Enpr-EMA priority activities
Table of contents
- Paediatric clinical trial preparedness
- Involving young people and families
- Collaboration with ethics committees
- Collaboration with paediatric research nurses
- Informed consent requirements for paediatric clinical trials
- Facilitating interactions between the pharmaceutical industry and networks
- International collaboration
- Input on public consultations
The aim of the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is to facilitate studies in order to increase the availability of medicines for children. It works by holding annual workshops to define priority tasks and setting up working groups to carry out specific activities.
These highlighted activities include priority working areas of Enpr-EMA and its working groups. For more details and regular updates on Enpr-EMA activities, see also Enpr-EMA's regular newsletters.
- Preparedness of medicines’ clinical trials in paediatrics: Recommendations by the Enpr-EMA working group on trial preparedness
‘Preparedness’ is the structured assessment of contributing factors that enable the smooth conduct of clinical trials in a timely manner.
The recommendations cover the following areas:
- Collecting relevant data and information
- Involving contributors, such as clinical-trial sites, expert groups, patients and their families, and regulators
- Following a structured approach
- Using appropriate resources
- Approaches for preparing plans and studies
Enpr-EMA published the recommendations in August 2020. The final recommendations take account of feedback from stakeholders received via a public consultation in 2019.
Involving young people and family members in clinical research has many benefits, including greater understanding of young people's perspectives and improvements in study design and the quality of clinical research.
Enpr-EMA has established young person advisory groups (YPAG) within the Enpr-EMA members. These members set up the European network of young people advisory groups (eYPAGnet), which aims to become the single point of contact for all YPAGs in Europe.
In 2017, in collaboration with Enpr-EMA, EMA has agreed on principles for involving patients and consumers below 18 years of age in the activities of its scientific committees and working parties:
This follows a 2014 Enpr-EMA survey with EMA's Paediatric Committee (PDCO) on the potential involvement of young people and families in PDCO activities and strengthening the participation of members representing patients' organisations in the Committee. For full details, see:
Enpr-EMA also surveyed its paediatric research networks on the involvement of young people and family members in 2012. The survey results indicated that, although more than half of the networks that responded actively involve young people or families, only three networks out of thirty-nine had developed strategies or guidelines for the involvement of young people and families. For these documents, see:
- National Institute for Health Research, United Kingdom: Medicines for Children Research Network consumer strategy 2012 - 2015;
- Scottish Children's Research Network, United Kingdom: Young Persons' Group strategy documentation;
- Maternal Infant Child and Young Research Network, Canada: Creating and connecting networks.
As of 2017, Enpr-EMA and the European Network of Research Ethics Committees (EUREC) are exploring ways of collaboration.
As a first step, Enpr-EMA and EUREC will collect ideas for collaboration and collect a survey on needs and expectations among their members.
The role of research nurses in coordinating clinical trials involving children has become more important in recent years, due to increasing complexities of clinical trial design.
Enpr-EMA is exploring ways to facilitate connections between paediatric research nurse networks and groups in Europe. As a first step, it has compiled a list of networks and groups via the Enpr-EMA network:
- Table of European Paediatric Research Nurse Networks and Groups provided by the Enpr-EMA Research Nurse Training Group
Enpr-EMA has also carried out a survey with research nurses to investigate the training needs of research nurses involved in paediatric trials across Europe and to compare roles across specialties and countries. The survey results were published in 2017:
Consent requirements are not harmonised in Europe and vary according to national laws and regulations. These discrepancies can present challenges for paediatric clinical trials, especially as many are conducted as multinational trials.
To provide a comprehensive multinational overview on informed consent and assent requirements of paediatric clinical trials across Europe, an Enpr-EMA working group has collated the legal requirements in EU Member States, plus Iceland and Norway:
Enpr-EMA updates this document as necessary.
For more information on the working group's findings, see:
The working group has also published recommendations regarding the contents of the various subject elements of consent / assent forms for each paediatric age group, to be considered when designing these documents:
Enpr-EMA aims to facilitate interactions between medicine developers and paediatric research networks on issues related to paediatric medicine development.
In 2018, Enpr-EMA published guidance to pharmaceutical industry stakeholders outlining the recommended model for consulting paediatric research networks, particularly on developing and conducting paediatric investigation plans.
This guidance was informed by a 2015 Enpr-EMA survey with industry representatives and paediatric clinical trial networks on how they engage with one another. Enpr-EMA published the survey results in 2016:
Enpr-EMA also facilitates interactions between SMEs and academic investigators in paediatric-medicine research, by providing them information on networking and partnership opportunities.
Enpr-EMA works with international partners specialising in the regulation of medicines for children, including with the:
- World Health Organization (WHO) through EMA membership of the Paediatric Medicines Regulators' Network (PmRN);
- United States Food and Drug Administration (FDA) through the Agency's existing interaction on paediatric therapeutics .
Enpr-EMA has set up a working group to explore further collaborative opportunities with international regulators and paediatric research networks.
Enpr-EMA contributes to important public consultations related to paediatric medicine research in Europe. It publishes some of the comments it submits to public consultations on this website.
Comments on Revision of Clinical Trials Directive 2001-20 On behalf of the European network of paediatric research at the EMA (PDF/72.76 KB)
First published: 21/03/2014
Last updated: 21/03/2014
Response to the concept paper for public consultation on the Commission guideline on the format and content of applications for paediatric investigation plans (PDF/50.37 KB)
First published: 21/03/2014
Last updated: 21/03/2014
Response to general report on experience acquired as a result of the application of the paediatric regulation of 19 September 2012 submitted for public consultation by the European Commission on ‘experience acquired’ and... (PDF/116.78 KB)
First published: 20/12/2012
Last updated: 20/12/2012