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All Times EDT

Thursday, September 24
Thu, Sep 24, 1:30 PM - 2:45 PM
Virtual
Application of Real-World Evidence to Drug Development: Statistical and Machine Learning Methodologies

Real-World External Control Arms: Design and Methods Considerations and Applications in Oncology Clinical Development (301228)

Gillis S Carrigan, Amgen 
*Khaled Sarsour, Genentech 

Keywords: External controls applications, pharmacoepidemiology, Data Quality and relevancy, Regulatory risks

The concept of using external data to supplement or replace the control arm in a clinical trial is not new (Pocock, 1976). Recently, progress on the tools and methodologies to consolidate, organize, and structure real-world data (RWD) have led to interest in leveraging RWD for external controls to inform drug development research go/no go decisions and to inform regulatory and payer approval and reimbursement decisions. This presentation will discuss prerequisite elements of RWD quality and relevancy and elements needed in the design and analysis phase of to ensure valid inferences drawn from RWD based external control studies. The presentation will also discuss six applications of RWD-based external control studies in oncology drug development. These are:

1.Benchmarking: providing contextual information about the performance of the control arm 2.Assessing efficacy in early phase single arm oncology trials to inform go/no go decisions 3.Assessment of RCT control arm performance in trial design phase providing evidence for sample size assumptions and effect size 4.Early evaluation of blinded phase III trials 5.Accelerate late phase trial enrollment/reduce time to complete trials: Hybrid Controls 6.Comparative effectiveness: Pre-marketing indirect treatment comparisons

Focus will be on best practices in pharmaoepidemiologic and statistical design and methodology considerations. Discussion will be made of limitations and regulatory risks.