulatory decision-making, their potential uses as well as challenges.

Proof-of-concept pilot on using data from clinical studies in medicines evaluation (24/10/2024)

EMA has published the interim report on experience gained with submission and analysis of patient-level (raw) data from clinical studies from September 2022 to December 2023. Regulators can already request this from applicants and the proposals for new pharmaceutical legislation foresees a systematic submission of such data at the time of initial marketing authorisation application.

ACT EU multi-stakeholder platform annual meeting (22/10/2024)

EMA is organising an annual meeting of the multi-stakeholder platform (MSP), established as part of the Accelerating Clinical Trials in the EU (ACT EU) initiative. Clinical trials stakeholders and regulators will come together and collaborate to improve the clinical trials environment for European patients and citizens.

In this issue (10/10/2024)

Welcome to the October 2024 issue of the Human Medicines Highlights newsletter. As usual, this issue is packed with the latest EMA news on human medicines. I would like to take the opportunity here to highlight a few important topics.

EMA recommends suspension of sickle cell disease medicine Oxbryta (27/09/2024)

EMA’s human medicines committee (CHMP) has recommended suspending the marketing authorisation for the sickle cell disease medicine Oxbryta (voxelotor). This measure is taken as a precaution while a review of emerging data is ongoing.

EMA recommends extending indication of mpox vaccine to adolescents (27/09/2024)

EMA has recommended extending the indication of the smallpox and mpox vaccine Imvanex to adolescents from 12 to 17 years of age.

Bruno Sepodes elected as new chair of EMA’s human medicines committee (CHMP) (19/09/2024)

Professor Sepodes started his 3-year mandate on 21 September. He has extensive experience working with EMA, including as member and vice-chair of the CHMP, member and chair of the Committee for Orphan Medicinal Products (COMP), and co-chair of EMA’s Emergency Task Force.

Seizing opportunities in a changing medicines landscape (10/10/2024)

EMA and the Heads of Medicines Agencies (HMA) have published their draft joint EU network strategy to 2028 for an eight-week public consultation.

Handling of competing interests: revised rules for committee members and experts (10/10/2024)

EMA is revising its policy on handling of competing interests of scientific committee members and experts (‘Policy 0044’). Stakeholders are invited to comment on the draft revised policy until 10 November 2024.

Antivirals/anti-infectives (23/09/2024)

Antivirals/anti-infectives

Cancer (23/09/2024)

Cancer

Dermatology (23/09/2024)

Dermatology

Diabetes (23/09/2024)

Diabetes

Gastro-intestinal system (23/09/2024)

Gastro-intestinal system

Gynaecology and obstetrics (27/09/2024)

Gynaecology and obstetrics

Haematology (27/09/2024)

Haematology

Immune system (27/09/2024)

Immune system

Metabolic system (27/09/2024)

Metabolic system

Nervous system (02/10/2024)

Nervous system

Ophthalmology (23/09/2024)

Ophthalmology

Respiratory system (23/09/2024)

Respiratory system

Urology (23/09/2024)

Urology

Vaccines (23/09/2024)

Vaccines

Other medicines (23/09/2024)

Other medicines

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

The EU Innovation Network (EU-IN) and the Italian Medicines Agency (AIFA) are organising a multi-stakeholder meeting 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.

Open consultations (19/09/2024)

Open consultations

Scientific committee and working party activities (19/09/2024)

Scientific committee and working party activities

Early dialogue with healthcare professional organisations for marketing authorisation applications: 1-year report (19/09/2024)

This pilot, supported by CHMP, aimed to capture patients' perspectives early in the evaluation of marketing authorisation applications. The insights gathered will assist assessors during their review, complement later engagement methods, and facilitate further interactions as the procedures progress.

Reflection paper on the use of artificial intelligence (AI) in the medicinal product lifecycle (02/10/2024)

This reflection paper provides considerations on the use of artifical intelligence/machine learning in the lifecycle of medicinal products, including medicinal products development, authorisation, and post-authorisation.

Reflection paper on establishing efficacy based on single arm trials submitted as pivotal evidence in a marketing authorisation application (08/10/2024)

This reflection paper outlines perspectives on single arm trials that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications.

From lab to patient: how EMA approves medicines (19/09/2024)

Follow the journey of a centrally authorised medicine: find out what happens at every stage and how stakeholders are involved. An interactive timeline is available in English and the PDF leaflet can be consulted in 22 European languages.

In this issue (07/08/2024)

Welcome to the September 2024 issue of the Human Medicines Highlights newsletter. As we start the busy autumn period, we bring you the latest updates on human medicines.

EMA recommends measures to minimise serious outcomes of known side effect with painkiller metamizole (09/09/2024)

Healthcare professionals should inform patients to look out for symptoms of agranulocytosis, a rare but serious side effect, and patients should seek immediate medical attention if symptoms arise. The product information will be updated to strengthen the existing warnings.

EMA publishes guidance on AI (12/09/2024)

EMA and the Heads of Medicines Agencies have published guiding principles that set out how EU medicine regulators can use large language models (LLMs) safely, responsibly and effectively. LLMs are a form of generative AI with many potential uses in medicines regulation, but also risks.

New pilot programme to support orphan medical devices (16/09/2024)

EMA has launched a pilot programme for expert panels to support the development and assessment of orphan medical devices in the EU. This programme offers free advice to selected manufacturers and notified bodies on the orphan device status and the data needed for clinical evaluation.

New chair elected for EMA’s Orphan Medicinal Products Committee (17/09/2024)

Tim Leest started his three-year mandate on 16 September. A Belgian national, Tim Leest has been a member of the COMP since 2017 and is a representative of EMA Patients’ and Consumers’ Working Party (PCWP) since 2019. COMP is responsible for evaluating applications for orphan designation for medicines developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious.

Cancer (14/08/2024)

Cancer

Diabetes (25/07/2024)

Diabetes

Haematology (23/08/2024)

Haematology

Immune system (23/08/2024)

Immune system

Nervous system (14/08/2024)

Nervous system

Pilot for expert panels' advice for orphan medical devices: information session (23/09/2024)

The online information session on 23 September will provide an overview of the new pilot and how manufacturers can apply for scientific support from expert panels. A recording will be made available on the event page afterwards.

ACT EU multi-stakeholder platform annual meeting (22/10/2024)

The ACT EU multi-stakeholder platform, established as part of the Accelerating Clinical Trials in the EU (ACT EU) initiative, will hold its annual meeting on 22 October 2024. The event will be broadcast and a video recording will be made available.

HMA/EMA multi-stakeholder workshop on artificial intelligence (AI) - enabling the safe and responsible use of AI (05/11/2024)

This workshop aims to update stakeholders on the state of the art of AI developments, including the revision of the AI reflection paper, activities under the multiannual AI workplan, and AI use-cases in the medicines lifecycle. In-person participation is by invitation and online participation is possible upon registration by 25 October.

Joint meeting of the Patients and Consumers' and Healthcare Professionals' Working Parties and all eligible organisations (20/11/2024)

The annual plenary meeting with representatives of all eligible patient, consumer and healthcare professional organisations will take place on 20 November 2024.

5th EMA/HMA big data stakeholder forum (28/11/2024)

The 5th big data stakeholder forum will take place on 28 November to discuss progress on data-driven medicines regulation with a wide range of stakeholders including EU regulators, industry, innovators, patients and healthcare professionals. Participation is by invitation. A live broadcast can be followed on the day without registration.

Open consultations (14/08/2024)

Open consultations

Scientific committee and working party activities (14/08/2024)

Scientific committee and working party activities

EMA guidance document on the use of medicinal products for treatment in case of exposure to chemical agents used as weapons of terrorism, crime, or warfare (23/08/2024)

This guidance has been updated after a review of the scientific literature and existing guidelines and a consultation with a panel of European experts in toxicology, pharmacology, clinical management, and military aspects related to chemical agents and other toxic chemicals

Guiding principles on the use of large language models in regulatory science and for medicines regulatory activities (16/09/2024)

This document provides guiding principles on the use of general-purpose large language models (LLM) in regulatory science and for regulatory activities and provides high-level recommendations to regulatory authorities to facilitate the safe, responsible and effective use of LLMs.

From lab to patient: how EMA approves medicines (05/09/2024)

Follow the journey of a centrally authorised medicine: find out what happens at every stage and how stakeholders are involved. An interactive timeline is available in English and the PDF leaflet can be consulted in 22 European languages.

EMA advises about risks of using weight loss medicine Mysimba with opioids (12/08/2024)

EMA recommends that the weight loss medicine Mysimba (naltrexone/ bupropion) must not be used in people receiving treatment with opioid medicines. EMA has given this advice following a routine safety review. Read more here.

EMA supports pilot for joint African continental assessment procedures (02/08/2024)

EMA has awarded a grant for a pilot to test and validate procedures for joint continental evaluation of medicines ahead of the establishment of the African Medicines Agency.

EMA confirms its recommendation to update the composition of COVID-19 vaccines for 2024-2025 (23/07/2024)

EMA's Emergency Task Force reconfirmed its April recommendation on updating COVID-19 vaccines' composition to target the new SARS-CoV-2 variant JN.1 for the 2024/2025 vaccination campaign.

New appointment in EMA's management team (23/07/2024)

Georgia Gavriilidou joined EMA as the new Head of the Legal Department following the retirement of Stefano Marino.

Antivirals/anti-infectives (06/08/2024)

Anti-infectives

Cancer (18/07/2024)

Cancer

Cardiovascular system (25/07/2024)

Cardiovascular system

Dermatology (30/07/2024)

Dermatology

Gastro-intestinal system (30/07/2024)

Gastro-intestinal system

Gynaecology & Obstetrics (23/07/2024)

Gynaecology & Obstetrics

Haematology (23/07/2024)

Haematology

Hepatology (14/08/2024)

Hepatology

HIV (01/08/2024)

HIV

Immune system (18/07/2024)

Immune system

Metabolic system (01/08/2024)

Metabolic system

Nephrology (01/08/2024)

Nephrology

Nervous system (16/07/2024)

Nervous system

Ophthalmology (25/07/2024)

Ophthalmology

Rheumatology (18/07/2024)

Rheumatology

Respiratory (30/07/2024)

Respiratory

Urology (01/08/2024)

Urology

Vaccines (01/08/2024)

Vaccines

Clinical Trials Regulation Collaborate Stakeholder meeting (11/09/2024)

A meeting will take place on 11 September 2024 to share insights from the CTR Collaborate initiative and to bring the views of stakeholders. For information on this initiative, please visit the event page. Registration is open until 30 August.

Meeting report of the multistakeholder workshop on psychedelics published (15/05/2024)

The full meeting report from this workshop, held in April, has now been published on the event page, together with the presentations and video recordings.

Interview on GLP-1 medicines with Dr Bart van der Schueren (08/08/2024)

The Instagram LIVE session with Dr Bart van der Schueren, seconded national expert at EMA, is now available to view on our YouTube channel.

Patients, consumers and healthcare professionals' joint meeting summary published (25/07/2024)

EMA has published the summary of the joint meeting of the Patients and Consumers Working Party and Healthcare Professionals Working Party, which took place on 2 and 3 July 2024.

Patients and consumers' July meeting summary published (25/07/2024)

EMA has published the meeting summary of the Patients and Consumers Working Party meeting that took place on 2 July 2024.

Healthcare professionals' July meeting summary published (07/08/2024)

EMA has published the meeting summary of the Healthcare Professionals Working Party that took place on 3 July 2024.

Open consultations (16/07/2024)

Open consultations

Scientific committee and working party activities (16/07/2024)

Scientific committee and working party activities

New EMA report on studies using real-world data (07/08/2024)

A second annual report on studies EMA conducted using real-world data summarises progress in integrating real-world data in regulatory decision-making, including research topics and use cases. The report, annexes and an info sheet are available on the EMA’s real-world evidence page.

Real-world evidence to support EU regulatory decision making - results from a pilot of regulatory use cases (07/08/2024)

A peer-reviewed article has been published to present the experience gained from EMA's pilot to generate real-world evidence to support evaluations by EU regulators, based on studies conducted between September 2021 and February 2023, highlighting key learnings to guide further efforts.

Revised guideline on good pharmacovigilance practices on risk minimisation measures (08/08/2024)

Revision 3 of Module XVI on risk minimisation measures together with its Addendum II on methods for the effectiveness evaluation of such measures have been published, taking into account comments received from public consultation and recent research. Healthcare professional and patient representatives contributed to the document.

Updated introductory note on good pharmacovigilance practice (08/08/2024)

The updated note gives an overview of good pharmacovigilance practice, including its legal basis, history and development, the structure of EU pharmacovigilance system and the roles of different actors.

From lab to patient: how EMA approves medicines (23/01/2024)

Follow the journey of a centrally authorised medicine: find out what happens at every stage and how stakeholders are involved. An interactive timeline is available in English and the PDF leaflet can be consulted in 22 European languages.

In this issue - July 2024 (09/07/2024)

Welcome to the July 2024 issue of the Human Medicines Highlights newsletter, the monthly newsletter published by the European Medicines Agency specifically for patients, consumers, healthcare professionals and their representative organisations. This month, we bring you updates from a multistakeholder workshop on GLP-1 medicines and the new recommendations for managing shortages of these medicines.

Multistakeholder workshop on GLP-1 medicines shortages (15/07/2024)

On 1 July 2024, stakeholders came together at EMA for a workshop to discuss the impact of, and solutions for, the shortages of glucagon-like peptide-1 (GLP-1) receptor agonists, including Ozempic, Victoza, Saxenda and Wegovy.

EU actions to tackle shortages of GLP-1 medicines (08/07/2024)

The Executive Steering Group on Shortages and Safety of Medicinal Products has issued recommendations to all stakeholders to tackle shortages of GLP-1 receptor agonists.

Faster access to clinical trial information in Europe (20/06/2024)

The launch of the new version of the Clinical Trials Information System will allow earlier and more efficient access to information about clinical trials in the European Union for patients, healthcare professionals and other stakeholders.

Two new advice pilots to improve clinical trials in Europe (20/06/2024)

The Accelerating Clinical Trials in the EU (ACT EU) initiative has launched two advice pilots aimed at improving the quality of applications for clinical trials, which is the foundation for the development of safe and effective medicines in Europe.

EMA’s safety committee elects new Chair (10/07/2024)

EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) has elected Ulla Wändel Liminga as its new Chair for a three-year mandate starting in September 2024.

EMA recommends revoking conditional marketing authorisation for Ocaliva (12/07/2024)

EMA ’s human medicines committee (CHMP) has concluded its review of the medicine Ocaliva (obeticholic acid) and has recommended that the medicine’s marketing authorisation be revoked, because its benefits are no longer considered to outweigh its risks.

Hydroxyprogesterone caproate medicines to be suspended from the EU market (12/07/2024)

Review of studies raises possible safety concern and finds no effect in preventing premature birth.

Antivirals/anti-infectives (02/07/2024)

Antivirals/anti-infectives

Cancer (20/06/2024)

Cancer

Cardiovascular system (09/07/2024)

Cardiovascular system

Dermatology (09/07/2024)

Dermatology

Diabetes (02/07/2024)

Diabetes

Gynaecology & Obstetrics (12/07/2024)

Gynaecology & Obstetrics

Haematology (20/06/2024)

Haematology

Immune system (20/06/2024)

Immune system

Ophthalmology (09/07/2024)

Ophthalmology

Nervous system (20/06/2024)

Nervous system

Ophthalmology (02/07/2024)

Ophthalmology

Respiratory system (20/06/2024)

Respiratory system

Rheumatology (09/07/2024)

Rheumatology

Urology (04/07/2024)

Urology

Vaccines (04/07/2024)

Vaccines

Other medicines (04/07/2024)

Other medicines

Multistakeholder workshop on shortages of GLP-1 receptor agonists: presentations published (09/07/2024)

EMA has published the presentations from this workshop held on 1 July 2024 on the event page.

HMA/EMA workshop on real-world evidence methods: recording published (10/07/2024)

The video recording of this workshop, which took place on 14 June 2024, is now available on the event page.

EMA workshop on haemoglobinopathies: presentations published (10/07/2024)

Presentations from the workshop on haemoglobinopathies, held on 1 July 2024, are available on the event page.

Open consultations (09/07/2024)

Open consultations

Scientific committee and working party activities (09/07/2024)

Scientific committee and working party activities

Public Engagement Highlights 2023 (10/07/2024)

The Public Engagement Highlights is a concise annual overview of the involvement of patients and healthcare professionals in the work of EMA.

EMA annual activity report 2023 (20/06/2024)

The Agency’s consolidated annual activity report provides an overview of its activities and achievements of in 2023. The report is a key component of the strategic planning and programming cycle and the basis upon which the Executive Director takes their responsibility for the management of resources and achievement of objectives.

A decade of collaboration in medicines regulation: healthcare professionals engaging with EMA (15/07/2024)

An article published on 5 June in Frontiers in Medicine highlights the role of healthcare professionals (HCPs) in the EU regulatory process and takes stock of the first 10 years of EMA’s framework for interaction with HCPs. Read about how this collaboration started and evolved.