Welcome and Opening Remarks

• Lynne Georgopoulos, RN, MSHS, RAC, Vice President, Regulatory Strategy, Certara

Keynote Session: Pediatric Regulation: Challenges Big and Small

• Brian Aylward, MD, Chair European Medicines Agency Paediatric Committee

Challenges and Opportunities in Conducting Pediatric Rare Disease Trials

• Ethical Considerations in Pediatric Clinical Trials Robert Nelson, PhD, MD,  Senior Director, Pediatric Drug Development, Johnson & Johnson
• Facilitating Pediatric Research Through Data Sharing – Patients and Sponsors Arthur L. Caplan, PhD, Drs. William F and Virginia Connolly Mitty Chair Director, Division of Medical Ethics, NYU Grossman School of Medicine
  
• Expanding the Clinical Trials Toolbox to Support Rare Pediatric Disease Research – Decentralized Clinical Trials Isaac Rodriguez-Chavez, PH.D., M.H.S., M.S., Senior Vice President, Scientific & Clinical Affairs, and Head of the Center for DCTs and Digital Medicine, ICON plc
• Developing Novel Endpoints for Paediatric Clinical Trials with Digital Health Technology David Nobbs, PhD, International Regulatory Policy Lead for Digital Health and Data Policy, Roche
• Five Voices Consortium: A Novel Framework for Improving Rare and Orphan Medical Product Lifecycle Development, Approval and Use Maryna Kolochavina, PharmD, PhD, PMP, Director, Five Voices Consortium
• Panel Discussion with session speakers and panel members moderated by Jeff Barrett, Aridhia DRE
  • Panel Members: Erwin Berthier, PhD, Chief Technology Officer, Tasso, Inc. | Elly Barry, MD, MMSc, Head of Clinical Development, Day One Biopharmaceuticals

Advancing Pediatric Drug Development for Rare Diseases through modeling and simulation

• Accelerating Rare disease Cures (ARC) Program, Kerry Jo Lee, MD, Associate Director for Rare Diseases, US FDA, Center for Drug Evaluation and Research
• ICH E11A Pediatric Extrapolation Framework, Lynne Yao, MD, Director, Division of Pediatric and Maternal Health, US FDA, Center for Drug Evaluation and Research
• Impact of Project Optimus on Pediatric Oncology Drug Development, Julie Bullock, PharmD, Senior Vice President, Global Head of Clinical Pharmacology & Translational Medicine, Certara
• Moving Towards a Question-Centric Approach for Regulatory Decision-Making in the Context of Drug Assessment, Flora Musuamba, PhD, Professor, University of Namur and Belgian Federal Agency for Medicines and Health Products
  
• Panel Discussion with session speakers and panel members moderated by: Dave Mitchell, PhD, Senior Vice President, Strategic Engagement, Certara
  • Panel Members: Krithika Arun Shetty, PhD, Associate Director, Certara | Ashley Strougo, PhD, Childhood Cancer Program Lead, Sanofi | Angela James, PhD, Vice President, Head of Clinical Pharmacology, MacroGenics Inc. | James Yates, MMath, PhD, Scientific Director, GSK | Glen Clack, MB, BS, MD, FFPM, Professor of Translational Medicine, University of Manchester | Samuel Blackman, PhD, MD, Founder & Chief Medical Officer, Day One Biopharmaceuticals | Lauren Price, PharmD, Clinical Pharmacology Reviewer, US FDA, Center for Drug Evaluation and Research

Closing Remarks

• Lynne Georgopoulos, RN, MSHS, RAC, Vice President, Regulatory Strategy, Certara

Welcome and Opening Remarks

• Amy Cheung, PhD, Senior Director of Integrated Drug Development, Certara

Keynote Session: Data, Informatics, and AI for Future Pediatric Drug Development

• Neil Sebire, MB BS, BClinSci MD, FRCR, FRCOG, FRCPath, FFFCI, Professor and Chief Research Information Officer, Great Ormond Street Hospital, HDR UK Chief Clinical Data Officer

Pediatric Treatment and Prophylactic Use in Infectious Disease

• Pediatric HIV: Challenges and Opportunities Lionel Tan, MBBS, FRCP, PhD, DTM&H, ViiV Healthcare, Research and Development
• MIDD Journey to Use MBMA for RSV mAb Development in Infants and Children Brian Maas, PharmD, Principal Scientist, Merck
• Quantitative Approaches to Support Antimicrobial Treatment Development for Neonatal Sepsis Joe Standing, PhD, Professor of Pharmacometrics, Great Ormond Street Hospital for Children/University College London
• Panel Discussion with session speakers and panel members moderated by: Amy Cheung, PhD, Senior Director of Integrated Drug Development, Certara
  • Panel Members: Jian Li, PhD, Professor, Monash University, Australia | Virna Schuck, PhD, VP, Head of Clinical Pharmacology, Ribon Therapeutics | Adrie Bekker, PhD, MD, Professor, Stellenbosch University

Rare Disease and Challenge of more Vulnerable Population

• How to Make Treatments for Rare Diseases Less Rare? The EFPIA Moonshot Initiative Solange Rohou, MD, Sr. Director of Regulatory Affairs & Policy for Europe, AstraZeneca
• Strengthening Children Participation in Decision Process in the Biomedical Sector: From Principles to Good Practices Annagrazia Altavilla, PhD, HDR, Lawyer, TEDDYNETWORK Chair, Espace Ethique Paca-Corse
• Studying Safe and Effective Dosing of Drugs in Neonates and Infants: Experience of the Pediatric Trials Network Rachel G. Greenberg, MD, MHS, Duke Clinical Research Institute
• FDA Perspective: Advancing Clinical Trials in Pregnancy Leyla Sahin, US FDA, Center for Drug Evaluation and Research
• PBPK Modelling to Support Pregnancy and Neonate Clinical Trial Development Alice Ke, PhD, Senior Director, PBPK, Certara
• Panel Discussion with session speakers and panel members moderated by: Solange Rohou, MD, Sr. Director of Regulatory Affairs & Policy for Europe, AstraZeneca
  • Panel Members: Felix Boakye-Agyeman, PhD, MD, Senior Director of Pharmacometrics, Certara | Karen Rowland Yeo, PhD, Senior Vice President of Client and Regulatory Strategy, Certara | John Van Den Anker, MD, PhD, Chair of Pediatric Clinical Pharmacology at Children’s National Medical Center

Closing Remarks

• Amy Cheung, PhD, Senior Director of Integrated Drug Development, Certara