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Friday, September 14
Fri, Sep 14, 1:30 PM - 2:45 PM
Thurgood Marshall South
Utility of Real-World Evidence in Medical Product Development and Life Cycle Management

Utility of Real-World Evidence in Medical Product Development and Life Cycle Management (300745)

*Frank W Rockhold, Duke University Medical Center 

Keywords: Pragmatic Trials, Real World Data

There is currently a drive and energy behind the use of “Big data” and “Real World Data” (RWD) to generate Real World Evidence (RWE). There are three main motivations: Use of RWD are viewed as 1) a quicker and less expensive approach to acquiring data for clinical development, 2) a way to answer questions of how treatments are or will be used in the real world and generate true RWE, and 3) a potential source of valid observational studies.

One of the current issues is the blurring in the use of RWD to answer scientific and medically important questions and if any use of RWD is somehow distinct from the conduct randomized trials and only used to do observational studies. In this sense, the debate is often posed as “should I do a RWD (Observational} study OR a clinical trial” or “how can I show that an observational study will yield the same answer as an RCT”. The truth for drug development is this should be an “AND” discussion not an “OR” discussion as both approaches can be valuable. In addition, more care should be given not to avoid the concept of randomization too quickly in the use of RWE as there many techniques allow us to bridge that gap. The use of Pragmatic Randomized Trials (including cluster randomized trials) will be discussed in that light. While RWD is usually restricted to post marketing trials, the potential use of RWD in preapproved treatments will also be considered.