Both an overview and a detailed agenda for Challenges and Strategies to Facilitate Formulation Development of Pediatric Drug Products are provided below for the convenience of workshop participants.
This two-day workshop, scheduled for June 8-9, 2016, will feature six sessions. Breakout discussions of smaller groups will also be used to promote participant discussion. The conference will be held at the College Park Marriott Hotel & Conference Center, located at 3501 University Blvd., East in Hyattsville, MD.
For a printable version of this agenda, please download this document.
Overview Agenda:
Day 1: June 8, 2016
| Time | Activity |
|---|---|
| 8:00-8:05 a.m. | Welcome and Workshop Goals |
| 8:05-8:20 a.m. | Plenary Opening |
| 8:20-10:30 a.m. | Session 1: Age Appropriate Formulation: General Considerations Session Chairs: Anne Zajicek (NIH), Hari Sachs (FDA), Rob Ju (AbbVie), and Brian Aylward (Irish Medicines Board) |
| 10:30-11:25 a.m. | Acceptability Assessment Opening |
| 11:25-3:00 p.m. | Session 2: Acceptability Assessment of Pediatric Formulations -- Swallowability Session Chairs: Arzu Selen (FDA), Fang Liu (University of Hertforshire), and Siri Wang (Norwegian Medicines Agency) |
| 3:00-5:15 p.m. | Session 3: Acceptability Assessment of Pediatric Formulations -- Palatability Session Chairs: Julia Pinto (FDA), Bob Ternik (Eli Lilly and Company), and Siri Wang (Norwegian Medicines Agency) |
| 5:15-5:30 p.m. | Wrap-Up for Day 1 |
Day 2: June 9, 2016
| Time | Activity |
|---|---|
| 8:00-12:00 p.m. | Session 4: Food Effects in Pediatric Medicines Development for Products Co-administered with Food Session Chairs: Andrew Mulberg (FDA), Hannah Batchelor (University of Birmingham), and Ann Marie Kaukonen (Finnish Medicines Agency) Session 5: Safety Qualification of Excipients Session Chairs: Darren Fegley (FDA), Lorrene Buckley (Eli Lilly and Company), Jacqueline Carleer (Belgian Federal Agency for Medicines), and Gerri R. Baer (FDA) |
| 1:00-4:00 p.m. | Session 6: Biopharmaceutics and Clinical Pharmacology Session Chairs: James Polli (University of Maryland School of Pharmacy), Jian Wang (FDA), Brian Aylward (Irish Medicines Board), and Tycho Heimback (Novartis) |
| 4:00-4:30 a.m. | Wrap-Up for Day 2 |
Detailed Agenda:
Day 1: June 8, 2016
| Time | Activity |
|---|---|
| 8:00-8:05 a.m. | Welcome and Workshop Goals |
| 8:05-8:20 a.m. | Pediatric Global Regulatory Overview: Status, Challenges, and Opportunities with Focus on Pediatric Formulation Development Lynne Yao, MD Director, Division of Pediatric and Maternal Health Office of Drug Evaluation IV Food and Drug Administration |
| Sessions 1-3: Age Appropriate Formulation and Acceptability Assessment Regulations in both the EU and US require that pediatric drug products be appropriate for use in the target population. There has been significant discussion on demonstrating acceptability of dosage forms for pediatric patients. Formulations can be optimized based on pharmacokinetics, taste and overall acceptability measures. Given the high heterogeneity of the global pediatric population and the wide variety of existing and emerging formulation options, attributes of product acceptability are complex. The session purpose is to discuss patient acceptability and clinical performance from a global perspective, and will focus on palatability and swallowability of oral dose forms. These attributes will serve as examples around which a rational risk based approach for determining acceptability can be proposed, including: sources of data to demonstrate acceptability of palatability and swallowability; example methodology for use in assessing palatability and swallowability; and approaches to establishing criteria for acceptability. |
|
| Session 1: Age Appropriate Formulation -- General Considerations Session Chairs: Anne Zajicek (NIH), Hari Sachs (FDA), Rob Ju (Abbvie), and Brian Aylward (Irish Medicines Board) |
|
| 8:20-8:30 a.m. | Gastrointestinal Physiology in Pediatrics: Implications for Pediatric Formulation Development Andrew Mulberg, MD, FAAP Deputy Director Division of Gastroenterology and Inborn Errors Products Food and Drug Administration |
| 8:30-8:40 a.m. | Pediatric Formulation Development: Opportunities from an Industry Perspective Robert Ternik, PhD Senior Research Advisor and Design Team Leader Eli Lilly and Company |
| 8:40-8:50 a.m. | EMA/PDCO Pediatric Formulation Working Group Experience Brian Aylward, MD Clinical Assessor Health Products Regulatory Authority Irish Medicines Board |
| 8:50-9:00 a.m. | FDA Clinical Perspectives Erica Radden, MD Pediatric Team Member Division of Pediatric and Maternal Health Office of Drug Evaluation IV Food and Drug Administration |
| 9:00-9:10 a.m. | EuPFI and Pediatric Formulation Development Catherine Tuleu, PhD Reader in Pharmaceutics University College London School of Pharmacy |
| 9:10-9:20 a.m. | Pediatric Formulations Research: NIH Perspectives Anne Zajicek, MD, PharmD Chief, Obstetric and Pediatric Pharmacology and Therapeutics Branch Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
| 9:20-10:05 a.m. | Panel Discussion Panelists: Drs. Yao, Mulberg, Ternik, Aylward, Radden, Tuleu, Zajicek, Ju, and Sachs |
| 10:05-10:30 a.m. | Break |
| Acceptability Assessment of Paediatric Formulations: Opening for Sessions 2 and 3 | |
| 10:30-10:55 a.m. | EMA Regulatory Perspectives Ann Marie Kaukonen, PhD Senior Researcher/Pharmaceutical Assessor Finnish Medicines Agency |
| 10:55-11:20 a.m. | FDA Product Performance/Chemistry Perspective Arzu Selen, PhD Associate Director, Scientific Development Office of Testing and Research/Office of Pharmaceutical Quality Center for Drug Evaluation and Research Food and Drug Administration |
| 11:20-11:25 a.m. | Sessions 2 and 3: Breakout Logistics and Goals |
| Session 2: Acceptability Assessment of Pediatric Formulations -- Swallowability Session Chairs: Arzu Selen (FDA), Fang Liu (U of Hertfordshire), and Siri Wang (Norwegian Medicines Agency) |
|
| 11:25-11:40 a.m. | Literature Review Fang Liu, PhD, MSc, BSc Senior Lecturer in Pharmaceutics and Drug Delivery University of Hertfordshire |
| 11:40-12:00 p.m. | Industry Perspective David Tan Cheng Thiam Associate Scientist AbbVie |
| 12:00-1:00 p.m. | Lunch |
| 1:00-2:20 p.m. | Breakout Discussions Case studies of swallowability questions will be provided. Participants will be broken up into smaller groups to discuss potential solutions. |
| 2:20-2:35 p.m. | Break |
| 2:35-3:00 p.m. | Summary Reports from Breakout Discussions |
| Session 3: Acceptability Assessment of Pediatric Formulations -- Palatability Session Chairs: Bob Ternik (Lilly) and Siri Wang (Norwegian Medicines Agency) |
|
| 3:00-3:15 p.m. | Literature Review Yuet Mei Khong, PhD Senior Scientist/Group Leader AbbVie |
| 3:15-3:30 p.m. | Pediatric Formulation Development: Industry Perspective on Palatability Challenges and Opportunities Jeremy Bartlett, PhD Associate Research Fellow Pharmaceutical Sciences Drug Product Design Pfizer, Inc. |
| 3:30-4:50 p.m. | Breakout Discussions Case studies of palatability questions will be provided. Participants will be broken up into smaller groups to discuss potential solutions. |
| 4:50-5:15 p.m. | Summary Reports from Breakout Discussions |
| 5:15-5:30 p.m. | Wrap Up for Day 1 |
Day 2: June 9, 2016
| Time | Activity |
|---|---|
| NOTE: Sessions 4 and 5 will be held in parallel. Session 4: Food Effects in Pediatric Medicines Development for Products Co-administered with Food Session Chairs: Andrew Mulberg (FDA), Hannah Batchelor (University of Birmingham), and Ann Marie Kaukonen (Finnish Medicines Agency) The objectives of this session include: identify best practices /process flow (based on current information) that could be used to de-risk the formulation development strategy; and plan appropriate clinical studies and subsequent label claims for pediatric products that are co-administered with food. Gaps where more research or data is required will also be identified. |
|
| 8:00-8:05 a.m. | Introduction Session Chairs: Hari Sachs (FDA), Hannah Batchelor (University of Birmingham), and Ann Marie Kaukonen (Finnish Medicines Agency) |
| 8:05-8:25 a.m. | In Vitro Tools to Risk Assess the Likelihood of a Food/Vehicle Effect in Pediatric Populations Sandra Klein, PhD Professor of Pharmaceutical Technology University of Greifswald |
| 8:25-8:50 a.m. | Preclinical In Vivo, Clinical PK and PKPD Tools to Risk Assess Food/Vehicle Effects Barbara Davit, PhD Executive Director Merck |
| 8:50-11:30 a.m. | Breakout Discussions Case studies of food/vehicle effect questions will be provided. Participants will be broken up into smaller groups to discuss potential solutions. |
| 10:00-10:15 a.m. | Break |
| 11:30-12:00 p.m. | Summary Reports from Breakout Discussions |
| Session 5: Safety Qualification of Excipients used in Pediatric Formulations Session Chairs: Darren Fegley (FDA), Lorrene Buckley (Eli Lilly and Company), Jacqueline Carleer (Belgian Federal Agency for Medicines), and Gerri R. Baer (FDA) The objective of this session is to identify how current regulatory guidance is interpreted by various stakeholders and to characterize approaches to qualify pediatric excipients. Experiences from both the nonclinical and clinical perspectives will be highlighted for possible paths forward towards more consistent, risk-based approaches. |
|
| 8:00-8:15 a.m. | Overview of the Issues Darren Fegley, PhD Pharmacologist/Toxicologist Food and Drug Administration Lorrene Buckley, PhD, DABT Toxicologist Eli Lilly and Company Jacqueline Carleer, DVM Non-clinical Assessor Belgian Federal Agency for Medicines Gerri R. Baer, MD Medical Officer Office of Pediatric Therapeutics Food and Drug Administration |
| 8:15-8:35 a.m. | Current Clinical Perspective on Risk Assessment Mark Turner, PhD Senior Lecturer in Neonatology University of Liverpool |
| 8:35-9:15 a.m. | Panel Discussion Facilitators: Lorrene Buckley (Lilly) and Mark Turner (University of Liverpool) Panalists: Darren Fegley (FDA), Brian Aylward (Irish Medicines Agency), Gerri Baer (FDA), Karen Thompson (Merck), Smita Salunke (UCL School of Pharmacy), and Jacqueline Carleer (Belgian Federal Agency for Medicines) |
| 9:15-11:30 a.m. | Breakout Discussions Case studies of safety qualification of excipients questions will be provided. Participants will be broken up into smaller groups to discuss potential solutions. |
| 10:00-10:15 a.m. | Break |
| 11:30-12:00 p.m. | Summary Reports from Breakout Discussions |
| 12:00-1:00 p.m. | Lunch |
| Session 6: Biopharmaceutics and Clinical Pharmacology Considerations Session Chairs: Jian Wang (FDA), James Polli (University of Maryland School of Pharmacy), and Brian Aylward (Irish Medicines Board) The objectives of this session include: discuss considerations from a perspective of clinical pharmacology and biopharmaceutics; and identify the role of physiologically-based pharmacokinetic (PBPK) modeling and a pediatric BCS in drug development, class boundaries, and necessary research for a pediatric BCS. Topics include age range, BCS class boundaries, and frameworks for predicting drug absorption in paediatric patients. |
|
| 1:00-1:15 p.m. | Formulation-dependent Pediatric Physiologically-Based Pharmacokinetic (PPBPK) Modeling to Aid Drug Development Wen Lin, PhD Senior Investigator Novartis |
| 1:15-1:30 p.m. | Biopharmaceutical Considerations in Pediatric Formulation Development Jack Cook, PhD Clinical Pharmacology Leader Pfizer, Inc. |
| 1:30-1:45 p.m. | Proposed BCS for Pediatrics and Implication on Bioequivalence Assessment James Polli, PhD Shangraw/Noxell Endowed Chair in Industrial Pharmacy and Pharmaceutics Department of Pharmaceutical Sciences University of Maryland School of Pharmacy |
| 1:45-2:00 p.m. | Clinical Pharmacology Considerations of Pediatric Formulation: Case Studies on Antiviral and Anti-infective Products Shirley Seo, PhD Clinical Pharmacology Team Leader, Antiviral Products Food and Drug Administration |
| 2:00-3:00 p.m. | Breakout Discussions Case studies of biopharmaceutics and clinical pharmacology questions will be provided. Participants will be broken up into smaller groups to discuss potential solutions. |
| 3:00-3:10 p.m. | Break |
| 3:10-4:00 p.m. | Panel Discussion of Breakout Discussions Panelists: Drs. Seo, Fang, Polli, Heimbach, Kim, Abernethy, Suarez, Cook, Green, Liu, Aylward, and Burckart |
| 4:00-4:30 p.m. | Wrap Up for Workshop |