Case Studies of Clinical Trials in Rare Diseases with Innovative Designs Utilizing Historical Data

• Activity Number: 570 - Addressing the Unique Analytic Challenges of Drug Development in Rare Diseases with the Use of Innovative Designs and Real World Evidence in Clinical Trials
• Type: Topic Contributed
• Date/Time: Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
• Sponsor: ENAR
• Author(s): Bingzhi Zhang*
• Companies: Sanofi
• Keywords: historical data borrowing; rare disease; innovative design; dynamic borrowing

Abstract

Innovative study design utilizing the information borrowed from external or historical data can greatly improve the efficiency of clinical trials by increasing power, reducing sample size and shortening study duration, leading to potentially a cost-effective trial design. FDA has initiated a Complex Innovative Trial Design (CID) Pilot program in 2018 to support the use of novel clinical trial designs and there have been many recent advancements in statistical methodology in this area. Dynamic or conditional borrowing based on the similarity between the concurrent and historical placebo is preferred in order to control the type I error. And comprehensive simulation studies are often needed to assess the operating characteristics across different scenarios. In this presentation, adaptation of dynamic borrowing methods will be illustrated with implementation in clinical trial designs.