Today, FDA published a notice in the Federal Register announcing a public workshop on July 11-12 entitled “Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes.”  This workshop builds on FDA’s ongoing efforts to implement the Real-World Evidence (RWE) Framework published in December 2018, which we previously discussed in this post.

According to the Federal Register notice, the public workshop is being convened by Duke University’s Robert J. Margolis, MD, Center for Health Policy (Duke Margolis) and supported by a cooperative agreement with FDA.  The workshop will cover considerations for using randomized clinical trial designs and real-world data (RWD) to generate RWE, including the following –

  • the selection of interventions appropriate in clinical care settings;
  • study design elements and study populations;
  • capturing outcomes in clinical care settings;
  • addressing potential challenges around blinding, randomization, and bias; and
  • regulatory considerations for randomized clinical trials using RWD (e.g., safety and product monitoring and maintaining data integrity).

This public workshop will follow on the heels of a meeting held by the Department of Health and Human Services (HHS) to “seek public input and comment on opportunities to leverage departmental resources, increase collaboration, and to partner with private stakeholders in the service of accelerating the process for clinical innovation in the United States.”  The HHS meeting also was announced recently in the Federal Register and will take place on June 20-21.