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Statistical Considerations for Using External Controls in Clinical Trials
Session Chair(s)
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)
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
Treatment Effect and Variability When Using External Comparators in RCTs: A Statistical Epistemological Approach
Luis Rojas, PhD, MS
Clinipace, United States
Vice President, Biostatistics
Industry Perspective
Andrew Mulberg, MD
Neurogene, United States
Senior Vice President
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