"Small and Medium-sized Enterprises (SMEs) Highlights" Newsletter Issues

Editorial (09/02/2024)

Welcome to the first edition of the SME newsletter for 2024. We are excited to bring you the latest updates and information on matters relevant to Small and Medium-sized Enterprises. Our goal is to provide valuable insights into regulatory and scientific topics, public consultations, and events that may be of interest to your business.

Major update of the SME user guide (09/02/2024)

The SME user guide provides information on the EU legislative framework for medicines, outlining requirements for the development and authorisation of medicines for human and veterinary use. The new release includes updates to reflect changes in the EU’s legal and regulatory framework for human and veterinary medicines.

Union list of critical medicines (13/02/2024)

The European Commission, the Heads of Medicines Agencies and EMA have released the Union list of critical medicines in order to ensure supply security and prevent shortages of critical medicines. It contains more than 200 active substances of medicines for human use considered critical for healthcare systems across the EU/EEA, for which continuity of supply is a priority.

PRIME/Breakthrough therapy applications (13/02/2024)

EMA’s PRIME scheme and FDA’s breakthrough therapy (BT) designation program are designed to help speed development of innovative products which address unmet medical needs. A joint EMA and Food and Drug Administration (FDA) questions-and-answers (Q&A) document has been released on how to address quality development and good manufacturing practice (GMP) challenges when developing medicines under these early access schemes. It covers topics including control strategy, process validation, stability and shelf-life and GMP considerations.

ACT EU workplan for 2023-2026 (13/02/2024)

A second HMA-EC-EMA ACT EU workplan setting out deliverables and timelines for the programme was published. It highlights key focus areas to further facilitate innovation in clinical trials, stakeholder engagement and regulatory network collaboration.

CTIS transparency rules (13/02/2024)

Revised CTIS transparency rules adopted on 5 October 2023 introduced simplifications to give access to clinical trial information to stakeholders, patients and healthcare professionals in a faster and more efficient way and remove the deferral mechanism to deliver earlier publication of key documents and protocols, as well as system simplification.

Transition of clinical trials to the Clinical Trials Regulation (13/02/2024)

All ongoing clinical trials in the EU must be transitioned to the Clinical Trials Information System (CTIS) by 30 January 2025. The deadline date will mark the end of a three-year transition period that began when the Clinical Trials Regulation (CTR) became applicable in the EU.

Other news on clinical trials (13/02/2024)

The following documents have been updated:

Quality guidelines (13/02/2024)

Quality guidelines

Clinical guidelines (13/02/2024)

Clinical guidelines

Medical Devices (13/02/2024)

Medical Devices

Artificial intelligence workplan (13/02/2024)

EMA and the Heads of Medicines Agencies (HMAs) have published a workplan on artificial intelligence to 2028, which sets out a strategy to guide the use of AI in medicines regulation and focuses on the following key areas: guidance, policy and product support, AI tools and technology, collaboration and training, and experimentation.

Invented names (13/02/2024)

Invented names

IRIS for human and veterinary regulatory procedures (13/02/2024)

IRIS

Standard for the Exchange of Nonclinical Data (SEND) proof of concept study (13/02/2024)

SEND

Veterinary medicines (13/02/2024)

New guidelines on the calculation of dose factor to be submitted to the Union Product Database and on the assessment criteria to determine the need for an MRL evaluation for chemical-unlike biological substances were published. Several draft guidelines were also released for consultation on topics such as GMP for active pharmaceutical ingredients and benefit risk evaluation.

Webinar on the HMA-EMA Catalogues of real-world data sources and studies (04/03/2024)

This event provides an overview of the HMA-EMA Catalogues of real-world data sources and studies including their benefits and significance in regulatory practices. It will also explain how to use the catalogues, user roles and responsibilities effectively.

Clinical Trials Information System (CTIS): Walk-in clinic (12/03/2024)

The walk-in clinic on CTIS functionalities provides an opportunity for sponsors to receive practical advice on the Clinical Trials Information System by asking questions to CTIS experts.

Veterinary Medicines Info Day 2024 (14/03/2024)

EMA is organising the annual Veterinary Medicines Info Day to provide updates on regulatory policy, scientific and legislative developments for veterinary medicines.

Clinical Trials Information System Webinar: Last Year of Transition (25/03/2024)

The event will provide reflection on the implementation of the Clinical Trials Regulation, transitional trials and how they are updated in CTIS and update on the current status of CTIS.

Cancer Medicines Forum workshop (05/04/2024)

EMA and the European Organisation for Research and Treatment of Cancer (EORTC) are co-chairing the Cancer Medicines Forum which aims to integrate the work of the academic sector into the regulatory decision-making process. The workshop will discuss challenges and solutions with regards to treatment optimisation taking into consideration the ICH E9(R1) guidance.

EMA multi-stakeholder workshop on psychedelics (16/04/2024)

The workshop will bring together patients, healthcare professionals, academia, regulators, and industry to discuss the development and therapeutic use of psychedelic substances to address unmet medical needs in the area of mental health.

Recent events (13/02/2024)

Recent events