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.
Printable version of this agenda: Challenges and Strategies to Facilitate Development of Pediatric Drug Products
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 |
| 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 Mulberg Presentation Notes 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 Ternik Presentation Notes 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 Aylward Presentation Notes Brian Aylward, MD Clinical Assessor Health Products Regulatory Authority Irish Medicines Board |
| 8:50-9:00 a.m. | FDA Clinical Perspectives Radden Presentation Notes 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. | EuPFl and Pediatric Formulation Development Tuleu Presentation Notes Catherine Tuleu, PhD Reader in Pharmaceutics University College London School of Pharmacy |
| 9:10-9:20 a.m. | Pediatric Formulations Research: NIH Perspectives Zajicek Presentation Notes 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 Kaukonen Presentation Notes Ann Marie Kaukonen, PhD Senior Researcher/Pharmaceutical Assessor Finnish Medicines Agency |
| 10:55-11:20 a.m. | FDA Product Performance/Chemistry Perspective Selen Presentation Notes 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 Liu Presentation Notes Fang Liu, PhD, MSc, BSc Senior Lecturer in Pharmaceutics and Drug Delivery University of Hertfordshire |
| 11:40-12:00 p.m. | Industry Perspective Tan Presentation Notes 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 Khong Presentation Notes 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 Bartlett Presentation Notes 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 Sachs Presentation Notes 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 Klein Presentation Notes 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 Davit Presentation Notes 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 Fegley Presentation Notes Darren Fegley, PhD Pharmacologist/Toxicologist Food and Drug Administration Buckley Presentation Notes Lorrene Buckley, PhD, DABT Toxicologist Eli Lilly and Company Carleer Presentation Notes 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 Turner Presentation Notes 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 Presentation Notes Wen Lin, PhD Senior Investigator Novartis |
| 1:15-1:30 p.m. | Biopharmaceutical Considerations in Pediatric Formulation Development Cook Presentation Notes Jack Cook, PhD Clinical Pharmacology Leader Pfizer, Inc. |
| 1:30-1:45 p.m. | Proposed BCS for Pediatrics and Implication on Bioequivalence Assessment Polli Presentation Notes 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 Seo Presentation Notes 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 |