Updates - December 2023 - January 2024
Clinical Trials' Transition to New EU System – One Year Left
All ongoing clinical trials in the EU must be transitioned to the Clinical Trials Information System (CTIS) by 31 January 2025. This date marks the end of a three-year transition period that began when the Clinical Trials Regulation (CTR) became applicable in the EU.
Clinical Data Publication
By proactively publishing clinical data, EMA intends to help:
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avoid duplication of clinical trials, foster innovation and encourage development of new medicines;
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build public trust and confidence in EMA's scientific and decision-making processes;
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academics and researchers to re-assess clinical data.
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“Resumption of clinical data publication for all medicines” was updated.
Big Data Steering Group (BDSG): 2023 Report
2023 marked significant progress in the transformation to data-driven regulation continued, in line with the Network Strategy to 2025 and BDSG workplan. The fourth BDSG workplan was published in July 2023 to progress the activities launched in 2020 and to address new topics.
This report is based on the third BDSG workplan and provides a summary of the key activities and achievements of the BDSG in 2023.
Major Update of the EMA User Guide for Micro, Small and Medium-sized Enterprises (SMEs)
The revised user guide offers comprehensive information on the EU legislative framework for medicines, outlining requirements for the development and authorization of medicines for human and veterinary use. It follows the chronological stages of medicine development, and has become a reference source of information for SME and academic developers, supporting them to navigate the system of medicine regulation in the EU. The new release incorporates significant updates to reflect major changes in the EU’s legal and regulatory framework for human and veterinary medicines:
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Veterinary Regulation: the document has been fully revised to align with the veterinary regulation
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Clinical Trials Regulation (new section 4.4): provides an overview of the clinical trial regulation and Clinical Trials Information System (CTIS)
Medical Devices Regulation (new section 4.8): offers insights into medical device regulation for human medicines
Policy on the Determination of the Condition(s) for a Pediatric Investigation Plan/Waiver (Scope of the PIP/Waiver)
The aim of this policy is to propose a systematic approach based on the characteristics of the product and an independent classification of diseases and conditions to provide a more reliable and predictable framework for applicants and the PDCO in identifying the scope of a PIP or a waiver, and to facilitate the evaluation.
Human Medicines: Highlights of 2023
The overview of the 2023 key recommendations published includes figures on the authorization of medicines and a selection of new treatments that represent significant progress in their therapeutic areas as well as newly issued negative opinions for 3 medicines and guidance on additional uses for 77 existing medicines.
Pharmacovigilance Inspectors' Working Group (PhV IWG)
Annual report of the Pharmacovigilance Inspectors' Working Group for 2021 and 2022
Work plan for the Pharmacovigilance Inspectors' Working Group (PhV IWG) for 2024-2026
eXtended EudraVigilance Medicinal Product Dictionary (XEVMPD) Training Course (self-paced)
The training focuses on:
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Explaining the guidance and specifically the mandatory data elements necessary for the electronic submission of information on authorized and unauthorized (referred to in the XEVMPD as 'development') medicinal products;
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Applying the format of the eXtended EudraVigilance Product Report Message (XEVPRM);
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The use of the XEVMPD data entry tool (EVWEB).
It includes exercises in the XEVPRM data entry tool (EVWEB) for the electronic submission and maintenance of development medicinal products.
ICH Q2(R2) Guideline on Validation of Analytical Procedures
This guideline presents elements for consideration during the validation of analytical procedures included as part of registration applications. ICH Q2(R2) provides guidance on selection and evaluation of the various validation tests for analytical procedures. This guideline includes a collection of terms and their definitions, which are meant to bridge the differences that often exist between various compendia and documents of the ICH member regulatory authorities.
EudraVigilance Training and Support
The dates for the virtual live hands-on training course on using the enhanced EudraVigilance system are as follows:
European Medicines Agency Pre- and Post- Authorization Procedural Advice for Users of the Centralized Procedure
The following questions were revised:
Pre-Authorization (link to questions)
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2.4. What is the procedure for appointment of Rapporteurs/Co-Rapporteurs and their assessment teams?
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5.1.11. Can EMA assessment or inspection documents be shared with regulators outside the EU?
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5.1.12. How can I change the applicant for an ongoing marketing authorization application?
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5.2.1. When can I expect a pre-authorization GMP inspection and how are they conducted?
Post-Authorization (link to questions)
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1.6. Can my Type IA/ IAIN be part of worksharing?
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16.14. When and how will the RMP Summary be published on the EMA website?
Products Management Services (PMS) – Implementation of International Organization for Standardization (ISO) Standards for the Identification of Medicinal Products (IDMP) in Europe
The EMA intends to migrate the Centrally Authorized Products (CAPs) and non-Centrally Authorized Products (non-CAPs) data held in the eXtended Eudravigilance Medicinal Product Dictionary (XEVMPD) and submitted by marketing authorization holders (MAHs) under the Art.57 (2) legal obligations into the ISO IDMP-compliant data format and terminologies.
This chapter provides information on the approach followed by the EMA to enable the transformation and migration of the data to the PMS.
Concept Paper on the Revision of the Guideline on the Clinical Evaluation of Medicinal Products Intended for the Treatment of Hepatitis B
(submit comments by 30 April 2024)
This concept paper addresses the need to update the guideline on the clinical evaluation of medicinal products intended for the treatment of Hepatitis B (CHMP/EWP/6172/03)1. This guideline was originally adopted by CHMP on the 23 February 2006 and came into effect on the 1st of September 2006. In recent years there have been several applications for scientific advice on new products and treatment strategies aimed at achieving functional cure, including finite and combination treatment regimens. Therefore, a revision of the guideline is necessary to reflect these new developments and the implications for clinical development programs.
IRIS Guide for Applicants
This guide has been produced to show applicants how to use the IRIS platform to prepare, submit and manage an application and/or data for a scientific procedure (orphan designation application, scientific advice, ITF briefing meeting requests, PRIME, marketing status reports, inspections and veterinary signal management) and related activities, or applications for Parallel Distribution procedures.
This revision added instructions to manage regulatory procedures in IRIS in existing section (main changes in yellow) and added a new section dedicated to Product Lifecycle Management procedures (section title in yellow).
Clinical Trials Information System (CTIS) Sponsor End User Training Programs
The following Training Programs are open to sponsor users of the new CTIS: commercial and non-commercial sponsors as well as Contract Research Organizations (CROs).
IRIS Guide to Registration and RPIs
This guide has been produced to help new users of IRIS to complete the prerequisite steps before accessing the platform. Most of these steps are independent from the IRIS platform and are similar to those to obtain registration to use other European Medicines Agency (EMA) systems, such as Management Services for Substances, Products, Organization and Referentials (SPOR).
Q & A to Stakeholders on the Implications of Regulation (EU) 2023/1182 for Centrally Authorized Medicinal Products for Human Use
This practical guidance document includes information related to the impact of Regulation (EU) 2023/1182 on medicinal products for human use authorized pursuant to Regulation (EC) No 726/2004 (centrally authorized medicinal products - CAPs). When not specifically mentioned the guidance below applies as of the date on which Regulation (EU) 2023/1182 becomes applicable (for further information please see Section 9 below). For the impact of Regulation (EU) 2023/1182 on medicinal products authorized by EU/EEA Member States (Nationally Authorized Products (NAPs), including medicinal products authorized through Mutual Recognition Procedures (MRPs) and Decentralized Procedures (DCPs)) for human use, applicants/marketing authorization holders are advised to contact the relevant national regulatory authorities.
ICH Q5A(R2) Guideline on Viral Safety Evaluation of Biotechnology Products Derived from Cell Lines of Human or Animal Origin
(effective 14 June 2024)
This guideline describes the evaluation of the viral safety of biotechnology products including viral clearance and testing, and it outlines what data should be submitted in marketing applications for those products.
Concept Paper on the Establishment of a Guideline on the Development and Manufacture of Human Medicinal Products Specifically Designed for Phage Therapy
(submit comments by 31 March 2024)
This concept paper proposes to establish a scientific guideline for the pharmaceutical development and manufacture of bacteriophage medicinal products intended for the therapeutic treatment or prophylaxis of one or more specific bacterial infection(s) or
18th Industry Stakeholder Platform - Operation of European Union (EU) Pharmacovigilance
This virtual meeting aimed to inform, get feedback and foster dialogue between industry and the EU regulators.
Topics included:
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artificial intelligence in pharmacovigilance;
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update on good pharmacovigilance practice;
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risk management plans, including EMA transparency measures and guideline on specific adverse reaction follow-up questionnaires;
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updates on PASS with the catalogues to replace the current ENCePP resources database, and the EU PAS register
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EudraVigilance update.
Risk Management Plans (RMP) in Post-Authorization Phase: Questions and Answers
The following question was revised:
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14. When and how will the full RMP be published on the EMA website? Rev. Dec 2023
All post-authorization RMP updates assessed and approved in procedures concluding on or after 20 October 2023 will trigger the publication of the full RMP (body and Annexes 4 & 6).