Real World Data and Evidence – FDA to Take Another Look

The FDA today, in the Federal Register, announced a public workshop to be held on September 12, 2017 to discuss the use of real world data (RWD) and real world evidence (RWE) in its regulatory decision making process.  The workshop, titled, “Developing a Framework for Regulatory Use of Real-World Evidence”, will be held in Washington DC. It is being convened by the Center for Health Policy at Duke University and supported by a cooperative agreement with FDA.  Its stated purpose “is to bring the stakeholder community together to discuss a variety of topics related to the use of real-world data (RWD) and real-world evidence (RWE) in drug development and regulatory decision making. Topics will include an update on FDA’s activities to address the use of RWE in regulatory decisions and the development of a framework for tackling challenges related to RWE’s regulatory acceptability. In addition, panelists will discuss opportunities to improve data development activities, study designs, and analytical methods used to create robust RWE.”

How many times in the world of pharmaceuticals have we heard, yes, it is not approved for that use but it really work, or the product has been around for 50 years and there are no bodies in the street.  Well, while it may be a leap to go that far, thinking that time alone or observational evidence could fully inform an FDA regulatory decision, it may be time to see how RWD and RWE could be incorporated into the decision framework.

RWD and RWE is currently being used in the healthcare field by payers to make formulary decisions, by health care providers to make clinical decisions (off label use), and “medical product developers can use RWE to further develop a product’s benefit-risk profile, monitor post market safety and adverse events, or generate additional hypotheses for continued clinical development.”

The 21st Century Cures Act directed FDA to establish a program with a framework to “include information describing the sources of RWE, the gaps in data collection, standards and methods for collection and analysis, and the priority areas and challenges.”  The notice goes on to say that FDA has used RWD and RWE in limited circumstances for instance in rare diseases “as part of the historical controls used for clinical study and, ultimately, regulatory submission. Postmarket safety surveillance has also relied heavily on RWD-generating network”

Issues to be discussed at the workshop will cover several issues, among which are the collection of such data and how to assure its quality, and” innovative methods for developing RWE from RWD, and promising areas for RWE pilot demonstrations.”

A full copy of the Federal Register notice can be found here. Maybe this will lead to better tools to assist in regulatory decision-making processes and reducing the need for some additional clinical testing or at least inform a better direction for such study.