Session 2: Synthetic Controls and Causal Inference

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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

Vice President, Data Science and Statistician

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)

Use of a Synthetic Control Arm in Medical Product Development

Xiang Yin, PhD

Medidata Solutions, A Dassault Systèmes Company, United States

Vice President, Statistics and Regulatory Sciences

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

Yeh-Fong Chen, PhD

Food and Drug Administration, United States

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER

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

Mehmet Burcu, PhD, MS, FISPE

Merck & Co., Inc., United States

Senior Director, Epidemiology

DIA Biostatistics Industry and Regulator Forum