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Activity Number: 154 - Integrating Real World Data with Clinical Trials: Opportunities and Challenges
Type: Topic Contributed
Date/Time: Tuesday, August 4, 2020 : 10:00 AM to 11:50 AM
Sponsor: Caucus for Women in Statistics
Abstract #312304
Title: Evaluating the Use of Real-World Data as a Source for Full or Hybrid Control Arms for Clinical Trials
Author(s): Douglas Faries* and Zhanglin Cui
Companies: Lilly Research Laboratories and Eli Lilly and Company
Keywords: real world data; control arm; single arm trial; randomized controlled trial
Abstract:

Real-world data has been a source for creating control arms to evaluate results from single arm trials (SAT) and randomized controlled trials (RCT) with underpowered control arms in rare diseases and scenarios where randomization to a control group is unethical/unfeasible. In rare cases real-world data as a control arm for a SAT has been used to support regulatory decisions regarding efficacy. However, reliable decision making based on this methodology depends heavily on the validity of the control arm data. In this presentation we first present examples using real-world data as a control arm for SATs and to supplement an existing underpowered RCT control arm (hybrid). Matching and reweighting algorithm methods will be presented. Second, we will discuss the assumptions and validity of real-world control arms. This evaluation includes the use of equivalence testing (real-world versus RCT data), internal validation using a resampling approach, concurrency testing, and adjustment using the underpowered control data. Conducting validation analyses as presented here is a critical step for reliable use of real-world data as a full or hybrid control arm in clinical trials.


Authors who are presenting talks have a * after their name.

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