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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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