Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop


Note: Proceedings contain the opinion of the presenters, but do NOT reflect the conclusions of the Health and Medicine Division or the National Academies. Learn more about the differences between Reports and Proceedings.

The traditional process for evaluating new therapeutics does not produce the evidence that patients, clinicians, and payers need for real-world decisions. The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data are numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to assessing how therapeutics affect patients’ lives through measurement of lifestyle changes. However, mechanisms to facilitate efficient use of real-world data to meet the decision-making needs of myriad stakeholders have not been established. This workshop, held in October 2016, examined opportunities and challenges for incorporating real-world evidence into evaluation of medical products, including consideration of quality, relevance, and validity of real-world data sources; methodologies and best practices for incorporation of real-world data throughout the product lifecycle; and use of novel approaches such as web-based or other digital technologies. A National Academy of Medicine Discussion Paper authored by the workshop co-chairs was made available in conjunction with the workshop.