Transcript Background information - European Medicines Agency

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
• Details
of the measures and timing to demonstrate:
- Quality
- Efficacy
- Safety
•
(paediatric investigation plan)?
Marketing
Authorisation
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.
PENTI
Network for
Research in
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
3 year
membership
Co-chaired by EMA
+ elected member
Coordinating
Group (20)
EC
(DG)
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Coordinating Group
Role of the Coordinating Group:
➣ to
➣ to
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contribute to the short and long-term strategy of the network
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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