Early dialogue for paediatric development plans Review of the pilot phase
1st industry stakeholder platform on research and development support (EMA, London) Presented by Gunter Egger on 25 April 2017 Scientific officer / Paediatric medicines
An agency of the European Union
Early paediatric interaction meeting (EPIM) Based on wish by industry for early dialogue with the Paediatric Committee (PDCO) Pilot initiated in June 2015 to explore concept
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Takes place at early phase of drug development
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Focuses on overall development strategies
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To discuss potential paediatric needs and scope of development for paediatrics
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Not intended for evaluation of data to support a PIP application
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Early paediatric interaction meeting - analysis after 22 months
Early interaction – Intended benefits •
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Early contact with the PDCO •
Early planning
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To optimise resources
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Helpful to applicants and beneficial to later PIP process
To discuss approach to paediatric development and timelines •
Integration with adult development
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Avoiding unnecessary studies in children
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Possible extrapolation
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Appropriateness of waiver
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PIPs more likely to meet PDCO requirements and children’s needs Early paediatric interaction meeting - analysis after 22 months
Applications for early interaction by therapeutic area June 2015 – March 2017 (pilot phase)
total: 36
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Early paediatric interaction meeting - analysis after 22 months
Applications received/accepted for early interaction
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Total number received 2 Ready for PIP For Scientific advice (SA) 3 Early interaction (EPIM) 3 Early interaction (EPIM) EMA/FDA 1 2 3
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36 25 2 8 1
5 (14%) from SMEs (1 for SA, 4 ready for PIP) 9 have now submitted a PIP EPIM = Early Paediatric Interaction Meeting
Early paediatric interaction meeting - analysis after 22 months
What the data tell us
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Most applications submitted at legal PIP submission stage (69%)
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Specific questions asked which are usually addressed during PIP review process or SA (e.g. primary EP ok?, sample size ok?)
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Early paediatric interaction meeting - analysis after 22 months
Examples of topics discussed at early interaction meetings
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Condition/indication/scope of PIP
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Paediatric age-groups/waiver
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Approach to modelling/simulation & extrapolation
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Type of clinical studies
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Early paediatric interaction meeting - analysis after 22 months
Feedback from applicants* Very much
Somewhat
Not so much
Overall satisfaction
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Better integration of paediatrics likely
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Improved PIP procedure likely
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Scope of EPIM clear
xxxxx
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Scope of PSM clear
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Not at all
*Feedback was only sought from applicants who had taken part in an early interaction meeting (total feedback received: n=6)
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Early paediatric interaction meeting - analysis after 22 months
Feedback from applicants Pros: •
Overall experience positive and deemed useful
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Good to get early interaction, preliminary view of PDCO
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Good to discuss PIP condition
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Good to also get input from beyond PDCO (e.g. members of CHMP, PDCO-working groups, SA, EMA staff)
Cons: •
Some important decision making deferred to PIP and SA procedures
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Questionable if it enables better PIP procedures
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Communication regarding timelines should be clearer
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Early paediatric interaction meeting - analysis after 22 months
Summary observations from the pilot phase •
Very limited number of requests were in scope.
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Feedback from applicants was generally positive if interaction occurred.
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Need for early interactions remains but scope and operations need to be fine-tuned to meet expectations of all parties.
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Early paediatric interaction meeting - analysis after 22 months
Thank you for your attention
European Medicines Agency
30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact
Early dialogue for paediatric development plans - European ...
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Name and signature of the PIP contact point: Signature available on file. Date: 30 Jan 2018. Contact for inquiries from interested parties: Boehringer Ingelheim International GmbH. Telephone: +49 6132 77-8271. Email: paediatrics@boehringer-ingelheim.