European Medicines Agency’s Post

EMA JUST RELEASED ‼️ Reflection paper on establishing efficacy based on single- arm trials submitted as pivotal evidence in a marketing authorisation application 💠Introduction and scope💠 #Randomisedcontrolledtrials (#RCTs) are the standard for providing confirmatory evidence on the efficacy of an #investigationaltreatment. If the pivotal clinical data in a #marketingauthorisationapplicationdossier are intended to stem from #singlearmtrials (#SATs), it is the responsibility of the applicant to justify to regulators the reasons for deviating from the expected standard and the appropriateness of the SATs as alternative. As part of such justification, it must be substantiated that the SATs provide adequate pivotal evidence of #efficacy. The purpose of this reflection paper is to outline perspectives on SATs that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications. It aims to identify and critically reflect on specific and important features of SATs. Systematic assessment of these features supports applicants and regulators in evaluating the adequacy of SATs to provide pivotal evidence for regulatory decision-making. Such considerations strongly depend on the full clinical context and attributes of the investigational treatment. Obtaining scientific advice is therefore strongly recommended to discuss whether pivotal evidence from SATs could be considered acceptable for seeking marketing authorisation for a specific development programme. The assessment of efficacy usually covers multiple endpoints and is an essential part of the benefit-risk assessment. The assessment of a marketing authorisation application is based on the totality of evidence across the drug development programme which usually includes the conduct of multiple clinical trials. The key concepts described in this reflection paper apply to all objectives and in all contexts the SAT is used for. Many of the considerations described in this document also translate to SATs which are not submitted as pivotal evidence, including those used for decision-making in early development. The general requirements for the design, planning, conduct, analysis, and reporting of clinical trials also apply to SATs and are not the focus of this reflection paper. Sections 1.1. and 1.2. specify the type of trials discussed in this reflection paper as well as characteristics specific to SATs. Following a list of relevant regulatory guidelines (Section 2. ), key concepts and definitions useful to articulate considerations for assessment and interpretation of SATs are described in Section 3. , whereas Section 4. translates these concepts into practical considerations. #EMA If you like this post follow me on LinkedIn 👇 https://lnkd.in/dcS-DJiZ

On November 15, the European Medicines Agency (EMA) issued a reminder to marketing authorisation holders to submit type IA and type IAIN variations for 2024 no later than November 30, 2024. This ensures the EMA can acknowledge submissions within the 30-day timeframe before the Agency’s holiday closure from December 20, 2024, to January 5, 2025. Key Recommendations: 1. Submission Deadlines: - Type IA and IAIN Variations: Submit by November 30 for acknowledgment in 2024. - Type IB Variations or Grouped Variations (IB and IA): Submissions after November 30 may not start until January 2025. 2. Procedure Guidance: - For procedural or regulatory queries, use the EMA Service Desk under “Human Regulatory,” selecting specific subcategories for variations. - For fee-related queries, contact the EMA at nfr@ema.europa.eu. 3. Preparation Resources: - Marketing authorisation holders are encouraged to consult the EMA’s guidance documents to ensure smooth submission processes. - The EMA emphasizes the importance of timely submissions to avoid delays and align with the Agency’s operational schedule. For more updates on regulatory developments, follow Global Regulatory Insights. #EMA #MedicinesRegulation #PharmaceuticalIndustry #TypeIVariations #RegulatoryUpdates #GRI

As a marketing authorisation holder with SME status, we’re able to support eligible companies with the European Medicines Agency’s (EMA) impending fee increase for regulatory submissions from 1 January 2025 — passing on significant discounts to save our clients thousands on their regulatory application. Find out more about our regulatory services below to see how we can help you navigate the complex regulatory landscape and bring your products to market efficiently and cost-effectively. 👇 #CRO #RegulatorySubmissions #EMA #MarketingAuthoriationHolder #SME

Improving efficiency of approval process for new medicines in the EU According to a recent report published by EMA: - In 2023, the percentage of MAAs submitted on time was only 35%. This is in line with 2018-2022 data, which indicates that each year only 30-40% of expected MAAs are submitted on the date indicated in the letter of intent. - 42% of companies seeking marketing authorisation in 2023 requested more time to respond to questions from EMA’s scientific committees during the assessment (also known as an ‘extended clock-stop’) because their data was not mature enough when it was submitted to EMA. - In 2023, the average duration of clock-stops for initial MAAs (198 days) was comparable to the average time of assessment (204 days). In 2022, the average clock-stop was longer (205 days) than the assessment time (196 days). https://lnkd.in/d5vCicGj

🌟 Key Reminder for 2024! Ensure Type IA and Type IA IN variations are submitted by 30 November 2024 to meet EMA timelines for processing before year-end closures. Type IB submissions should also follow this timeline for evaluation to start within 2024. 🛠️ Why It Matters Whether it’s a Type I or Type II variation, these regulatory processes are vital to ensure medicines meet the highest standards of safety and efficacy while allowing flexibility for ongoing improvements. EMA’s guidance to meet deadlines ensures efficient handling and timely approvals, keeping critical medicines accessible to patients. 📢 Understanding Type I and Type II Variations: Simplifying Regulatory Changes As the European Medicines Agency (EMA) advises marketing authorisation holders to submit Type IA and Type IA IN variations for 2024 no later than 30 November 2024, this is a timely opportunity to explore the differences between Type I and Type II variations in regulatory submissions. 🔍 Type I Variations – Minor Changes with No Significant Impact Type I Variations cover changes that do not significantly impact the quality, safety, or efficacy of a medicinal product. They are designed to streamline minor adjustments while maintaining regulatory compliance. ▶️ Type IA (Post-Implementation Reporting): Changes like manufacturing site address updates or analytical method improvements. Can be reported within 12 months (Type IA) or immediately after implementation (Type IA IN). ▶️ Type IB (Pre-Evaluation): Slightly more impactful changes, such as adding a new manufacturing site using existing technologies. Requires prior evaluation, with a 30-day approval process. 🔍 Type II Variations – Major Changes Requiring Comprehensive Review These variations are more complex and significantly affect the product’s quality, safety, or efficacy, requiring a thorough evaluation by EMA. ▶️ Examples: Changes in dosage forms (e.g., tablet to capsule). Expanding therapeutic indications. Significant updates to manufacturing processes. Adding new clinical data to support efficacy or safety. ▶️ Process: A detailed submission is required, followed by a 60-day evaluation (extendable). 📩 Need More Info? Please feel free to contact me regarding these requirements or if you have any questions. Let’s work together to ensure a smooth and efficient submission process! #EMA #Type1Error #CNRResearch #CRO

Update of the Q&A Documents for "Centralised Procedures" EMA just updated below: In October 2024, the "Questions & Answers (Q&A)" documents relating to centralised marketing authorisation procedures were again updated and published on the website of the European Medicines Agency (EMA). These Q&A catalogues are intended to provide assistance and guidelines for marketing authorisation holders and applicants for centralised procedures and provide answers to possible questions at different stages of the centralised marketing authorisation application. The list of questions relating to topics before and during the application for a centralised procedure 'European Medicines Agency pre-authorisation procedural advice for users of the centralised procedure' has been supplemented or updated to include the following points: Chapter 3 Preparing the dossier:: The paragraph "3.3.9. Can I apply for Design Space or Process Analytical Technology (PAT) in my application?" has been updated and now contains four subsections. Furthermore, the paragraph "3.4.2 When do I have to submit an Environmental Risk Assessment (ERA)?" has been updated. Chapter 5 Assessment of the application: In Chapter 5, the subsection "5.2.2 When can I expect a pre-approval GCP inspection and how are they conducted?" was revised. The new version of the Q&A document 'European Medicines Agency post-authorisation procedural advice for users of the centralised procedure', which addresses issues that may be relevant after the marketing authorisation has been granted, contains some additions and revisions in the following passage: Chapter 4 Extension of marketing authorisation The following question has been updated: "4.15. When do I have to submit revised product information? In all languages?" The new versions of the 'Q&A' documents for centralised procedures can be found at: 1. Topics before and during the application 2. Topics after the authorisation has been granted

Update of the Q&A Documents for "Centralised Procedures" EMA just updated below: In October 2024, the "Questions & Answers (Q&A)" documents relating to centralised marketing authorisation procedures were again updated and published on the website of the European Medicines Agency (EMA). These Q&A catalogues are intended to provide assistance and guidelines for marketing authorisation holders and applicants for centralised procedures and provide answers to possible questions at different stages of the centralised marketing authorisation application. The list of questions relating to topics before and during the application for a centralised procedure 'European Medicines Agency pre-authorisation procedural advice for users of the centralised procedure' has been supplemented or updated to include the following points: Chapter 3 Preparing the dossier:: The paragraph "3.3.9. Can I apply for Design Space or Process Analytical Technology (PAT) in my application?" has been updated and now contains four subsections. Furthermore, the paragraph "3.4.2 When do I have to submit an Environmental Risk Assessment (ERA)?" has been updated. Chapter 5 Assessment of the application: In Chapter 5, the subsection "5.2.2 When can I expect a pre-approval GCP inspection and how are they conducted?" was revised. The new version of the Q&A document 'European Medicines Agency post-authorisation procedural advice for users of the centralised procedure', which addresses issues that may be relevant after the marketing authorisation has been granted, contains some additions and revisions in the following passage: Chapter 4 Extension of marketing authorisation The following question has been updated: "4.15. When do I have to submit revised product information? In all languages?" The new versions of the 'Q&A' documents for centralised procedures can be found at: 1. Topics before and during the application 2. Topics after the authorisation has been granted

News from regulation – The December #CHMP meeting highlights are online. The Paul-Ehrlich-Institut reports on new recommendations for marketing authorisation, extensions of therapeutic indication, newly published EPARs and recently started marketing authorisation procedures. 👉 https://lnkd.in/e3_8MQhv The Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) is responsible for the scientific assessment of the application dossier on the quality, safety and efficacy, including environmental safety, of a new medicinal product for human use as part of the centralised marketing authorisation procedure. The Paul-Ehrlich-Institut is represented in the CHMP as a co-opted member.

🔬 Unlock the Secrets of the EU Marketing Authorisation Application (MAA) The MAA is the sum of years of drug development, culminating in a dossier submitted to the European Medicines Agency (EMA). Preparing and submitting an MAA is a significant undertaking, involving a multidisciplinary team preparing the Common Technical Document (CTD) dossier over 18–24 months. For medicine developers planning EMA or MHRA marketing applications within two years, we detail key considerations in a webinar collaboration with Essjay Solutions. We discuss: 1️⃣ What comes under the mandatory scope of the centralised procedure, 2️⃣ Regulatory deliverables to complete in the lead-up to filing an MAA, 3️⃣ The timelines for the #MAA evaluation, 4️⃣ Everything that goes into the region-specific Module 1 of the MAA dossier, 5️⃣ How the rest of the dossier fits into Module 1, and 6️⃣ #Pharmacovigilance requirements, particularly the risk management plan. Watch the full webinar here 🔗 https://lnkd.in/eguvaysy

For sponsors of #clinicaltrials in the #EU. 🇪🇺 The European Medicines Agency #EMA has finalized its reflection paper on establishing efficacy based on single-arm trials (SATs) submitted as pivotal evidence for marketing authorization. Following the public consultation of the draft version in April 2023, the final document incorporates extensive revisions in response to stakeholder feedback, including 45 comments from various contributors. EMA reaffirms that RCTs are the regulatory gold standard for confirmatory evidence of efficacy. SATs will only be considered as an alternative when a robust justification is provided. Applicants must now clearly demonstrate why SATs are appropriate and provide pivotal evidence that supports efficacy. The updated paper excludes guidance on the use of external controls, removing a dedicated section and clarifying that designs including non-randomized external control arms are not considered SATs, though certain principles from the paper may still apply. Other revisions include more targeted implementation of ICH E9(R1), with enhanced clarity on key definitions and terminology. Specifically, the updated document emphasizes that regulatory assessment of efficacy requires comparative information, with SATs merely observing outcomes under the investigational treatment. Do you need support with your clinical trial design and benchmarking your #clinialdata? Contact us for hands-on guidance. ☞ meetus@taoexcellence.ch #EMA #ClinicalResearch #RegulatoryAffairs #PharmaceuticalDevelopment #Efficacy #ClinicalTrials #MedicalRegulation #publichealth #innovation #medtech #medicaldevices #MDR #IVDR #regulatorycompliance #clinicalevaluation