Already a DIA Member? Sign in. Not a member? Join.

Sign in

Forgot User ID? or Forgot Password?

Not a Member?

Create Account and Join

Virtual Event

Jun 14, 2020 9:00 AM - Jun 18, 2020 5:00 PM

(Eastern Standard Time)

Fort Washington, PA 19034

DIA 2020 Global Annual Meeting

Statistical Considerations for Using External Controls in Clinical Trials

Session Chair(s)

Jennifer  Clark, PhD

Jennifer Clark, PhD

Mathematical Statistician, OB, OTS, CDER

FDA, United States

A synthetic control arm (SCA) has been suggested as possibly advantageous as an external control to aid in the interpretation of clinical trials where a randomized concurrent control is clinically unethical, practically infeasible, or unacceptable to patients. Because the SCA is made up of patient level data from the real world or from historical clinical trials, there is opportunity to match the patient composition of the SCA to that of the experimentally treated group in baseline demographics and disease specific characteristics. This may be a considerable advantage over traditional external controls such as benchmarking with static results from medical literature or clinical intuition with populations that may not be sufficiently similar to the experimentally treated group. Speakers will provide a brief introduction to SCA and will discuss a variety of statistical approaches for the creation and use of SCAs. Discussion may include the use of clinical trials data and real-world data for construction of SCAs, various statistical matching and weighting methods for creation of the SCA, the possible impact of unobserved or unavailable historical information on the balancing process and the treatment effect, and others.

Learning Objective : Define a synthetic control arm (SCA) and the proposal for use of SCA in indications where a concurrent randomized control is not ethical or not feasible; Discuss the advantages and disadvantages of real world data and clinical trials data in a SCA; Describe a variety of statistical methods associated with construction and use of SCAs.

Speaker(s)

Ruthie  Davi, PhD, MS

Synthetic Control Arms as External Controls in Evaluation of Treatment Effects in Drug Development

Ruthie Davi, PhD, MS

Medidata, a Dassault Systèmes Company, United States

Vice President, Data Science and Statistician

Luis  Rojas, PhD, MS

Treatment Effect and Variability When Using External Comparators in RCTs: A Statistical Epistemological Approach

Luis Rojas, PhD, MS

Clinipace, United States

Vice President, Biostatistics

Andrew  Mulberg, MD

Industry Perspective

Andrew Mulberg, MD

Neurogene, United States

Senior Vice President

Have an account?

Be informed and stay engaged.

Don't miss an opportunity - join our mailing list to stay up to date on DIA insights and events.