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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

Global Head of Biostatistics and Data Sciences Inflammation, Boehringer Ingelheim Pharmaceuticals Inc., United States
Dr. Susan Wang is the global head of biostatistics and Datas Sciences in inflammatory diseases at Boehringer-Ingelheim Pharmaceuticals, Inc., in Ridgefield, Connecticut. She has many years of experiences working on global drug developments and registrations as a lead statistician... Read More →

avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, FDA
Dr. Chen is the Team Leader of the Division of Biometrics IX within CDER of FDA, supervising reviewers for the Division of Non-Malignant Hematology Products. She joined FDA in 2000 after receiving her Ph.D. from the University of Iowa. She has had years' experience in reviewing drug... Read More →
avatar for Munish Mehra

Munish Mehra

Managing Director, Tigermed India, Tigermed, United States
Munish Mehra serves as Executive Director and Principal Biostatistician at Tigermed US and as Managing Director at Tigermed, India Pvt. Ltd. During a career spanning 33 years, he gained extensive experience in the design, analysis and reporting of phase I-IV clinical trials across... 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 CDT
S502ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  07: SpecialPops, Session