The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is a network of research networks, investigators and centres with recognised expertise in performing.
What are general and long term goals of the project?
Enpr-EMA aims at facilitating studies in order to increase the availability of medicinal products authorised for use in the paediatric population. Enpr-EMA provides a neutral platform for dialogue and collaboration between all stakeholders involved in the development of medicines for children: investigators and networks, regulators, industry and parents/patients representatives and young people.
Please state a few more specific objectives of the project.
The goal will be achieved by:
- Fostering high quality ethical research on the safety and effectiveness of medicines for children.
- Efficient inter-network and stakeholder collaboration in order to build up necessary competences at EU level and to avoid unnecessary duplication of studies.
- Informing parents, carers, children and young people about clinical trials and encouraging their participation.
- Raising awareness among health care professionals of the need for clinical trials in all ages of children and supporting their involvement in such studies.
- Assisting and entering into discussion with ethics committees on issues relevant to research and clinical trials in children.
What Enpr-EMA can offer to industry:
- Pool of patients for inclusion
- Speeding up recruitment
- Expert advice
- treatment options (standard of care)
- paediatric needs
- feasibility of paediatric clinical trials
- Access to academic partners through collaboration with the EMA Small and Medium-sized Enterprise (SME) office
What centres/networks can gain:
- Publicise centre/network – at a European and global level through Enpr-EMA’s international collaborations
- Be visible as potential site(s) for industry-sponsored studies (requested by the paediatric committee (PDCO) under the EU Paediatric Regulation within paediatric investigation plans)
- Save resources by sharing work
- Share skills and expertise of other networks
- Shape/influence future development paediatric research
- Access information and procedure of application for EC framework programmes
- Access to SME’s through established collaboration with SME office at EMA
- Collaboration / dialogue with PDCO
Describe the methodology, approach and technologies used.
Enpr-EMA is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA aims to foster high-quality ethical research on quality, safety and efficacy of medicines to be used in children, through networking and stakeholder collaboration with members from within and outside the European Union (EU). Enpr-EMA works through meetings and working groups. The working groups involve networks, industry and regulators to disseminate existing good practice rather than developing new solutions or comprehensive guidance.
To ensure certain quality standards, Enpr-EMA introduced a set of minimum recognition criteria which have to be fulfilled in order to become so called category 1 member of Enpr-EMA and to be eligible as coordinating group member. The recognition criteria were developed by the networks themselves by consensus formation techniques (Arch Dis Child 2012;97:185-188 ) and focus on:
- Research experience and ability
- Efficiency requirements
- Scientific competencies and capacity to provide expert advice
- Quality management
- Training and educational capacity to build competences
- Involvement of patients, parents or their organisations
Networks have to update their self-assessment every 2 years and are published within the Enpr-EMA fully searchable database (enprema.ema.europa.eu/enprema/), which provides easy access to data about each individual Enpr-EMA network. The information includes sources of expertise and research expertise across Europe and outside of the EU.
A coordinating group, co-chaired by one network member (elected by networks) and one EMA staff member, is tasked
- to contribute to the short and long-term strategy of Enpr-EMA
- to address operational and scientific issues
- to act as a forum for communication
The coordinating group consits of 18 network members and 2 PDCO representatives.
How is the project progressing, any results you wish to highlight?
Enpr-EMA is becoming a pan-European voice for promoting research into medicines for children:
- Set up of several working groups (WG):
- How to establish communication between Enpr-EMA, networks and
- Sharing good practices within Enpr-EMA and with industry
- Dialogue and interaction with Ethics Committees
- Neonatology (with PDCO)
- Strategies for funding and maintaining a paediatric clinical trial network
- Joint ENCePP (European Network of Centres for Pharmacoepidemiology and Pharmacovigilance) and Enpr-EMA WG on Pharmacovigilance
- How to establish communication between Enpr-EMA, networks and
- Collaboration with the Paediatric Committee (PDCO) at the EMA:
- Standard Paediatric Investigation Plans (PIPs) for rhabdomyosarcoma and Acute Myeloid Leukaemia
- Participation at EMA-expert meetings (Cystic Fibrosis; Type 2 Diabetes Mellitus)
- Contribution to PDCO advice to the European Commission (EC) on communication of arrangements available for conducting paediatric research
- Collaboration with the EMA Small and Medium-sized Enterprise (SME) office
- Support for the development of new specialty networks: Gastroenterology, Cardiology, Diabetes/ Endocrinology
- Involvement of children – young person’s groups:
- Survey among Enpr-EMA members on young people and family involvement
- Publication of available strategies for young people advisory groups
- Training/tips on strategies for the involvement of children and young people for Enpr-EMA members
- Enpr-EMA responded to public consultation of
- 5 year review on EU Paediatric Regulation
- revised Clinical Trial Regulation
- EC Guideline on the format and content of PIP applications
- Involvement in international activities
- Global Alliance for Pediatric Therapeutics Assent Project
- Global Pediatric Clinical Trials Network
Enpr-EMA is cited as one of the successes of the EU Paediatric Regulation in the EC 5-year progress report on the Paediatric Regulation .
Funding source and funding duration:
Enpr-EMA was established by the European Medicines Agency according to requirement of the European Paediatric Regulation, Art 44: “The EMA shall, with the scientific support of the Paediatric Committee, develop a European network of existing national and European networks, investigators and centres with specific expertise in the performance of studies in the paediatric population.” Enpr-EMA does not receive any funding. Enpr-EMA was officially launched in 2011 and will continue to run; no end foreseen.