Wednesday, June 21 • 2:00pm - 3:15pm
#350: Innovative Designs and Statistical Approaches for Small Trials: Rare Disease or Pediatric Indications

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-547-L04-P; CME 1.25; IACET 1.25; RN 1.25

With the advance of the computer technology, developing personalized medicine is not an unachievable dream in the 21st century, although it remains a challenging task to efficiently conduct clinical trials with a limited number of patients. It is important to develop new methods by utilizing all available information and resources without lowering the current standard for assessing drug safety and efficacy.

Learning Objectives

Discuss how implementation of innovative designs and newly developed statistical approaches for small clinical trials such as rare disease or pediatric populations will aid the drug development process and draw all relevant stakeholders’ attention and interests.


Susan Wang, PhD


Adaptive Cloud Based eClinical Design Vital to Embrace Value-Based Outcomes in Rare Disease
MaryAnne Rizk

Innovative Statistical Methods for Measuring Benefit to Patients with Rare Diseases
Munish Mehra, PhD

Leveraging Available Information in Pediatric Trial Designs
Susan Wang, PhD

Statistical Challenges in Assessing Treatment Efficacy from Small-Sized Clinical Trials
Yeh-Fong Chen, PhD

avatar for Susan Wang

Susan Wang

Director, Biostatistics, Boehringer Ingelheim Pharmaceuticals Inc.
Dr. Susan Wang is a Director in Biostatistics at Boehringer-Ingelheim Pharmaceuticals, Inc., in Ridgefield, Connecticut. She has many years of experiences working on global drug registrations as the lead statistician. She received her Ph.D. in statistics from State University of New... Read More →

avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician, Office of Translational Sciences, CDER, FDA
Dr. Chen is the Acting Associate Director of the Division of Biometrics II within CDER of FDA, supervising reviewers for the Division of Metabolism and Endocrinology Products and Division of Anesthesia, Analgesia, and Addiction Products. She joined FDA in 2000 after receiving her... Read More →
avatar for Munish Mehra

Munish Mehra

Executive Director, Tigermed
Dr. Munish Mehra, is a drug development expert with Tigermed, providing services to Pharmaceutical and Biotechnology companies to design, analyze and report on Phase 2 and 3 studies. He has represented several sponsors at pre-IND, End of Phase 2 and pre-NDA meetings. He is a frequent... Read More →
avatar for MaryAnne Rizk

MaryAnne Rizk

VP, Global CRO Partner Business, Oracle Health Sciences
Dr. MaryAnne Rizk is a data-driven lifescience alliance strategist that optimizes clinical outsourcing partnerships across global drug/device firms and their CROs to accelerate time-to-value with innovative technologies. Dr. Rizk received her Bachelor of Engineering and Master in... Read More →

Wednesday June 21, 2017 2:00pm - 3:15pm
S502ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616