Comprehensive Course in Pediatric Drug Development April 17 18, 2013 PERI Training Facility Arlington, Virginia Wednesday, April 17, 2013 Tab No. 7:30 8:00 AM Registration & Continental Breakfast 8:00 8:15 AM PERI Welcome Marian E. Selby Course Manager The Pharmaceutical Education and Research Institute, Inc. (PERI) 8:15 8:30 AM Introductions and Course Overview Ernest A. Kopecky PhD, MBA - Course Director VP, Clinical Development Head, Neuroscience TA Collegium Pharmaceutical, Inc. Meeting notes Introduction Why study children? Adult drug-to-pediatric extrapolation issues Short overview of what is to come in the course References 8:30 9:30 AM Anatomical/Physiological Differences between Children and Adults Myron Yaster, MD, PhD Richard J. Traystman Distinguished Professor Departments of Anesthesiology/Critical Care Medicine and Pediatrics The Johns Hopkins University School of Medicine 4 Age categorization by physiological development Blood volumes by age Anatomical changes eg., impact on drawing blood, heart, lungs, liver, kidney, intestines, brain Development of the CNS 9:30 10:15 AM PV: Safety Monitoring Issues Myron Yaster MD, PhD 5 10:15 10:30 AM Refreshment Break Safety monitoring requirements by age category Age-appropriate threshold vales o Vitals, clinical labs, capnography Clinical signs of unwellness Therapeutic drug monitoring what is it? When do you use it? 1
10:30 11:15 AM Blood Volume Conserving Strategies Brookie M. Best, PharmD, MAS Clinical Research Associate Professor of Clinical Pharmacy and Pediatrics University of California, San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences Pediatrics Department, School of Medicine 6 Number of blood samples/24h Blood sampling techniques eg. - Discard, re-injection, push/pull Population-based PK Analytical techniques Dry blood spot analysis 11:15 AM 12:15 PM PK Simulation as a Prerequisite to FIC 7 12:15 1:15 PM Lunch Break What is it? What is needed for it? Who can do it qualifications? Getting the dose right how is it done? Benefits and limitations Case study 1:15 1:45 PM Panel Discussion Myron Yaster, MD, PhD 1:45 2:30 PM Pediatric Regulations Office of Clinical Pharmacology, CDER US Food & Drug Administration 8 2:30 2:45 PM Refreshment Break Exclusivity v. indication requirements PREA v. the written request FDAAA2007 and pediatric drug development requirements Orphan drug development The FDA today v. 3, 5 years ago a changing Agency 2:45 3:30 PM The Written Request 9 What is a written request and what it is not Asking for one v. getting one advantages and disadvantages Timing within clinical development Agency timelines for response managing expectations actual v. published 2
Wednesday, April 17, 2013 continued 3:30 4:15 PM Designing Pediatric Protocols Ernest A. Kopecky PhD, MBA 10 Differences between adult and pediatric protocols Elements of good pediatric protocols Passing the scientific and ethics reviews 4:15 4:45 PM Panel Discussion 4:45 5:00 PM Workshop Preview 11 Provide Case for Workshop Provide pre-workshop preparation instructions 5:00 PM Day One Wrap up, Q&A Thursday, April 18, 2013 7:30 8:00 AM Continental Breakfast 8:00 9:00 AM The EU Pediatric Regulation and PIP (TBD) 12 Aims and requirements of the regulation The PIP how to write a successful PIP Expectations and evaluation of a PIP getting feedback EMA/PDCO PIP approval process and timing 9:00 9:45 AM Designing Clinical Trials Kathryn Bohannon Executive Director INC Research 13 Pediatric clinical trial designs Trial designs by physiological age Choice of comparator including the placebo control IDMC What is it? When do you need one? Who should participate? FIC clinical trials Utilizing pediatric networks 9:45 10:30 AM Clinical Operations Kathryn Bohannon 14 Site and staff considerations Clinical trial operations 3
Training Monitoring pediatric trials Operational challenges Cross-cultural pediatric trials 10:30 10:45 AM Refreshment Break 10:45 11:30 AM Patient Recruitment & Retention Plans Ernest A. Kopecky PhD, MBA 15 Teaching children and parents to understand clinical research and participation in clinical trials Recruitment and monitoring plans Retention plans 11:30 AM 12:15 PM Bioethics and Global Pediatric Research Albert Allen MD, PhD Senior Medical Fellow, Bioethics and Pediatric Capabilities Eli Lilly and Company 16 12:15 1:15 PM Lunch Break Foundational principles of bioethics Bioethical issues before, during, and after a clinical trial US v. global Special ethical considerations in pediatric drug development Planning for successful IRB interactions Implementation The placebo control US and global perspectives 1:15 2:00 PM Efficacy Assessments in Children TBD 17 Valid scales and assessments Types of assessments in children eg., pain, behavior, QoL Age-appropriate assessments How to decide on the tool? Common errors and the consequences 2:00 3:00 PM Clinical Laboratory Medicine in Children Bhushan Kapur DPhil, CChem, FRSC, FACB, FCACB Hospital for Sick Children (Tentative) 18 Reference ranges by age eg., CBC, chemistry, LFTs and what this tells us Alternative sampling for drug levels eg., hair, saliva, urine Analytical methods and considerations for clinical trials Via webcast 3:00 3:15 PM Refreshment Break 4
3:15 4:00 PM Formulations for Children Frank Diana PhD Vice President, Pharmaceutical Development Endo Pharmaceuticals Inc. 19 Different pediatric formulations advantages and disadvantages Appropriateness by physiological age Optimizing formulations for children for efficacy and safety Formulation timelines within clinical development Key formulation companies 4:00 4:30 PM Workshop Faculty & Students 20 4:30 PM Wrap-Up Day Two, Course Adjournment, Course Director PERI reserves the right to change session topics, order or faculty due to circumstances beyond its control. CONTINUING EDUCATION CREDIT Pharmaceutical Education & Research Institute, Inc. (PERI) is pleased to make continuing education credit available to you for your attendance at this program. To receive credit, you must attend the entire program and submit both the Continuing Education Application form and the program evaluation directly to a PERI on-site coordinator. A continuing education certificate or statement of credit will be distributed to you within three weeks of this program. Pharmaceutical Pharmaceutical Education & Research Institute, Inc. (PERI) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. ACPE Universal Activity Number is 0708-0000-12-019-L03-P. 1.4 Continuing education units (CEUs) are available for this program. Initial Release Date: 12/10/2012. This is a knowledge based CPE Activity. Continuing Medical Education PERI, Inc. is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. PERI, Inc. designates this educational activity for a maximum of 14.0 AMA PRA Category 1 Credits TM. Physicians should only claim credit commensurate with the extent of their participation in the activity. 2013 Pharmaceutical Education & Research Institute, Inc. (PERI) All Rights Reserved 5