"Clinical Trials Highlights" Newsletter Issues

Logos (12/07/2023)

This newsletter provides the latest news on clinical trials from the European Medicines Regulatory Network (EMRN), including news on the joint Accelerating Clinical Trials in the EU (ACT EU) initiative and on the Clinical Trials Information System (CTIS).

Intro (11/04/2024)

Welcome to the April 2024 issue of Clinical Trials Highlights.

Multi-stakeholder platform Advisory Group established (11/04/2024)

The first meeting of the MSP Advisory Group on 20 March 2024 officially launched the ACT EU Multi-stakeholder platform (MSP). The establishment of the MSP is a key milestone for the ACT EU initiative, as it provides a channel for stakeholders to exchange views and voice priorities on various aspects of clinical research.

Consolidated advice pilots (11/04/2024)

In Q2 2024, ACT EU is launching two pilots offering: a) harmonised scientific advice to support the submission of marketing authorisation and clinical trial applications (CTAs) in the EU, and b) consolidated technical/regulatory advice on CTA dossiers prior to their submission in CTIS.

Outcome of SNSA training webinar (11/04/2024)

The new pilots complement the existing pilot on National Scientific Advice (SNSA). On 19 April, over 1100 viewers followed a webinar hosted by ACT EU and the EU Innovation Network, providing information on the ongoing SNSA pilot and how it can be used, highlighting benefits for applicants, and answering their questions. A video recording will be available on the event page in due course.

Report of clinical trial analytics workshop (11/04/2024)

The ACT EU workshop on 25-26 January 2024 highlighted a collective ambition to improve the use of data about clinical trials, with a multi-stakeholder approach to identify research priorities. This will speed up the development of an analytics ecosystem that maximises health benefits for EU citizens. A report summarising the discussion has been published on the ACT EU website.

Report of methodology workshop (11/04/2024)

The ACT EU multi-stakeholder workshop on methodology guidance facilitated discussions on key topics of clinical trial methodology in order to identify the challenges, propose ways for improving patient centricity and suggest possible solutions. An outcome report has been published, summarising the discussions in all the break-out sessions and identifying common themes.

Summary gap analysis of assessor training needs (11/04/2024)

As part of the work to deliver a clinical trials training curriculum, ACT EU worked with stakeholders from the regulatory network to identify the training needs of assessors of clinical trials. A summary of the gap analysis has been published on the ACT EU website.

Recent improvements in CTIS (11/04/2024)

The latest improvements include enhancements in the authorisation and supervision of trials; collaboration between Member States on ad-hoc/safety information; communication between sponsors and Member States; display of trial sites in saved/submitted applications. More details on recent improvements are available on the CTIS website.

Updated guidance for sponsors transitioning trials to CTR (11/04/2024)

Sponsors transitioning their ongoing trials from EudraCT to CTIS may now consult the updated guidance documents from the European Commission and CTCG. All materials from the European Medicines Regulatory Network are available on the CTIS website, under the section “Transitioning Trials”.

Access to CTIS Training Environment (11/04/2024)

Sponsors can express their interest in gaining access to the CTIS Training Environment by filling in the open survey. The training environment is a simulation of CTIS used in production and allows users to get familiar with system functionalities.

CTIS bitesize talk 24 April 2024 (24/04/2024)

This CTIS bitesize talk aims to provide sponsors with information on alternate Investigational Medicinal Product Dossier – Quality (IMPD-Q) and new guidance on Auxiliary Medicinal Products (AxMP).

CTIS walk-in clinic 15 May 2024 (15/05/2024)

CTIS walk-in clinics allow sponsors to raise questions about any CTIS functionality and receive advice from CTIS experts. CTIS users can submit and upvote questions in advance via Slido until 8 May, with the code #clinic245.

CTIS training for sponsor users (10/06/2024)

EMA offers a virtual training programme, organised by DIA, to support sponsor user preparedness for CTIS. A hands-on approach is taken to explain and demonstrate the functionalities of the system, such as user management, how to submit an initial application as well as modifications, both substantial and non-substantial.

Open consultation until 31 May 2024 (11/04/2024)

Interested stakeholders are invited to provide their feedback until 31 May 2024 on the concept paper on the development of a guideline on non-inferiority and equivalence comparisons in clinical trials. Comments should be provided using this EU Survey form.

Final year of transition to the Clinical Trials Regulation (12/02/2024)

Welcome to the February 2024 issue of Clinical Trials Highlights.

Multi-stakeholder platform (MSP) Advisory Group (12/02/2024)

The composition of the MSP Advisory Group is now published, including key stakeholders’ representatives who will meet regularly to provide advice on ACT EU activities. The Head of the Spanish Medicines Agency, María Lamas, is the regulatory co-chair. The process to select the stakeholder co-chair will be launched at the group's inaugural meeting on 20 March 2024.

Save the date: SNSA training webinar on 19 April 2024 (14/02/2024)

ACT EU and the EU Innovation Network are organising a webinar on Simultaneous National Scientific Advice (SNSA) on 19 April 2024. The event will provide information on the ongoing SNSA pilot and how it can be used, present the optimised procedure for the pilot’s phase 2, highlight benefits for applicants, and answer questions. An event page will soon be available on the EMA website.

Clinical trial analytics workshop (12/02/2024)

Over 200 participants joined the workshop on Clinical Trials Analytics on 25-26 January 2024. Discussions highlighted the need for improved access to detailed and up-to-date clinical trial data, as well as the importance of patient involvement, standardisation, and identification of the right data sources. The video and presentations are available on the event page.

SAFE CT training for clinical trial safety assessors (01/02/2024)

On 15-16 January 2024 EMA hosted the annual SAFE-CT Safety Assessor event, as part of the activities of ACT EU’s priority action on safety monitoring.

Transparency rules in CTIS (12/02/2024)

EMA is working on the technical implementation of the revised CTIS transparency rules, foreseen for Q2 2024. In the meantime, for initial clinical trials applications sponsors may already follow the principles of the revised CTIS transparency rules. Further information is available under section 4 of the updated Q&A on protection of confidential information and personal data in CTIS.

Recent improvements in CTIS (12/02/2024)

Latest improvements include enhancements in application creation, preparation of documents and data; authorisation and supervision of trials; downloading information from the public portal; communication between sponsors and MS; user registration and authentication. More details on recent improvements are available on the CTIS website.

Resources for sponsors transitioning trials to CTR (12/02/2024)

Clinical trials authorised under the Clinical Trials Directive likely to be ongoing beyond 30 January 2025 need to be transitioned to the Clinical Trials Regulation. Resources and guidance from the European Medicines Regulatory Network are available on the CTIS website, under the section “Transitioning Trials”.

Access to CTIS Training Environment (12/02/2024)

Sponsors may express their interest in accessing the CTIS training environment via an open survey, which collects information to identify the needs and intention for use of CTIS and support decisions on granting access to the CTIS Training Environment. Access will expire after 6 months to allow as many sponsors as possible to benefit from the training environment.

Save the date for upcoming CTIS events (12/02/2024)

EMA and the EMRN continue to provide training events and information sessions to support CTIS users. All EMA-run events are live broadcast and a video recording is made available after each session on the respective event pages.

Revised SME user guide (29/06/2023)

EMA has released a major revision of its user guide for micro, small, and medium-sized enterprises (SMEs) in the pharmaceutical sector.

Big Data Steering Group - Annual report 2023 (12/02/2024)

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Clinical trials ‘raw data’ pilot: last chance to register (12/02/2024)

The pilot, which will include ten regulatory procedures in total, recently selected its sixth and seventh procedures: two initial marketing authorisation applications in oncology and gastroenterology.

Implementation of multi-factor authentication (12/02/2024)

As part of continuous efforts to further reinforce the security of user accounts, since January 2024 the EMA Account Management platform has introduced Multi Factor Authentication (MFA). Detailed instructions on setting up MFA for EMA systems are available.

Enhancements in the management of organisation merges (12/02/2024)

Users with User Administrators roles in EMA Account Management, e.g. CTIS Member State Administrators or CTIS Sponsor Administrators, receive email notifications if their organisation has been merged with any identical duplicate organisation(s).

Opt-in to email authentication for EMA systems (12/02/2024)

EMA has introduced Bring Your Own Identity (BYOI) authentication capabilities, to improve security and usability of EMA applications. BYOI allows email address authentication for EMA applications, removing the need for users to remember their EMA username and password. Users can opt-in to email authentication in EMA Account Management by following the detailed instructions.

Spotlight on CTCG (07/11/2023)

Welcome to the November 2023 issue of Clinical Trials Highlights.

ACT EU workplan for 2023-2026 published (14/11/2023)

The revised workplan outlines the updated priorities for ACT EU, taking into account feedback from the Multi-stakeholder platform (MSP) kick-off and public health emergencies workshops in June 2023. The full plan is available on the ACT EU website.

Multi-stakeholder platform: Call for MSP Advisory Group members (14/11/2023)

The Multi-stakeholder platform Advisory Group is an opportunity for key stakeholder groups to advise on clinical trials topics. A call for stakeholder representatives to the group is open until 24 November 2023.

Clinical Trials Methodology workshop on 23 November 2023 (23/11/2023)

During the workshop, participants will assess the need for new or revised methodology guidance documents on selected clinical trials topics. Interested stakeholders can follow the workshop via a live broadcast. More information and a detailed agenda are available on the event page.

Recent improvements in CTIS (14/11/2023)

Latest improvements include the 24/7 security monitoring of CTIS through EMA’s Security Operations Centre; token-based authentication to improve security in the Member State API; and other enhancements in application creation, authorisation and supervision of trials, communication between sponsors and MS, user registration. More details on recent improvements are available on the CTIS website.

Transparency rules in CTIS (14/11/2023)

In October 2023, the EMA Management Board adopted the revised transparency rules in CTIS. The simplifications will ensure earlier access to key clinical trial information for patients and healthcare professionals. The revised transparency rules will apply after their technical implementation in CTIS, including its public portal, which is foreseen in the second quarter of 2024.

Access to CTIS Training Environment (14/11/2023)

Sponsors may express their interest in accessing the CTIS training environment via an open survey, which collects information to identify the needs and intention for use of CTIS and support decisions on granting access to the CTIS Training Environment. Access will expire after 6 months to allow as many sponsors as possible to benefit from the training environment.

CTIS training material updates (14/11/2023)

Module 10 of the CTIS modular training programme on how to “Create, submit and withdraw a clinical trial” has been updated to clarify frequently asked questions; provide details on partial submission of trials under the CTR; add details of medical devices associated with medical product of CTA under CTR. Additionally, the references to other sources in Module 13 have been updated.

Resources for sponsors transitioning trials to CTR (14/11/2023)

Clinical trials authorised under the Clinical Trials Directive likely to be ongoing beyond 30 January 2025 need to be transitioned to the Clinical Trials Regulation. Resources and guidance from the European Medicines Regulatory Network are available on the CTIS website, under the section “Transitioning Trials”.

Recent and upcoming CTIS events (13/09/2023)

EMA and the EMRN continue to provide training events and information sessions to support CTIS users. All EMA-run events are live broadcast and a video recording is made available after each session on the respective event pages.

"COMBINE" project (16/11/2023)

Member State’s competent authorities for clinical trials and medical devices and the European Commission have published information on the "COMBINE" project, which addresses the challenges at the interface between the Regulations on clinical trials of medicinal products, medical devices and in vitro diagnostics.

Open consultation: reflection paper on AI in medicines (14/11/2023)

Interested stakeholders are invited to provide their feedback until 31 December 2023 on the draft reflection paper on the use of artificial intelligence (AI) to support the safe and effective development, use and regulation of human and veterinary medicines.

Halfway point on clinical trials ‘raw data’ pilot: register to participate (14/11/2023)

Applicants or marketing authorisation holders (MAHs) that are planning to submit an initial or post-authorisation marketing authorisation application to EMA during the first quarter of 2024 are still welcome to express their interest to participate in the proof-of-concept pilot by writing to rawdatapilot@ema.europa.eu.

Welcome to the September 2023 issue of the Clinical Trials Highlights (13/09/2023)

Welcome to the September 2023 issue of the Clinical Trials Highlights.

Launch of the ACT EU website (13/09/2023)

A dedicated website has been launched on the ACT EU initiative, co-led by the European Commission, HMA and EMA. The website contains key resources, latest news and upcoming events, including updates on the Multi-stakeholder platform, the implementation of the CTR, and information on scientific advice procedures such as the Simultaneous national scientific advice (SNSA) pilot.

Multi-stakeholder platform: kick-off meeting report (20/09/2023)

A first multi-stakeholder platform workshop was held in June 2023, to understand different stakeholder perspectives on how to transform the EU environment for clinical trials and agree on a model for the establishment of the platform. The summary report of the workshop is now available on the ACT EU website.

Clinical trial methodology workshop 23 November 2023 (13/09/2023)

ACT EU is hosting a multi-stakeholder methodology workshop on 23 November 2023. The focus will be on key topics proposed by stakeholders, to discuss the status of methodology guidance documents and assess the need for new or revised guidance. A call to register and propose discussion topics concluded on 17 September 2023. A detailed agenda will be published in due course.

Outcome of the ICH E6 R3 workshop on 13-14 July 2023 (13/09/2023)

The virtual workshop was attended by around 1000 relevant stakeholders, including patients, healthcare professionals, assessors, inspectors, industry and academia. A summary report of the outcomes is available on the event page.

CTR implementation: Revised KPI report (13/09/2023)

A revised monthly report on the implementation of the CTR will soon be available on the ACT EU website, with Key Performance Indicators on clinical trials (CTs) transitioned from the Clinical Trials Directive to CTR. Data on the EudraCT database, which is no longer in use for submitting new CT applications (while still in use for ongoing CTs and posting of results), will no longer be reported.

CTR implementation: Survey of sponsor users (13/09/2023)

Following the survey led by the European Commission's Directorate-General for Health and Food Safety (DG SANTE) in 2022, sponsor users with clinical trials with a decision in CTIS have now been invited to fill in a follow-up survey to monitor the implementation of the CTR and CTIS use. The feedback will be analysed to identify solutions that will further support sponsors in implementing the CTR.

Clinical trials in public health emergencies (13/09/2023)

The scope of ACT EU has been expanded to include activities enabling multinational clinical trials in the EU during public health emergencies. This will tackle different aspects of the regulatory approval process to enable large trials by increasing collaboration across regulators and ethics committees, and the role of the EMA Emergency Task Force in fostering alignment across Member States.

Recent improvements in CTIS (13/09/2023)

Latest improvements include a faster, more reliable search for organisations in the Organisation Management Service (OMS) via CTIS and other enhancements in the preparation of documents, authorisation and supervision of trials. More details on recent improvements are available on the CTIS website.

Transparency rules in CTIS (13/09/2023)

The public consultation on the CTIS transparency rules, which was launched on 3 May 2023, concluded on 28 June 2023. Over 200 responses were received from CTIS users and stakeholders. The feedback reflects diverse views on the use of deferrals and redaction functionalities, with agreement on the need for simplification.

Revised guidance on transitioning trials (18/09/2023)

The CTCG has published revised versions of the Best Practice Guide and cover letter template for sponsors transitioning multi-national clinical trials to the Clinical Trials Regulation / CTIS, available under news and events on the group's webpage.

Updated CTR Quick guide for sponsors (13/09/2023)

The European Commission has published a new version of the CTR Quick Guide for sponsors. This revised version includes clarifications on how to submit the IMPD-Q on the manufacturing of the Investigational Medicinal Product in case this is done at a decentral point of care, at one of the clinical trial sites in the additional Member State.

Access to CTIS Training Environment (29/06/2023)

Sponsors may express their interest in accessing the CTIS training environment via an open survey, which collects information to identify the needs and intention for use of CTIS and support decisions on granting access to the CTIS Training Environment. Access will expire after 6 months to allow as many sponsors as possible to benefit from the training environment.

Open consultations (13/09/2023)

Interested stakeholders are invited to provide their feedback in the following open consultations:

Introduction (29/06/2023)

Welcome to the July 2023 issue of Clinical Trials Highlights.

Feedback from kick-off meeting of the multi-stakeholder platform (29/06/2023)

With a view to understand different stakeholder perspectives on how to transform the EU environment for clinical trials, the kick-off meeting of the multi-stakeholder platform (MSP) took place on 22 and 23 June 2023.

Upcoming sponsor survey on CTR implementation (29/06/2023)

The follow-up sponsor survey on the CTR implementation is foreseen to be launched in September 2023.

Update on the ICH E6 R3 Workshop on 13-14 July 2023 (10/07/2023)

Following the revision of the ICH E6 R2 guideline on Good Clinical Practice (GCP) that aims to address the application of GCP to the increasingly diverse range of clinical trial types and data sources, ACT EU Priority Action 4 on GCP Modernisation held a Workshop on the ICH E6 R3 Public Consultation (principles and Annex 1) on 13 and 14 July 2023.

Overarching methodologies workshop on 22-23 November 2023 (29/06/2023)

With a view to progress the alignment process and ensure stakeholder needs are recognised and incorporated, an on-site multi-stakeholder Methodology Workshop will be held on 22-23 November 2023. The focus will be on discussing key topics related to Methodology guidance, with the aim to identify guidance needs and to propose scopes.

Open consultation on single-arm trials reflection paper (29/06/2023)

As previously announced, a reflection paper has been drafted to outline considerations on single-arm trials that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications. The reflection paper remains open for public consultation on the EMA website. Stakeholders are invited to send their comments by 30 September 2023.

CTIS now a registered WHO data provider (29/06/2023)

Data from authorised trials published on the CTIS website - excluding those with deferrals - is now included in the search portal of WHO’s International Clinical Trials Registry Platform (ICTRP). This applies to relevant clinical trial data, as required by WHO, which has been published on CTIS since the launch of the system on 31 January 2022.

Move of CTIS User Service to ServiceNow platform (29/06/2023)

In alignment with the EMA’s information security strategy, a new IT service management solution called ServiceNow will replace the current tool (JIRA) for CTIS User Support Service (USS) requests, starting on 31 July 2023.

Recent improvements in CTIS (05/07/2023)

Latest improvements include the implementation of multi-factor authentication to enhance security of user accounts; the migration to high availability infrastructure to enhance system performance; and the enhancement of the WHO API to improve the process of data extraction from the CTIS public portal. More details on recent improvements are available on the CTIS website.

Guidance on protection of personal data and CCI in CTIS – final document published (05/07/2023)

The final guidance document on the protection of personal data and commercially confidential information while using the current transparency rules for CTIS is now available on the EMA website, including chapter 4 on protection of CCI and chapter 5 on GCP inspection reports.

Review of CTIS transparency rules (05/07/2023)

The public consultation on the CTIS transparency rules, which was open from 3 May to 28 June 2023, is now concluded. EMA is currently reviewing the comments received, with the aim of publishing a concept paper with the proposed simplified transparency rules later this year.

Transitional Trials: dedicated workshop and updated CTCG guidance (13/07/2023)

In collaboration with the Member States and the European Commission, EMA held a Workshop on 5 July 2023 on the joint actions for transitioning ongoing trials to the Clinical Trials Regulation.

Recent and upcoming CTIS events (29/06/2023)

EMA and the EMRN continue to provide training events and information sessions to support CTIS users. All EMA-run events are live broadcast and a video recording is made available after each session on the respective event pages.

ICH M11 Scientific guideline (29/06/2023)

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