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Enpr-EMA

European Network of Paediatric Research


at the European Medicines Agency

Background information

An agency of the European Union

Irmgard Eichler, MD
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)

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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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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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What is a PIP (paediatric investigation plan)?

• Details of the measures and timing to demonstrate:


- Quality
Marketing
- Efficacy Authorisation
- Safety Criteria

• Basis for development and authorisation of a


medicinal product for children
• To be agreed by the PDCO
• Binding on company  compliance check

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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
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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.

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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.”

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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

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Mission statement

Enpr-EMA will facilitate studies in order to


increase availability of medicinal products
authorised for use in the paediatric population.

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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.
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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

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Breakdown of networks by type and category

NIHR-MCRN EBMT

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.

Network for
Research in
PENTI Pediatric Critical
Care INN GRIP

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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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Structure - Coordinating Group
Co-chaired by EMA
3 year + elected member
membership

Coordinating EC
(DG)
Group (20)

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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

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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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Interaction with stakeholders

• Annual workshop – open to all stakeholders

• Virtual meetings

• Mail exchange

• Scientific/regulatory conferences

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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

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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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Enpr-EMA information: http://www.ema.europa.eu

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