返回 Agenda
Pediatric and Rare Disease Drug Development
Session Chair(s)
Munish Mehra, PHD, MS, MSC
Managing Director and Sr. Principal Biostatistician
Quantum Biopharma, 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 : 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.
Speaker(s)
The Use of Historical Controls From Register Data in Randomized Clinical Trials in Rare Diseases
Paolo Morelli
CROS NT, Italy
CEO
Challenges and Strategies: Cases Studies for Pediatric Rare Disease Clinical Trials
Yeh-Fong Chen, PHD
Food and Drug Administration, United States
Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER