Pediatric Drug Development: Challenges and Opportunities in Extrapolation
WEDNESDAY, MARCH 21, 2018 | 10:00 AM – 5:00 PM

Half-Day Symposium and Mock-team Workshop 
Co-Sponsors: IQ Consortium, Pediatric Working Group, Clinical Pharmacology Leadership Group; and ASCPT Special Populations Community 
A recent paradigm change in pediatric drug development has centered on earlier and more innovative approaches to clinical research in children, with the goal of generating data that can support regulatory approval and/or provide useful information for practitioners in labeling, thereby minimizing the potential risk associated with “off-label” use. This pre-conference session will explore these issues through the lenses of both oncology and Type 2 Diabetes for the pediatric patient.

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Pharmacometrics Meets Health Economics: Quantitative Approaches in the Translation from Efficacy to Real World Effectiveness and to Cost-Effective Patient Care
WEDNESDAY, MARCH 21, 2018 | 1:00 PM – 5:00 PM

Half Day Pre-Conference with Multiple Engaging Lectures and Opportunities for Discussion
Co-Sponsors: ASCPT Quantitative Pharmacology and Development and Regulatory & Outcomes Networks; and International Society for Pharmacoeconomics and Outcomes Research. 

Translation from efficacy in randomized controlled trials (RCT) to real world effectiveness and to cost-effective patient care is an emerging interest and the ultimate goal for clinical pharmacology and therapeutics. Featuring prominent leaders in the fields of clinical pharmacology, outcome research, and pharmacoeconomics, this pre-conference will promote inter-disciplinary collaboration and present state-of-the-art science and application of quantitative approaches at these junctures.

Please click to access the schedule for the day.

Pediatric Drug Development: Challenges and Opportunities in Extrapolation Pre-conference
Oncology Symposium (2 hours)
Chairs: Konstantina M. Vanevski, MD and Dionna Green, MD 
Gregory H. Reaman, MD
Associate Director, Office of Hematology and Oncology Products 
Office of New Drugs/CDER/U.S. Food and Drug Administration 

Hubert Caron, MD, PhD
Senior Medical Director Ped. Oncology at Roche and Global Development Team Leader
Pediatrics (iPODD) Genentech, a member of the Roche Group 
Research Professor at AMC, Amsterdam

The pediatric oncology symposium will focus on the limited scope of pediatric extrapolation, including challenges and opportunities for this specific therapeutic area. Two didactic sessions will:
  1. Discuss challenges and opportunities in pediatric oncology drug development and provide an overview of the attributes of on-going and planned trials and strategies for enhancing pediatric drug development
A follow up panel discussion will be dedicated to considerations for a pediatric master protocol. Within this context, panelists may discuss trial design and molecular prioritization criteria; the role of a multi-stakeholder decision-making body and governance; logistical and operational considerations and challenges; and fulfilling regional pediatric regulations and addressing globalization challenges.

Type 2 Diabetes Extrapolation Workshop (3 hours)
The Extrapolation in Type 2 Diabetes Workshop is designed to mimic the multidisciplinary project team discussion environment that every pharmaceutical industry needs to undergo in order to propose an extrapolation plan. Hence, participants of this mock-project team will involve members from the academia and industry with the various expertise required for this exercise. The expertise identified to be of relevance is: clinical pharmacology, medical, statistics and translational science. A facilitator will be included to ensure that the discussions are focused to achieve the objectives of this proposal. Finally, regulators will be available on the call to provide input and participate as needed.

The extrapolation exercise will be for a hypothetical drug indicated for the treatment of diabetes type 2. The exact mechanism of action is still to be decided. The level of detail on this hypothetical drug will be adjusted to allow discussion of difference scenarios. The workshop will allow participants to gain insight on evidence synthesis and team member contributions and will provide an opportunity for participants to observe the pediatric planning process from inception to submission.  

Chairs: Ashley Strougo, PhD and Lily (Yeruk) Mulugeta, PharmD PhD
Mock-Team Participants:
Facilitator Lawrence Lesko, PhD, FCP 
Clinical Professor and Director of the Center for Pharmacometrics and Systems Pharmacology in the University of Florida, College of Pharmacy at Lake Nona in Orlando, FL 
Clinical Pharmacology Jeffrey Barrett, PhD
Vice President and Global Head of the Interdisciplinary Program in Pharmacometrics   and Global Head of Pediatric Clinical Pharmacology at Sanofi Pharmaceuticals
Physician Jan Marquard, MD
Medical Expert Pediatrics & Endocrinology
Team Member Medicine (Medical project lead TA metabolism / pediatric programs in diabetes) at Boehringer-Ingelheim Pharma, Ingelheim, Germany
Statistician Margaret Gamalo-Siebers, PhD
Principal Research Scientist, Eli Lily and Company, member of the Pediatric & Small Population Drug Development Team, DIA Bayesian Working Group
Pediatrics-Endocrinology Phil Zeitler, MD, PhD 
Professor and Section Head, Endocrinology 
Medical Director, Children's Hospital Colorado Clinical & Translational Research Center
(Virtual Attendance)
Lynne Yao, MD
Director, Division of Pediatrics, Office of New Drugs
Chair, Pediatric Review Committee (PeRC)
U.S. Food and Drug Administration (FDA)

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Pharmacometrics Meets Health Economics: Quantitative Approaches in the Translation from Efficacy to Real World Effectiveness and to Cost-Effective Patient Care
Chairs: Jing Liu, PhD and Richard Willke, PhD
Topics and Speakers:
Opening Remarks  Jing Liu, PhD
Background/Introduction: Role of Pharmacometrics and Health Economics for Cost-effective Patient Care  
Richard Willke, PhD
Chief Scientific Officer, ISPOR, USA
Understanding the comparative efficacy and effectiveness via meta-analysis:  Health economics approach
Jeroen P. Jansen, PhD, MSc
Chief Scientist, Precision Health Economics, USA
Understanding the comparative efficacy and effectiveness via model-based meta-analysis:  Pharmacometrics approach Jaap Mandema, PhD
President, Certara Strategic Consulting
Linking pharmacometrics and health economics: Quantitative approaches to cost-effectiveness evaluations of health care decisions
Dyfrig Hughes , PhD
Professor, Centre for Health Economics and Medicines Evaluation, University of Bangor, UK
Leveraging novel simulation techniques to incorporate pharmacometrics in pharmacoeconomic models
Jaime Caro,  MDCM, FRCPC, FACP
Chief Scientist, Evidera, USA
Industry Perspectives: Examples on how to assess values in drug development 
Myoung Kim, PhD
Vice President, Health Economics & Outcomes Research, Janssen Pharmaceuticals, Inc.
HTA Perspectives: Can linked pharmacometric-health economic evidence improve early evaluation of new medicines?
Speaker TBA
Panel discussion and closing remarks Leader: Scott Marshall, PhD
Pfizer, UK 
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