Small But Mighty: Innovative Clinical Trial Designs in Patient Populations of Limited Size - Day 1

Chair(s)

• 

  Angela J. James, PhD

  Vice President, Head of Clinical Pharmacology
  MacroGenics, Inc.
• 

  Amy S. Y Cheung, PhD

  Senior Director
  Certara
• 

  Bernard Sebastien, PhD

  Local Head of Clinical Modeling and Evidence Integration France
  Sanofi

Speaker(s)

• 

  Dominic Bram, MSc

  PhD Student
  University Children's Hospital Basel
• 

  Sebastian Haertter, PhD

  Clinical Pharmacology Expert
  Boehringer Ingelheim
• 

  Elin Haf Davies, PhD

  Aparito
• DH

  D'Anna Holmes, MS
• GK

  George Kirk, PhD

  AstraZeneca
• 

  Gilbert Koch, PhD

  UKBB
• 

  Anna Largajolli, PhD

  Certara
• 

  Jing Liu, PhD

  Senior Director, Clinical Pharmacology
  Pfizer
• 

  Marc Pfister, MD

  Co-Chair Pediatric Pharmacology & Pharmacometrics
  UKBB
• 

  Cécile Ollivier, MS

  Regulatory Science Advisor, Director NL
  Critical Path Institute
• 

  Daniel Weiss, MD

  Head of Early Clinical Development
  Schrodinger
• 

  Larissa Wenning, PhD

  Distinguished Scientist
  Merck & Co., Inc.
• 

  Sarah Zohar, PhD

  Professor of Research
  Inserm

Session Description

Effective strategies to develop therapies used to treat rare diseases, pediatric indications or a subpopulation of oncology patients, (e.g., targeted therapies) are challenging mainly due to small patient populations. Innovative thinking is needed to design clinical trials for small patient populations that is both feasible, patient-centric and able to achieve maximum statistical benefit by regulatory requirements. The "Small But Mighty" workshop is designed to foster disruptive ideas and generate novel strategies to move beyond the "3+3" paradigm and help drug developers address this “unmet need”.

Day 1 will include panel discussions with experts from diverse backgrounds who will cover topics spanning from patient-centric study designs, use of digital biomarkers in pediatric and rare disease clinical trials and the application of machine-learning (ML) and artificial neural networks (ANN) to help identify key covariates in pediatric and rare disease patient populations.

Learning Objectives

1. Utilize innovative strategies to leverage data and patient perspective to design clinical trials that incorporate novel statistical approaches and use of biomarker-based endpoints to demonstrate clinical benefit for indications with small patient populations.

2. Apply Machine Learning (ML)/Artificial neural network (ANN) to identify covariates based on specific research questions and facilitate pharmacometrics analyses in pediatric and rare disease patient populations.

Session Schedule

8:00 AM - 8:02 AM ET
Introduction
Angela J. James, PhD, Amy S. Y Cheung, PhD, and Bernard Sebastien, PhD

8:02 AM - 9:00 AM ET
Session 1: Design of Clinical Trials with Small Patient Populations - Challenges and Considerations
Bernard Sebastien, PhD and Amy S. Y Cheung, PhD

9:00 AM - 9:50 AM ET
Patient-centric Study Design Panel Discussion
Angela J. James, PhD, D'Anna Holmes, MS, George Kirk, PhD, Daniel Weiss, MD, and Elin Haf Davies, PhD

9:50 AM - 10:00 AM ET
BREAK

10:00 AM - 11:00 AM ET
Session 2: Use and Validation of Biomarkers
Sebastian Haertter, PhD, Cécile Ollivier, MS, Anna Largajolli, PhD and Larissa Wenning, PhD

11:00 AM - 11:55 AM ET
Session 3: Quantitative Methods for Trials with Small Patient Populations
Jing Liu, PhD, Sarah Zohar, PhD, Marc Pfister, MD, Dominic Bram, MS, and Gilbert Koch, PhD

11:55 AM - 12:00 PM ET
Closing
Angela J. James, PhD