An agency of the European Union

Enpr-EMAEuropean Network of Paediatric Research

at the European Medicines Agency

Background information

Irmgard Eichler, MD

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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

- 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

PIP

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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

PIP

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Unless an EMA decision on a PaediatricInvestigation Plan or waiver is included, the validation of a marketing authorisationapplication – even for adults only – will berefused.

European Paediatric Regulation

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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 statementThis 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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EBMTNIHR-MCRN

GRIPPENTI

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Breakdown of networks by type and category

Network for Research in Pediatric Critical Care INN

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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.

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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Coordinating Group (20)

Co-chaired by EMA+ elected member3 year

membership

EC (DG)

Structure - Coordinating Group

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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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➣ Pharmaceutical Industry

➣ Patients, parents and patient organisations

➣ National Competent Authorities

➣ Ethics Committees

➣ Medical devices industry

➣ CRO’s

➣ Hospital pharmacists

Main Stakeholders

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Interaction with stakeholders

• Annual workshop – open to all stakeholders

• Virtual meetings

• Mail exchange

• Scientific/regulatory conferences

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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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Networks

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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Brochure

Thank you - Questions ??