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Future of Evidence and Smart Health Conference

Leveraging RWE to advance healthcare decision-making.

Session 8: Unwrapping RCT Duplicate

Session Chair(s)

Claudia  Cabrera

Claudia Cabrera

  • Senior Director Epidemiology & Evidence Excellence, AstraZeneca AB
  • Adj Researcher Karolinska Institute, Dept Epidemiology and Biostatistics, Sweden

The demand for data quality and quantity has expanded exponentially in the past decade. There has been a tremendous effort to investigate the potential of real world data (RWD) to provide real world evidence (RWE) based on real world science (RWS). Industry and governments are assessing the power of data to potentially drive health strategy, safety, clinical trial investigations, and more general public health decisions. This challenge was clearly elucidated through the passing of the 21st Century Cures Act in 2016, mandating legislation and guidance around the use and interpretation of RW data and evidence. It also highlighted that RWE will need to consider methodologies used to generate evidence and the reliability and relevance of the RWD. Currently available data sources often reflect local health policies and services, RW data may be generated from commercial sources, reflecting claims and billing activities. Other data source types include electronic health records (EHRs) from hospital and primary care facilities, an example being the UK CPRD; while national health registries are prominently available for research purposes in the Nordic countries for the monitoring and guidance of public health.

The RCT Duplicate Framework embraces this challenge. It is an FDA sponsored ~ 3-year series of studies conducted by Brigham Women's & Harvard University and AETION. It involves the replication of 30 published RCTs and contains additional FDA funding to predict the results of 7 ongoing Phase IV RCTs i.e. CAROLINA. Unwrapping RCT Duplicate will be a session where we describe an attempt to emulate their current efforts and invite others to discuss the topic, along with FDA representatives allowing for a more in-depth understanding of the work being conducted and the outcomes expected.

Objectives of this session will be:

  • To support Safety related questions such as changes to labelling regarding drug product effectiveness, including adding or modifying an indication (i.e. dose, dose regimen, or route of administration)
  • To strengthen and impact knowledge on quality assessment of RW data-based studies in order to for example: Accelerate RCT programs through enhanced methodology including RWE


Theodor  Framke

Overview of Innovative Designs seen by European Regulators

Theodor Framke

  • Seconded National Expert
  • European Medicines Agency, Netherlands
Sebastian  Schneeweiss, DrSc, MD

Calibrating Real-World Evidence Against RCT Evidence: Key Findings From the RCT-Duplicate Program

Sebastian Schneeweiss, DrSc, MD

  • Professor, Medicine and Epidemiology; Chief of the Div of Pharmacoepidemiology
  • Harvard Medical School and Brigham and Women's Hospital, United States
Elizabeth  Garry, PhD, MPH

The RCT Duplicate Process

Elizabeth Garry, PhD, MPH

  • Senior Principal Scientist
  • Aetion, Inc., United States
Claudia  Cabrera

Unwrapping RCT Duplicate: A Discussion on Clinical Trial Emulations

Claudia Cabrera

  • Senior Director Epidemiology & Evidence Excellence, AstraZeneca AB
  • Adj Researcher Karolinska Institute, Dept Epidemiology and Biostatistics, Sweden

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