Recent Updates
EMA Pre- / Post-Authorization Procedural Advice for Users of the Centralized Procedure
13 November 2023 – the EMA updated guidance on pre-/post-authorization for users of the centralized procedure.
Updates:
3.3.6. How should I submit an Active Substance Master File (ASMF)?
Human Medicines Division - Active Substance Master File (ASMF)
1.4. How shall I present and submit my Type IA/ IAIN Variation(s)?
5.4. What procedure number will be given to grouped variation applications?
6.3. What pre-submission steps will apply to a work sharing procedure?
Biosimilars in the EU: Information Guide for Healthcare Professionals
13 November 2023 – the EMA published an Information Guide prepared jointly by the European Medicines Agency and the European Commission on the use and implementation of biosimilars for healthcare professionals.
This guide has been prepared with the important objective of providing healthcare professionals with reference information on both the science and regulation underpinning the use of biosimilars.
Questions and Answers on Data Requirements when Transitioning to Low Global Warming Potential (LGWP) Propellants in Oral Pressurized Metered Dose Inhalers
8 November 2023 – the EMA published updated Q&A guidance on data requirements when transitioning to LGWP in oral pressurized metered dose inhalers.
This questions and answers document aims at providing advice regarding data requirements for such replacements. It applies to all medicinal products irrespective of the legal basis for marketing authorization.
CTIS Training Information
7 November 2023 – the EMA published updated guidance on a variety of CTIS trainings as well as an upcoming webinar and updated Clinical Study Reports Quick Guide.
FAQs--How to Create, Submit and Withdraw a CTA
FAQs--How to Create, Submit and Withdraw a CTA
Clinical Trials Information System (CTIS) Bitesize Talk
Quick Guide—Clinical Study Reports
Concept Paper on the Revision of the Non-Clinical and Clinical Module of the Influenza Vaccines Guideline
31 October 2023 – the EMA published the draft concept paper on the revision of the non-clinical and clinical module of the influenza vaccines guideline.
Overall, there is some degree of urgency to revise the guideline on non-clinical and clinical development of influenza vaccines. In particular, to add sections relevant to the development of mRNA-based influenza vaccines and to reflect on how lessons learned from the COVID-19 pandemic could be relevant to the development of influenza vaccines, including those intended only for pandemic usage.
Reflection Paper on Ethical and GCP Aspects of Clinical Trials of Medicinal Products for Human Use Conducted Outside of the EU/EEA
26 October 2023 – the EMA published the reflection paper on ethical and GCP aspects of clinical trials conducted outside of the EU/EEA and submitted in marketing authorization applications to the EU regulatory authorities.
The scope of this Reflection Paper is to clarify the practical application of requirements for clinical trials conducted outside of the EU/EEA and submitted in Marketing Authorization Applications to the EMA (through the centralised procedure) or to National competent Authorities (through decentralised, mutual recognition, or national procedures). The Paper sets up practical steps to be undertaken during the provision of guidance and advice in the medicine development phase and during the evaluation of Marketing Authorization Applications.
Draft Proposal for an Addendum, on Transparency, to the “Functional Specifications for the EU Portal and EU Database to be audited - EMA/42176/2014”
26 October 2023 – the EMA published the draft proposal for an addendum on transparency in the EU clinical trial portal and database.
This consultation document sets out proposals and options on the application of the exceptions in relation to the transparency provisions of the European Clinical Trial Regulation.
Section 6 and the part of Table 2 Section 4.3 will be revised following the consultation and added to the “Functional specifications for the EU portal and EU database” as an addendum.
Procedural Advice for Post-Orphan Medicinal Product Designation Activities
20 October 2023 – the EMA published Rev. 14 of the guidance on post-orphan medicinal product designation activities for sponsors.
This guideline covers detailed information and procedures applicable to orphan designated products:
1. Incentives
2. Annual reports
3. Transfer of orphan designation
4. Change of sponsor’s name or address
5. Amendment of designated condition
6. Marketing authorisation application
7. Removal of orphan designation
8. Review of the period of market exclusivity of orphan medicinal products.
What EMA Publishes and When: Guide to Information on Human Medicines Evaluated by EMA
13 November 2023 – the EMA published the guide to information on human medicines evaluated by EMA.
This guide describes the different types of information the Agency currently publishes for both centrally and non-centrally authorized medicines, as well as publication times and location on EMA’s website. It aims to help stakeholders know what kind of information to expect on medicines undergoing evaluations and other regulatory procedures.
Clinical Trials Information System (CTIS) — Sponsor Handbook
10 November 2023 – the EMA published v3.03 of the CTIS Sponsor Handbook.
This Handbook addresses key questions on CTIS and provides a compilation and references to key guidance, technical information, recommendations, training materials, and supportive documentation to facilitate the submission and assessment of CTAs and additional information during the lifecycle of a trial.
Guidance on Good Manufacturing Practice and Good Distribution Practice: Questions and Answers
9 November 2023 – the EMA published updated Q&A on GMP and GDP. Guidance
The guidance provided by the working group in the form of questions and answers (Q&As) provides additional interpretation of the European Union (EU) GMP guidelines and GDP guidelines published by the European Commission.
First Electronic Product Information (ePI) Published for Selected Human Medicines
8 November 2023 – the EMA announced the first ePI published for selected human medicines.
The Heads of Medicines Agencies (HMA), the European Commission (EC) and EMA have published for the first time, electronic product information (ePI) for selected human medicines harmonized across the European Union (EU).
The product information of a medicine includes its summary of product characteristics, labelling and package leaflet.
Guideline on the Clinical Requirements for Non-Replacement Therapy in Hemophilia A and B
31 October 2023 – the EMA published the draft scientific guideline on clinical requirements for non-replacement therapy in hemophilia A and B.
This guideline describes the main clinical data needed to support an application for a marketing authorization for non-replacement therapy for use in prevention of bleeding in patients with hemophilia A and/or hemophilia B.
Draft Revised Consolidated 3-year Work Plan for the Methodology Working Party (MWP)
31 October 2023 – the EMA published the draft revised consolidated 3-year work plan for the MWP.
The Methodology Working Party aims to leverage the cross-disciplinary expertise to support methodological innovation in global drug development and support advice and interpretation of complex methodology across (clinical) drug development.
Appendix on Disclosure Rules, to the “Functional Specifications for the EU Portal and EU Database to be Audited - EMA/42176/2014”
26 October 2023 – the EMA published the appendix on disclosure rules in the EU clinical trial portal and database.
The key instrument to ensure transparency of clinical trials is the EU clinical trial portal and database that will be used for submission and maintenance of clinical trial applications and authorizations within the EU. This Appendix sets out rules and criteria for the application of the exceptions in relation to the disclosure provisions of the European Clinical Trial Regulation.
Public Consultation on Implementation of Transparency Requirements of the European Clinical Trial Regulation
26 October 2023 – the EMA published the updated Q&A guidance on public consultation on implementation of transparency requirements of the European clinical trial regulation.
The document now open for consultation describes how it is proposed to implement the transparency requirements of the European Clinical Trial Regulation, and when and which information on clinical trials conducted in the EU should be made public.
Procedural Advice for Orphan Medicinal Product Designation
20 October 2023 – the EMA published Rev. 13 of the guidance on procedural advice for orphan medicinal product designation for sponsors
In examining an application for orphan medicinal product designation, the COMP will focus on determining whether the sponsor has established that the designation criteria are met. All orphan applications should be created and submitted via the IRIS portal, which also contains the relevant procedural advice.
Position Statement on DNA and Host Cell Protein Impurities, Routine Testing Versus Validation Studies
19 October 2023 – the EMA published updated scientific guideline on DNA and host cell protein impurities, routine testing versus validation studies.
The “validation approach” appears to be an acceptable way, in most cases, to approach the question of residual host cell DNA.
This position is not as readily applicable for host cell proteins (HCP) impurities where a “case by case” review is proposed.
It is noteworthy that in any case, processes and procedures should undergo periodic critical re- evaluation to ensure that they remain capable of achieving the intended results.