Collection of papers on Real-World Evidence from the National Academies workshop series
The National Academies of Sciences, Engineering, and Medicine convened a three-part workshop series, sponsored by the U.S. Food and Drug Administration, examining how real-world evidence development and uptake could enhance medical product development and evaluation. The workshops advanced discussions and common knowledge about complex issues relating to the generation and utilization of real-world evidence, including fostering development and implementation of the science and technology of real-world evidence generation and utilization.
- Workshop One (September 19–20, 2017) focused on how to align incentives to support collection and use of real-world evidence in health product review, payment, and delivery. Incentives need to address barriers impeding the uptake of real-world evidence, including barriers to transparency.
- Workshop Two (March 6–7, 2018) was a “town-hall” style meeting with active audience participation to illuminate what types of data are appropriate for what specific purposes and to suggest approaches for data collection and evidence use by developing and working through example use cases.
- Workshop Three (July 17–18, 2018) examined and suggested approaches for operationalizing the collection and use of real-world evidence.
Below are materials for the workshop, including an agenda, briefing book, and presentations. View archived webcast footage of the presentations and discussions here.
For additional information on the workshop, including the planning committee and sponsors, please visit our project page.
In June 2021, some individual workshop speakers and attendees published a set of 4 journal articles inspired by what they learned at this workshop series and taking into account lessons learned from the COVID-19 pandemic. Links to the open access articles published in Clinical Pharmacology & Therapeutics are below:
- When Can We Rely on Real-World Evidence to Evaluate New Medical Treatments? outlines the paper series’ scope and context, and dimensions considered where real-world evidence studies may differ from traditional trials.
- When Can We Trust Real-World Data to Evaluate New Medical Treatments? outlines specific questions to help assess potential error or bias at each step of data generation from clinical presentation (including with a device) to inclusion in a database.
- When Are Treatment Blinding and Treatment Standardization Necessary in Real-World Clinical Trials? outlines some questions to help determine whether blinding and treatment standardization may be necessary for internal validity of a study or may distort meaningful differences between treatments in specific cases.
- When Can Nonrandomized Studies Support Valid Inference Regarding Effectiveness or Safety of New Medical Treatments? discusses “questions that can help investigators or evidence consumers evaluate the potential impact of confounding or other biases on their findings.”