Global and regional guidance

European Medicines Agency

The European Medicines Agency (EMA) is the regulatory authority of the European Union, responsible for the scientific evaluation, supervision, and safety monitoring of medicinal products intended for the European market. Two legal documents, Directive 2001/83/EC and Regulation 726/2004 define the requirements and procedures for the authorization of medicinal products in the European Union. These comprise a centralized and a decentralized procedure. The centralized procedure is managed directly by the EMA and is used for innovative products including medicines and therapies for diseases, including rare diseases.

The EMA has released a new guideline providing recommendations on how to include and, if relevant, to retain pregnant and lactating women in clinical trials. Adoption of this guideline will support robust clinical data generation to enable informed, evidence-based decisions for the use of medicinal products.

Recognizing the important knowledge and information gaps that exist, the EMA has developed an online resource on the use of medicines during pregnancy and the breastfeeding period. The website includes several initiatives with the goal of improving access to high-quality evidence for patients and their health care providers on the benefits and risks of medicinal products taken during pregnancy and the breastfeeding period.

These initiatives cover the pre-clinical phase focusing on the better use of pharmacokinetics and the development of new technologies. For the pre-authorization phase, the strategies include promoting the inclusion of pregnant and breastfeeding women in clinical trials and enhancing understanding of the benefits and risks of using medicinal products before birth. This approach secures health benefits whilst protecting patients. The initiatives also cover the post-authorization phase by striving to improve methods and infrastructure. For all phases of a medicine’s lifecycle, initiatives focus on transparent communication to all stakeholders, including women and their families, particularly when there is uncertainty about the benefit–risk ratio.

In the context of this strategy, and similar to existing paediatric investigation plans, a maternal investigation plan, funded by the European Union, is being developed by a collaboration of institutions specializing in maternal and infant health, infectious disease research, clinical trial design, and public health policy. This project is a combination of European Union initiatives for maternal health research and regulation including the EMA’s project PIPELINE – a collaboration between 16 European research institutions working to create a multicountry adaptive clinical trials platform and Horizon Europe funding – the European Union’s funding programme for research and innovation. These efforts aim to strengthen research, improve data collection, ensure the safety of pregnant and lactating participants in clinical trials, and enhance maternal and newborn healthcare access and quality for all women, including vulnerable groups.

African Medicines Agency

The African Medicines Agency (AMA) Treaty was adopted by the African Union in 2019 and established as a specialized agency of the African Union in 2021. Its stated mission is to provide leadership in creating an enabling regulatory environment for the development of the pharmaceutical sector in Africa. In the future, AMA will seek to publish guidance for different essential regulatory functions including marketing authorization, inspection, market surveillance, safety monitoring, oversight of clinical trials and quality control.

African Vaccine Regulatory Forum

In 2006, the African Vaccine Regulatory Forum (AVAREF) – a network of African national regulatory authorities and ethics committees – was established. The Forum has evolved into a network of partners and healthcare professionals who work with WHO Member States to improve the harmonization of regulatory practices on the African continent. AVAREF collaborates with several stakeholders, including members of the Trial Regulatory and Clinical Ethics Optimization (TRACE) initiative to design and implement sustainable regulatory and ethics systems.

International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) is a convening body bringing together national regulatory authorities and marketing authorization holders. Several of its guidelines include pregnant and breastfeeding populations. The ICH E21 guideline on the inclusion of pregnant and breastfeeding individuals in clinical trials draft document states that, in principle, the inclusion of pregnant and breastfeeding women in clinical trials should be considered for all products where women of childbearing potential are among the anticipated user population. Publication of this guideline is anticipated for the last quarter of 2027.