DIA Biostatistics Industry and Regulator Forum

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Session 2: Synthetic Controls and Causal Inference

Session Chair(s)

Hana Lee, PHD

Senior Statistical Reviewer, OB/OTS/CDER, FDA, United States

Ruthie Davi, PHD, MS

Senior Vice President, Medidata, a Dassault Systèmes Company, United States

This session will explore the use of propensity scores or other balancing algorithms to create synthetic or external controls to aid in medical product development. Examples include

alignment of a synthetic control to a single arm trial early in development to facilitate go/no-go decisions
utilizing a synthetic control arm for enhanced understanding of a treatment effect observed in a single arm trial to support an accelerate approval submission

The main advantage of the synthetic control, over traditional benchmarks referencing medical literature or created with clinical intuition, is the potential for well-balanced condition of the synthetic control patients and the investigational patients at baseline. This session will discuss general principles with a focus on examples. We will discuss cases where synthetic controls have been used, the statistical methods applied, and the implications of doing so. Examples may include regulatory or nonregulatory applications.

Learning Objective :
Define what a synthetic control is

Describe how a synthetic control may enhance the interpretation of a single arm trial

Identify uses of synthetic controls in the regulatory and nonregulatory spaces

Speaker(s)

Xiang Yin, PHD

Vice President, Statistics and Regulatory Sciences, Medidata Solutions, A Dassault Systèmes Company, United States

Use of a Synthetic Control Arm in Medical Product Development

Yeh-Fong Chen, PHD

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, Food and Drug Administration, United States

Regulatory Review Experience of Single-Arm Studies Utilizing External Trial Information

Mehmet Burcu, PHD, MS, FISPE

Senior Director, Epidemiology, Merck & Co., Inc., United States

Real-world Evidence to Support Regulatory Decision-making for Medicines: Considerations for External Control Arms.