Enpr-EMA priority activities
HumanPaediatrics
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.
Paediatric clinical trial preparedness
Recommendations are available from Enpr-EMA on clinical trial preparedness, for sponsors, principal investigators and triallists involved in paediatric clinical trials:
‘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. The recommendations of the working group were published in Archives of Disease in Childhood in 2021: Recommendations by Enpr-EMA on preparedness of clinical trials.
Involving young people and families
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:
- Presentation - Results from the questionnaire to PDCO members
- Questionnaire for the PDCO members on involvement of children, adolescents, their parents, carers or legal representatives and members representing patients' organisations in the activities of the PDCO
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.
Collaboration with ethics committees
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.
Collaboration with paediatric research nurses
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:
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:
Informed consent requirements for paediatric clinical trials
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:
For more information on the working group's findings, see:
Facilitating interactions between the pharmaceutical industry and networks
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 Enpr-EMA Working group on public-private partnership: Network consultation recommendation 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.
International collaboration
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 Pediatric Cluster Terms of Reference.
Enpr-EMA has set up a working group to explore further collaborative opportunities with international regulators and paediatric research networks.
Input on public consultations
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.