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FDA and Duke University to Explore Expanded Use of Real-World Evidence for Drugs in Public Workshop

On September 13, 2017, FDA and the Duke-Robert J. Margolis, MD Center for Health Policy will bring stakeholders to discuss a variety of topics related to the use of real-world data (RWD) and real-world evidence (RWE) in drug development and decision making. Topics in general will include an update on FDA’s activities to address the use of RWE in regulatory decision and development of a framework for handling challenges related to accepting RWE. Panelists are expected to discuss opportunities to improve data development activities, study designs, and analytical data to create robust RWE. The FDA contact lead is its Center for Drug Evaluation and Research, although its Center for Devices and Radiological Health has a parallel initiative underway.

The 21st Century Cures Act was enacted on December 13, 2016, directed FDA to establish a program to evaluate the potential use of RWE in the drug context, including describing its sources, the gaps from data collection, standards and methods for data collection and analysis, and priority areas and challenges (Pub. L. 114-255, §3022). In the past, FDA has used RWE for historical controls in clinical trials and post-market safety surveillance. Under the new paradigm, FDA will be establishing a program to support new indication(s) and to satisfy post-approval study requirements.

In July 2016, CDRH and the Center for Biologics Evaluation and Research published a draft guidance, “Use of Real-World Evidence to Support Regulatory Decision-Making.” This guidance defines RWD as “data collected from sources outside of traditional clinical trials … typically derived from electronic systems used in health care delivery, data contained within medical devices, and/or in tracking patient experience during health care, including in home-use settings.” The guidance further defines RWE as “the evidence derived from the aggregation and analysis of RWD elements.” We expect input at this public meeting to be considered when FDA finalizes the CDRH guidance, an action prioritized for Fiscal Year 2017.

As stated in the Guidance, FDA has recognized that a “wealth of data covering medical device experience exists and is routinely collected in the course of treatment and the management of patients.” While RWE has the potential to contribute to a better understanding of the benefit/risk profile of a medical device, particularly with an expanded population base beyond a well-controlled clinical study, data quality varies greatly and needs to be limited in terms of bias and needs to be pooled in terms of source in order to be deemed “regulatory grade” and have the ability to influence regulatory decision-making.

The Guidance elaborates that RWE may be used in the context of medical devices for:

  1. Expanded indications for use;
  2. Postmarket surveillance studies;
  3. Post-approval device surveillance as a condition of approval;
  4. Developing a control group;
  5. Supplementary data to well-controlled clinical studies; and
  6. Objective performance criteria and performance goals.

Appropriately leveraging such uses can provide insight into how a medical device meets its objectives during its product life cycle, as well as generate hypotheses, controls, registries, patient populations, and the evolution of acceptable benefit/risk profiles.  

Just how similar will CDER’s guidance for the use of RWD and RWE look to the model proposed by CDRH and CBER? The public meeting will be held on September 13, 2017 from 9:00 am to 4:30 pm EST at 1777 F Street NW, Washington, DC 20006. At this point in-person registration is full and cannot be accepted on site, but the webinar is available for registration.

Arent Fox's Food, Drug, Medical Device & Agriculture group will continue to monitor developments in this area. If you have any questions, please contact Brian Malkin or Paul Gadiock (who plans to attend the public meeting), or the Arent Fox professional who regularly handles your matters.