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EFGCP ANNUALPAEDIATRIC CONFERENCEV IRTUAL E VENTB ETTER M EDICINES FOR C HILDREN18&19OctoberGlobal Paediatric Drug Development2022Progress made & always remaining challenges

AGENDA AT A GLANCETUESDAY 18 OCTOBER 202211:45Opening of Conference12:00Welcome and Introduction to the Conference12:10Revision of the Paediatric & Orphan Regulations12:45Implementation of the CT Regulation 536/2014 – Impact on Paediatric trials13:30Panel discussion: Global / transatlantic alignment for the development of paediatric products – what can belearnt from paediatric oncology drug development?14:30Coffee break14:45Panel session: Rare diseases – what is happening and can be expected?EU Commission/EU joint program on rare diseases15:45Breakout sessions: How to improve Patient & Parents involvement in Paediatric Research? Paediatric Lay Summaries Is it time to prioritise?17:15Fireside Chat18:15End of Day 1WEDNESDAY 19 OCTOBER 202211:45Opening of Conference12:00Welcome and Debrief from Day 112:10Panel Session: Optimising Paediatric Drug Development – what progress has been made since the 2021Paediatric Conference?13:30Panel Session: Neonatal Research – where are we and what could be next steps?14:30Panel Session: What’s new at ICH for paediatric drug development?15:30Coffee break15:50Panel Session: Can drug repurposing benefit drug development in Children & in Paediatric Rare Diseases?16:50Feedback from the Breakout Sessions17:50End of Day 2 – Conclusion & Farewell1

RATIONALEThe EFGCP “Better Medicines for Children” Conference is an annual flagship event providing a uniqueopportunity to share best practice, reflect on what has happened since we last met in October 2021, anddiscuss paediatric updates with a truly global outreach. Despite an ever changing and challengingenvironment, and the need to meet virtually again, we believe we have been able to organise a dynamic,innovative, and highly engaging conference to inform our delegates of the latest scientific and regulatorydevelopments.The conference will focus on Global Paediatric Development, its challenges and solutions, and inparticular on what has been achieved during the pandemic, and the remaining opportunities and challenges.To this effect, the conference brings together not only distinguished speakers from all around the world butalso all the stakeholders involved in paediatric drug development.Day One will bring regulators and patient representatives to discuss the potential impact on R&D of therevision of paediatric regulation and of the implementation of the Clinical Trial Regulation.Stakeholders from around the world while focusing on global alignment will share their view on what canbe learnt from paediatric oncology drug development. A panel committed to finding solutions for rarediseases, which present a unique public health challenge, will discuss the latest information on rarediseases.Active engagement of conference delegates will be then welcomed in Breakout Sessions to discusschallenges and identify solutions on what could be done to optimise children’s access to new medicines,with a focus on important topics:1. How to improve Patient and parents’ involvement in Paediatric research?2. Paediatric Lay Summaries3. Is it time to prioritise?At the end of Day 1, a fireside chat will be the opportunity to hear from Dr Koen Norga (PDCO chair), DrSabine Scherer (PDCO vice-chair) and Dr Angeliki Siapkara (MHRA and former UK representative on PDCO)about their respective activities and priorities; and also to hear their view and answers to questions raisedahead of the conference.Day two will be the opportunity to keep the momentum with experts who joined the 2021 conference. Hotpaediatric topics for which multi-stakeholders’ collaboration is more than ever valued to address gaps inscientific knowledge, will explore innovative development avenues that could foster paediatric drugdevelopment.The programme committee looks forward to welcoming you all to the conference for a global educationaland networking event that is a unique opportunity to learn from one another, to meet and interact withcolleagues from all parts of the world.We do hope that the 2022 conference will be the last one organised remotely, and 2023 will give us to goback to face-to-face meetings.NEWS! For those interested in paediatric drug development, a pre-conference Workshop onthe "Fundamentals of Paediatric Extrapolation" has been set up on October 17. This will be theopportunity to learn from the experts the value of paediatric extrapolation, a topic detailed in the ICH E11guideline recently released for public consultation. For further details on this pre-conference Workshop,please visit the webpage.Thank you for joining us!2

ACKNOWLEDGEMENTEFGCP Thanks its Partners & Members for their continued supportEFGCP Corporate MembersEFGCP Institutional Members3

PROGRAMME COMMITTEEAlexandru CostescuEuropean Commission, BelgiumAngelika JoosMSD, BelgiumAngeliki SiapkaraMHRA, United KingdomBegonya Nafria EscaleraSjd Children's Hospital Barcelona, SpainGilles VassalGustave Roussy Institute, FranceHelen ShawProveca, United KingdomKoenraad NorgaAntwerp University Hospital, Belgium & Chair of EMA PDCO, NLMartine Dehlinger-KremerEFGCP, EUCROF & ICON Plc., GermanyRhian Thomas-TurnerNHS, United KingdomSabine Fuerst-RecktenwaldRoche, SwitzerlandSolange Corriol-RohouAstraZeneca, FranceThomas SeverinNovartis, SwitzerlandFacultyAlexandru CostescuEuropean Commission, BelgiumAnn Marie Janson LangSwedish MPA, SwedenAngeliki SiapkaraMHRA, United KingdomBegonya Nafria EscaleraSjd Children's Hospital Barcelona, SpainChristina Bucci-RechtwegNovartis, USAChristina KyriakopoulouEuropean Commission, BelgiumClaudio FracassoPfizer, ItalyCorinne de VriesEMA, The NetherlandsDimitrios AthanasiouEURORDIS, GreeceDominik KarresEMA, The NetherlandsGregory ReamanFDA, USAGilles VassalGustave Roussy Institute, FranceGunter EggerEnpr-EMA, EMA, the NetherlandsHelen MaoHealth Canada, CanadaHelen ShawProveca, United KingdomHannah BatchelorInstitute of Pharmacy and Biomedical Sciences, University of Strathclyde, ScotlandIsaac Rodriguez-ChavezICON, USAJeffrey BarrettCritical-Path Initiative, USAJohn van den AnkerChildren’s National Medical Center Washington, DC , USAKaye RobertsonTGA, AustraliaKoenraad NorgaAntwerp University Hospital, Belgium & Chair of EMA PDCO, The NetherlandsKristin KarlssonSwedish MPA, SwedenKristina An HackSanofi, FranceLynne YaoFDA, USAMarie-Yvonne Douste-BlazyMYDB Paediatric Clinical Insights, FranceMark TurnerLiverpool University, United KingdomMartine Dehlinger-KremerEFGCP, EUCROF & ICON Plc., GermanyNick SireauAK, United KingdomNicola RupertoPRINTO, ItalyNicolas FreyRoche, SwitzerlandPamela DicksNHS, United KingdomRalph BaxEMA, the NetherlandsRhian Thomas-TurnerNHS, United KingdomSabine Fuerst-RecktenwaldRoche, SwitzerlandSabine SchererBfArM & Vice-Chair of EMA PDCO, GermanySam BlackmanDayOne, USASolange Corriol-RohouAstraZeneca, FranceSusan McCunePPD, USAZhimin YangNMPA, China4

DAY ONE – 18 OCTOBER 202111:45Opening of Conference12:00Welcome and Introduction to the ConferenceMartine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.12:10Revision of the Paediatric & Orphan Regulations12:45Implementation of the CT Regulation 536/2014 – Impact on Paediatric trialsAnn Marie Janson-Lang, Swedish MPA & Nicola Ruperto, PRINTO13:30Panel discussionGlobal / transatlantic alignment for the development of paediatric products – what can belearnt from paediatric oncology drug development?Moderator: Gilles Vassal, IGRPanel: Gregory Reaman, FDA Dominik Karres, EMA Helen Mao, Health Canada AngelikiSiapkara, MHRA Zhimin Yang, NMPA Kaye Robertson, TGA14:3014:45Coffee breakPanel sessionRare diseases – what is happening and can be expected?EU Commission/EU joint program on rare diseasesModerator: Angeliki Siapkara, MHRAPanel: Kristina An-Haack, Sanofi Christina Kyriakopoulou, EU Commission Dimitrios Athanasiou,EURORDIS15:45Breakout sessionsHow to improve Patient & Parents involvement in Paediatric Research?Moderators: Pamela Dicks, NHS & Rhian Thomas-Turner, NHSPaediatric Lay SummariesModerators: Begonya Nafria Escalera, SJD Hospital Nicola Ruperto, PRINTOIs it time to prioritise?Moderators: Gilles Vassal, IGR & Solange Corriol-Rohou, AstraZeneca17:15Fireside Chat withKoenraad Norga, PDCO Chair Sabine Scherer, PDCO Vice-Chair Angeliki Siapkara, MHRAModerator: Claudio Fracasso, Pfizer18:15End of Day 1Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.5

DAY TWO – 19 OCTOBER 202111:45Opening of Conference12:00Welcome and Debrief from Day 1Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.12:10Panel SessionOptimising Paediatric Drug Development – what progress has been made since the 2021Paediatric Conference?Moderators: Sabine Fuerst-Recktenwald, Roche & Gilles Vassal, IGRoooo13:30Innovative Techniques towards Drug Development: Hannah Batchelor, Instituteof Pharmacy and Biomedical Sciences, University of StrathclydeUse of RWD in paediatric drug development: Marie-Yvonne Douste-Blazy, MYDBPaediatric Clinical InsightsDecentralised Clinical Trials: Isaac Rodriguez-Chavez, ICONAdolescents in Adult Clinical Trials: Christina Bucci-Rechtweg, NovartisPanel SessionNeonatal Research – where are we and what could be next steps?Moderators: Solange Corriol-Rohou, AstraZeneca & Mark Turner, Liverpool UniversityPanel: Jeff Barrett, C-Path Initiative John van den Anker, Children’s National Medical CenterWashington Ralph Bax, EMA Susan McCune, PPD14:30Panel SessionWhat’s new at ICH for paediatric drug development?Moderators: Solange Corriol-Rohou, AstraZeneca & Sabine Fuerst-Recktenwald, Rocheooo15:3015:50ICH E11A: Lynne Yao, FDAMIDD: Kristin Karlsson, MPA & Nicolas Frey, RochePregnant & Breast feeding Individuals in CTs: Corinne de Vries, EMACoffee breakPanel SessionCan drug repurposing benefit drug development in Children & in Paediatric Rare Diseases?Moderators: Alexandru Costescu, EU Commission & Helen Shaw, ProvecaPanel: Gunter Egger, Enpr-EMA, EMA Sam Blackman, Dayone Nick Sireau, AKU16:50Feedback from the Breakout Sessionsooo17:50How to improve Patient involvement in Paediatric Research?Paediatric Lay SummariesIs it time to prioritise?End of Day 2 – Conclusion & FarewellMartine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.6

EFGCPA N N U A L PA E D I AT R I C C O N F E R E N C EBETTER MEDICINESFORCHILDRENGlobal Paediatric Drug DevelopmentProgress made & always remaining challengesConference Language: EnglishInformation & Registrations- Click Here events@efgcp.euV IRTUAL E VENT

DAY ONE - 18 OCTOBER 2021 11:45 Opening of Conference 12:00 Welcome and Introduction to the Conference Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc. 12:10 Revision of the Paediatric & Orphan Regulations 12:45 Implementation of the CT Regulation 536/2014 - Impact on Paediatric trials Ann Marie Janson-Lang, Swedish MPA & Nicola Ruperto, PRINTO