We are updating our Privacy Policy and Terms and Conditions to help you clearly understand how your personal information is collected, stored and managed. Learn More
Menu Back toPediatric-and-Rare-Disease-Drug-Development

DIA 2018 Global Annual Meeting

The DIA 2018 Global Annual Meeting brings together thousands of innovators from around the globe.

Pediatric and Rare Disease Drug Development

    Session Chair(s)
      Munish  Mehra, PhD

      Munish Mehra, PhD

      • Executive Director
      • Tigermed Co., Ltd., United States
    This session will highlight some of the new drugs approved in the last few years for rare disease and the statistical approaches used. Regulatory, industry and patient perspectives will be shared and approaches proposed that are realistic based upon needs of patients and scientific rigor necessary. Additionally, for drugs approved where there were significant differences of opinion, regulatory, industry, and patient perspectives will be shared on experience of these marketed products post-approval and whether post-marketing studies supported the treatment to be efficacious and safe.
    Learning Objective : recognize the assumptions of historical controlled trials; design a trial
      Thomas  Zwingers

      The Use of Historical Controls From Register Data in Randomized Clinical Trials in Rare Diseases

      Thomas Zwingers

      • Director of Consultancy
      • CROS DE Gmbh, Germany
      Yeh-Fong  Chen, PhD

      Concerns Related to Pediatric Trials

      Yeh-Fong Chen, PhD

      • Mathematical Statistician, Office of Translational Sciences, CDER
      • FDA, United States