Small and Medium-sized Enterprises (SMEs) Highlights

We would like to highlight the upcoming SME Info Day that will take place on 18 October 2024. This is the first face-to-face SME Info Day in Amsterdam. It will be an opportunity to learn about current topics relevant to SMEs and interact with EMA staff to better understand how we work and how we can support your activities. The Info Day will cover scientific advice, clinical trials, medical devices, the new HTA regulation, the new EMA fee regulation and medicines’ shortages. We hope to see many of you at the EMA in Amsterdam.

Please also take some time to complete the EMA SME survey. Your feedback is important. It will help us to understand the challenges faced by SMEs to help tailor future activities to your needs. 

We invite you to explore the newsletter's contents and hope you will find it informative and useful for your activities.

Thank you.

On behalf of the EMA’s SME Office team

Constantinos Ziogas,

Head of SME Office

A working arrangements document is available to clarify the requirements and terminology of the new regulation, to set fee reductions for certain types of services and to provide further details on payment modalities. Further guidance will be published by the end of 2024.

We invite you to consult this webpage regularly. We encourage you to follow presentations on this topic at the SME Info Day on 18 October and the webinars for veterinary industry stakeholders and human industry stakeholders on 24 October 2024.

While the pilot programme is currently scheduled to run until the end of 2025, the aim is to establish a long-term process for orphan device support.  Medical device manufacturers can submit their proposals here.

For more information on how the EMA supports medical device manufacturers, please visit the EMA website. Please also refer to the information session on the orphan medical devices pilot held on 23 September 2024.

The EMA is setting up the European Shortages Monitoring Platform (ESMP) to collect information on the supply and demand of medicines to prevent, detect, and manage human medicines’ shortages in the European Union and the European Economic Area (EU/EEA). The EMA plans to deliver the first functionalities of the platform available to Marketing Authorisation Holders (MAHs) by the fourth quarter of 2024. 

New guidance and training materials are available to support MAHs:

• ESMP Essentials: industry and network reporting requirements
• FAQs on the ESMP
• Implementation Guide for Marketing Authorisation Holders (MAHs)
• Data submission templates for MAHs (prototype)
• ESMP stakeholder engagement plan

We invite you to view the webinar on ESPM and industry reporting requirements held in June 2024 and to regularly consult the ESMP webpage on EMA’s website for updates, including upcoming events and training.

Pack sizes should be submitted to XEVMPD to support the implementation of standardised product information in the EU/EEA, which also supports the readiness for reporting in the European Shortages Monitoring Platform (ESMP). A list of products mapped in XEVMPD/PMS from the first version of the Union list of critical medicines is now available to help MAHs prioritise the mapping of their pack sizes in XEVMPD. More information can be found in the dedicated webinar held recently.

The EMA encourages marketing authorisation holders (MAHs) in the EU/EEA to develop a shortage prevention plan to help reduce the likelihood of shortages of their marketed medicines. The plan should identify potential risks in the supply chain and set out the measures to manage these risks. MAHs should also develop shortage mitigation plans when there is a potential or actual shortage of their marketed medicines. 

Templates are now available for MAHs to draft their shortage prevention and mitigation plans (SPMPs). 

The implementation of SPMPs will take place through a pilot project and further details will be available soon. The development of SPMPs is one of the recommendations included in the Good Practices for industry on the prevention of human medicinal products' shortages.

• A guidance document on outcomes for joint clinical assessment, which will help Member States authorities to define the scope of the assessments and the assessors to report on the elements needed by Member States for the national assessment of clinical added value.
• A guidance document on reporting requirements, which describes how assessors should deal in practice with multiplicity issues and complementary analyses in joint clinical assessment reports.

The HTA Regulation will be adopted in a phased approach. From January 2025, all cancer medicines and/or advanced therapy medicinal products (ATMPs) will be assessed jointly, followed by orphan medicines from January 2028 and all other centrally authorised medicines from 2030.

A document has been published to clarify the scientific specifications of ‘new active substance’ and ‘therapeutic indication of treatment of cancer’ to determine whether a medicinal product will be subject to Joint Clinical Assessment between 12 January 2025 and 13 January 2030.

New features have been introduced on the CTIS public portal to make it easier to search for publicly available information on clinical trials.  Users can now perform an advanced search, download their search results and information on specific trials and subscribe to a search via an RSS feed. A number of interface changes have also been made, to improve the overall user experience. More details can be found on this webpage. 

Two consolidated advice pilots have been launched by ACT EU to provide:

• Scientific advice to support the submission of Marketing Authorisation Applications and Clinical Trial Applications (CTAs) in the EU.
• Technical and regulatory advice on CTA dossiers prior to submission to CTIS.

Further information and guidance for applicants is available in the news announcement and on the ACT EU website.

The ‘CTIS Sponsor Handbook’ and ‘Guide to CTIS Training Material Catalogue’ have recently been updated with several modules of the CTIS Online Training Programme (Modules 05, 10, 17 and 23). The updated material reflects the changes brought by the revised CTIS transparency rules and the implementation of new features in the system.  

All CTIS training material is available on the Clinical Trials Information System: Training and Support and  Clinical Trials Information System (CTIS): Online Training Modules pages.

The next CTIS Walk-In Clinic will take place on 20 November 2024.

Two new pages have been launched on the ACT EU website:

• An interactive map of support initiatives in each Member State for non-commercial sponsors conducting clinical trials. The initiatives have been compiled with input from the Clinical Trials Coordination Group (CTCG).
• A list of stakeholders within the European Medicines Regulatory Network that are involved in the clinical trial lifecycle. Detailed information on each group or forum is published, together with a visual representation of their interactions. 

Implementing Regulation (EU) 2024/2174 laying down the format of the labels for certain products and equipment containing fluorinated greenhouse gases will apply from 1 January 2025. It applies to medicines with a Meter-dosed inhalers (MDI) and propellant gases for respiratory use. A Q&A developed by the Quality Review of Documents (QRD) group on labelling requirements for centrally authorised MDIs containing fluorinated greenhouse gases was also published.

The Guideline on Good Pharmacovigilance Practices (GVP) Module XVI (Addendum II, Annex I), which provides guidance on risk minimisation measures has been revised to include clarifications on terminologies, new terminologies, categorisations of risk minimisation measures and methods for evaluating the effectiveness of risk minimisation measures. The EMA held an event to explain the changes brought to the revised guideline.

Marketing authorisation holders are required to manually add the email addresses of the Qualified Person for Pharmacovigilance (QPPV) for their products to the Union Product Database by 31 October 2024. This information will be used to send advice notes, listings of chargeable units line, and other supporting communications resulting from the forthcoming Regulation (EU) 2024/568 on fees and charges payable to the European Medicines Agency.

The EU Implementation Guide on veterinary product data in the UPD has been updated accordingly. To request assistance marketing authorisation holders are advised to contact the relevant national competent authority (a list of Member States’ UPD contact points is available here). To contact the EMA, please use the EMA Service Desk (ServiceNow).

An ICH reflection paper on opportunities for harmonisation in the use of real-world data to generate real-world Evidence (RWE), with a focus on the effectiveness of medicines, was adopted by the CHMP in July 2024. 

An ICH guideline E11A providing recommendations for harmonised approaches to the use of paediatric extrapolation to support the development and authorisation of medicines for children will come into force on 25 January 2025.

A revised guidance document on the use of medicines for the treatment and prophylaxis in case of exposure to biological agents used as weapons of terrorism, crime or warfare was adopted by the EMA’s Emergency Task Force in July 2024. The revised version uses the disease categorisation developed by the US Centers for Disease Control and Prevention (CDC). 

Following a public consultation, the Methodology Working Party (MWP) has finalised a Reflection Paper, which discusses key issues and requirements for single-arm clinical trials that are submitted as pivotal evidence in support of marketing authorisation applications for medicines in the EU.

At the request of the European Commission, the EMA published of a list of substances which are essential for the treatment of equine species, or which provide an additional clinical benefit compared to other treatment options available for equine species and for which the withdrawal period is six months. 

Guidelines defining the acceptable data requirements on safety and residues for marketing authorisation applications for non-immunological veterinary medicines and for immunological veterinary medicines intended for limited markets under Article 23 of the Veterinary Medicines Regulation (Regulation (EU) 2019/6) have been published.

The following documents have been released for consultation:

• An addendum to the guideline on clinical evaluation of vaccines, which provides guidance on clinical studies to be conducted in immunocompromised individuals before or after initial marketing authorisation in order to support recommendations for use in the product information; by 31 October 2024
• A draft guideline on data requirements to demonstrate pre-clinical and clinical efficacy of products for intramammary use in cattle; by 31 October 2024
• A draft guideline on the demonstration of efficacy for veterinary medicinal products containing antimicrobial substances; by 31 October 2024
• A draft VICH GL23 guideline on genotoxicity testing of residues veterinary medicines in human food; by 30 November 2024
• A Q&A outlining quality requirements for co-processed excipients (CoPE) used in solid oral dosage forms of human and veterinary medicinal products; by 31 December 2024
• A draft guideline specifying the type of information required for the development, manufacture and control of synthetic oligonucleotides used in human and veterinary medicinal products in order to obtain a marketing authorisation; by 31 January 2025
• A draft guideline on the type of information required for the manufacture and control of active substances used in a human medicinal product; by 31 January 2025
• A revised guideline on the clinical development of bipolar disorder in adults and adolescents, including targeting specific specifiers or symptom domains; by 31 March 2025

• Joint EC/HMA/EMA multi-stakeholder workshop on pharmacogenomics
• Joint HMA/EMA multi-stakeholder workshop on submission predictability
• Info session on web-based electronic Application Form (eAF) add package
• Joint HMA/EMA Big Data Steering Group workshop on real-world evidence (RWE) methods 
• EMA workshop on the challenges in drug development, regulation and clinical practice in hemoglobinopathies
• Cancer Medicines Forum: May 2024
• Clinical Trials Regulation (CTR) Collaborate Stakeholder Meeting, supported by ACT EU