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Wednesday, June 27 • 8:00am - 9:15am
#316: Opportunities for Efficient and Innovative Study Designs

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-635-L04-P; CME 1.25; IACET 1.25; RN 1.25

Drug development has been facing both opportunities and challenges. On one hand, a variety of discovery technologies have helped drive growth in the drug development pipeline. On the other hand, the biopharmaceutical industry has had to manage a challenging operating environment characterized by increasing costs, inefficient and lengthy cycle times, and high levels of risk, uncertainty, and complexity. More efficient and innovative study designs have the great potential of improving success rates and optimizing clinical trial performance and data quality.
This session will review and discuss opportunities for efficient and innovative study designs with some real world examples in the areas of effective and efficient phase 2 study design, leveraging natural history data for rare diseases drug development, and use of innovative Bayesian designs for non-inferiority studies.

Learning Objectives

Evaluate case studies in efficient and innovative study designs; Discuss challenges and opportunities in innovative designs and analyses.


Amy Xia, PhD


Understanding the Special Importance of Phase 2 Clinical Studies
Ron Marks, PhD

Leveraging Natural History Data for Rare Diseases Drug Development: A Bayesian Perspective
Shu Han, PhD, MBA

A Bayesian Approach in the Non-Inferiority Setting
Cristiana Mayer, PhD


Shu Han

Head of Biostatistics, Moderna Therapeutics
Shu Han is a statistician by training and has over 13 years of drug development experience in multiple therapeutic areas including rare diseases, oncology, immunology, and neuroscience. Shu received PhD of Statistics from the joint program of M. D. Anderson Cancer Center and Rice... Read More →

Ron Marks

Chief Scientific Officer, Clinipace Worldwide
The first 30 years of my career was in academia, serving as Professor of Biostatistics at the Univ of Florida, and also as Director for 18 years. I was a founding member of Clinipace (CPWW), bringing an early version of a web-based clinical trial system to market. I started the CPWW... Read More →
avatar for Cristiana Mayer

Cristiana Mayer

Director, Statistics and Decision Sciences, Janssen Research & Development, LLC
Cristiana Mayer is Director at Statistics and Decision Sciences, at Janssen R&D, Johnson & Johnson, with 20+ years of experience in the pharmaceutical industry. Cristiana is also the Head of the Adaptive Clinical Trials Center of Excellence at Janssen, and Vice-Chair of the DIA Adaptive... Read More →
avatar for Amy Xia

Amy Xia

Vice President, Biostatistics, Design & Innovation, Amgen Inc.
Amy Xia is Vice President, Biostatistics, Design & Innovation at Amgen. Amy has worked on designing, implementing, and analyzing Phase I-IV clinical trials as well as observational studies in the past 22+ years. Currently, she heads up the Biostatistics and Design & Innovation organizations... Read More →

Wednesday June 27, 2018 8:00am - 9:15am EDT
Room 256 Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  11: Statistics, Session