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
The Use of Historical Controls From Register Data in Randomized Clinical Trials in Rare Diseases
Director of Consultancy
CROS DE Gmbh, Germany