An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will plan and conduct a three-part workshop series to be held over the course of a two-year period. As part of FDA’s continued focus on building a national governance system for evidence generation, the proposed focus of these workshops will be on the generation and utilization of real-world evidence to evaluate efficacy, effectiveness, tolerability, and safety for both review of new indications and post-approval studies. These workshops would include presentations and perspectives from thought and knowledge leaders representing a range of disciplines, including but not limited to federal regulatory and funding agencies, clinical and academic medicine and research, medical professional organizations, the regulated biopharmaceutical industry, patients and patient-focused and disease-advocacy organizations, payers, consumer organizations, health systems, and other interested stakeholders that represent the myriad views of those involved in drug, biologic, and device discovery, development, translation, and regulation. The workshop audiences are expected to be similarly diverse, and they will have opportunities to engage in discussion during the workshops. Objectives:

• To advance discussions and common knowledge among key stakeholders (including FDA and the public) about complex issues relating to the generation and utilization of real-world evidence.

• To foster development and implementation of the science and technology of real-world evidence generation and utilization.

• Topics the workshops will address:

  • Definitions surrounding the core components of real-world evidence.

  • Sources of data that are curated, standardized, and analyzed to derive real-world evidence, such as safety surveillance, observational studies, registries, claims, or patient-centered outcomes research.

  • Gaps in data collection activities, and priority areas and pilot opportunities that real-world evidence incorporation could address.

  • Standards and methodologies for collecting and analyzing real-world evidence in support of new indications or post-approval studies, and the circumstances under which that evidence could be applied.

  • Applications for using real-world evidence to supplement traditional clinical trials, pragmatic/effectiveness trials, or routine clinical application.

  • Mechanisms for determining which discrete types of real-world evidence could support regulatory decisions.

  • Operational challenges and barriers for generating and incorporating real-world evidence in the context of a learning health system and how clinicians can best be involved in the collection and utilization of real-world evidence.

The planning committee will develop the agenda for each workshop, select and invite speakers and discussants, and moderate or identify moderators for the workshop discussions. A proceedings of the workshops shall be prepared by a designated rapporteur in accordance with institutional guidelines and shall be released to the public after the three workshops have been conducted. The workshop proceedings will summarize and aggregate the key elements of the workshop series.