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DIA/FDA Biostatistics Industry and Regulator Forum

This event is now offered in a new entirely virtual format.


Session 5: New Uses of External Controls Leveraging Patient Level Data and Statistical Matching or Weighting in a Regulatory Setting

Session Chair(s)

Ruthie  Davi, PhD, MS

Ruthie Davi, PhD, MS

  • Vice President, Data Science and Statistician
  • Medidata, a Dassault Systèmes company, United States
Meiyu  Shen, PhD

Meiyu Shen, PhD

  • Expert Mathematical Statistician, Office of Translational Sciences, CDER
  • FDA, United States
Increased availability of patient-level data and recent regulatory decisions encouraging the use of innovative approaches to accelerate medical product development are fueling the impetus to incorporate external control data into clinical development settings. Potential patient-level data sources are wide-ranging and include historical clinical trials data available through growing data sharing programs, registry data collected in order to advance the understanding of a particular disease, and real-world data electronically stored as part of clinical practice. This session will 1) discuss statistical methods, such as matching and weighting, that are fueling the ability to create well-balanced external control cohorts, 2) examine statistical considerations specific to the various types of patient-level data sources, and 3) present case studies and examples of the use of external controls in clinical development and regulatory settings.
Learning Objective : At the conclusion of this session, participants should be able to:
  • Describe the general approach for creating an external control from patient-level data
  • Identify potential areas where these types of external controls may facilitate product development
  • Discuss key factors affecting the adequacy of external controls for elucidating treatment effects

Speaker(s)

Antara  Majumdar, PhD

Speaker

Antara Majumdar, PhD

  • Director of Statistics
  • Acorn AI by Medidata, a Dassault Systèmes company, United States
Brent  McHenry, PhD

Across-Trial Propensity Score Analysis to Support Contribution of Components of Oncology

Brent McHenry, PhD

  • Director, RCC and Prostate Biostatistics Lead
  • Bristol-Myers Squibb, United States
Aloka  Chakravarty, PhD

Panelist

Aloka Chakravarty, PhD

  • Senior Statistical Advisor, Office of the Commissioner
  • FDA, United States
Pallavi  Mishra-Kalyani, PhD, MS

Panelist

Pallavi Mishra-Kalyani, PhD, MS

  • Lead Mathematical Statistician, CDER
  • FDA, United States