1. Background information
Enpr-EMA
European Network of Paediatric Research
at the European Medicines Agency
Background information
Irmgard Eichler, MD

2. European Paediatric Regulation
Improve the health of children
• Increase high quality research into medicines for children
• Increase availability of authorised medicines for children
• Increase information on medicines
• Avoid unnecessary studies in children
• Avoid delaying authorisation for adults
Main Pillars of the Regulation:
- the paediatric committee (PDCO)
- the paediatric investigation plan (PIP)
Under this law, companies are obliged to conduct studies in children under the age of 18 in accordance with an agreed Paediatric Investigation Plan (PIP), unless a formal waiver has been agreed.
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3. Paediatric Committee (PDCO)
27 Members (plus alternates)
including 5 from Approval Committee (CHMP)
2 from Norway, Iceland
3 Patient representatives
3 HealthCare Professionals
1 Chair elected
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4. Paediatric Committee members
• Anaesthesiology, neonatology, cardiology, immunology, transplantation, respiratory, ICU, haematology, oncology, endocrinology and diabetes, adolescent medicine, infectious diseases, gastroenterology and nutrition, general paediatrics, methodology, pharmacology, pharmacovigilance, vaccines
• Formulation Working Group
• Non-clinical Working Group
• Extrapolation Working Group
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5. European Paediatric Regulation
Improve the health of children
• Increase high quality research into medicines for children
• Increase availability of authorised medicines for children
• Increase information on medicines
• Without unnecessary studies in children
• Without delaying authorisation for adults
Main Pillars of the Regulation:
- the paediatric committee (PDCO)
- the paediatric investigation plan (PIP)
Under this law, companies are obliged to conduct studies in children under the age of 18 in accordance with an agreed Paediatric Investigation Plan (PIP), unless a formal waiver has been agreed.
©EMA

6. What is a PIP (paediatric investigation plan)?
• Details of the measures and timing to demonstrate:
- Quality
- Efficacy
- Safety
• Basis for development and authorisation of a medicinal product for children
• To be agreed by the PDCO
• Binding on company  compliance check
Marketing Authorisation Criteria
Quite an important role of regulators in allergen product development
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7. PIP
What is a Waiver?
• EMA Decision waiving the need for paediatric
development of the product in a certain condition
• Can apply to some or all subsets of paediatric population
• List of ‘class’ waivers (conditions)
• Product-specific waivers because of
- Probable lack of efficacy, or safety concern
- Disease/condition occurring only in adult population
- Lack of significant therapeutic benefit
Quite an important role of regulators in allergen product development
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8. European Paediatric Regulation
Unless an EMA decision on a Paediatric
Investigation Plan or waiver is included,
the validation of a marketing authorisation
application – even for adults only – will be
refused.
These data can then be used for all allergen products belonging to the same homologous group, but not across groups
More than 100 PIP applications from 8 different companies
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9. Introduction and background
Legal basis
European Paediatric Regulation:
“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.”
The European Medicines Agency set up Enpr-EMA in accordance with the Paediatric Regulation, which aims to improve the health of children in the EU. The Agency's Management Board adopted an implementing strategy for the network on 15 January 2008.
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 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). 9
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10. Introduction and background
• Enpr-EMA is a network of research networks, investigators and centres with recognised expertise in performing clinical trials in the paediatric population
• Members perform research with children (newborns to adolescents), in multiple therapeutic areas, and ranging from pharmacokinetics to pharmacovigilance
The European Medicines Agency set up Enpr-EMA in accordance with the Paediatric Regulation, which aims to improve the health of children in the EU. The Agency's Management Board adopted an implementing strategy for the network on 15 January 2008.
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 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). 10
©EMA

11. Mission statement
Enpr-EMA will facilitate studies in order to increase availability of medicinal products authorised for use in the paediatric population.
The European Medicines Agency set up Enpr-EMA in accordance with the Paediatric Regulation, which aims to improve the health of children in the EU. The Agency's Management Board adopted an implementing strategy for the network on 15 January 2008.
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 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). 11
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12. Mission statement This 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 encourage 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.
The European Medicines Agency set up Enpr-EMA in accordance with the Paediatric Regulation, which aims to improve the health of children in the EU. The Agency's Management Board adopted an implementing strategy for the network on 15 January 2008.
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 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). 12
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13. Recognition criteria
• Networks to be recognised by quality of paediatric research
• 6 recognition criteria and quality standards for self-assessment
➣ 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
• Each criterion composed of several sub items
• Set of minimum criteria to be fulfilled
• Self-assessment to updated annually 13
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14. Breakdown of networks by type and category
EBMT
NIHR-MCRN
Category 1: Networks fulfilling all minimum criteria for membership of Enpr-EMA.
Category 2: Networks potentially fulfilling all minimum criteria – but needing to
clarify some issues before becoming a member of Enpr-EMA.
Category 3: Networks currently not yet fulfilling minimum criteria.
GRIP
PENTI
Network for Research in
Pediatric Critical Care
INN 14
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15. What Enpr-EMA does not do
• fund studies
• act as a CRO and manage studies
• decide on research priorities which remain the responsibility of
the Member States
the Commission through the Community programmes
each individual network
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16. Structure - Coordinating Group
Co-chaired by EMA
+ elected member
3 year
membership
Patient/family representative,
Ethics committee (max 4)
PDCO members (2)
Coordinating Group (20) EC
(DG)
One representative per RECOGNISED network/ group
The Coordinating Group should not exceed 20 members and will consist of - 1 representative per RECOGNISED network or group of centres. - 2 members of the Paediatric Committee -1 representative of the European Commission (namely DG Research) and a maximum of 4 additional members for additional expertise as needed, such as patients’ representatives or Ethics Committee representative.
The Coordinating Group will be co-chaired by the EMEA and a chair elected from among the members.
Networks or centres may have to group themselves to be represented once the maximum number has been reached.
CLICK
Membership will be for 3 years only.
Representatives of networks under construction or still in the recognition process may attend as observers. Other participants may be invited to discuss specific issues on an ad-hoc basis, including representatives of stakeholders.
Industry will not be represented in the Coordinating Group through membership, but is expected to be a major stakeholder in the discussions.
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17. Coordinating Group Role of the Coordinating Group:
➣ to contribute to the short and long-term strategy of the network
➣ to address operational and scientific issues for the network
➣ to agree scientific quality standards
➣ to act as a forum for communication
The operational centre of Enpr-EMA is a co-ordinating group which is responsible for the network's long- and short-term strategy. Its tasks include:
facilitating access of the pharmaceutical industry to paediatric clinical study centres and experts;
identifying new networks to include in Enpr-EMA;
developing common educational tools for children and parents, to increase their willingness to take part in clinical trials. 17
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18. Main Stakeholders ➣ Pharmaceutical Industry
➣ Patients, parents and patient organisations
➣ National Competent Authorities
➣ Ethics Committees
➣ Medical devices industry
➣ CRO’s
➣ Hospital pharmacists
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19. Interaction with stakeholders
• Annual workshop – open to all stakeholders
• Virtual meetings
• Mail exchange
• Scientific/regulatory conferences
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20. Why should you join Enpr-EMA ?
Networks
Why should you join Enpr-EMA ?
• increase visibility as potential site(s) for industry-sponsored studies (requested by PDCO)
• gain access to SMEs for collaboration
• present your centre/network - at a European level
• save resources by sharing work, avoiding duplication
• share skills and expertise with other centres/networks
• shape the future development in paediatric research
• access information on EC framework programmes
Joining the European network is not mandatory -
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21. 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 SME office at EMA
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22. Enpr-EMA information: http://www.ema.europa.eu22
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23. Scroll
down to
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25. Brochure

26. Thank you - Questions ??