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Tuesday, June 26 • 8:00am - 9:15am
#215: Pediatric and Rare Disease Drug Development

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Component Type: Session
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-568-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

Identify approaches to design and analyze data for studies for developing new medical products for rare diseases; Describe how use of historical controls can help show therapeutic benefit in rare diseases.


Munish Mehra, PhD


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

Challenges and Strategies: Cases Studies for Pediatric Rare Disease Clinical Trials
Yeh-Fong Chen, PhD

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 →

Paolo Morelli

Paolo is General Manager of CROS NT and associate professor of statistics at Bologna University. He is the representative for the BIAS at the European Federation of Statisticians in the Pharmaceutical Industry.

Tuesday June 26, 2018 8:00am - 9:15am EDT
Room 256 Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  11: Statistics, Session