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Innovative Designs and Statistical Approaches for Small Trials: Rare Disease or Pediatric Indications
Session Chair(s)
Susan Wang, PhD
Global Head of Biostatistics and Data Sciences Inflammation
Boehringer Ingelheim, United States
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 Objective : 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.
Speaker(s)
Adaptive Cloud Based eClinical Design Vital to Embrace Value-Based Outcomes in Rare Disease
MaryAnne Rizk, PhD
Rizk Management Advisors, United States
Chief Executive Officer
Innovative Statistical Methods for Measuring Benefit to Patients with Rare Diseases
Munish Mehra, PhD, MS, MSc
Quantum Biopharma, United States
Managing Director and Sr. Principal Biostatistician
Leveraging Available Information in Pediatric Trial Designs
Susan Wang, PhD
Boehringer Ingelheim, United States
Global Head of Biostatistics and Data Sciences Inflammation
Statistical Challenges in Assessing Treatment Efficacy from Small-Sized Clinical Trials
Yeh-Fong Chen, PhD
United States
Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER
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