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. Possibilities for medical product development in the context of this wealth of real-world data are great, 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. An ad hoc committee will plan and conduct a one-day public workshop that will examine opportunities and challenges for incorporating real-world evidence into evaluation of medical products.

Subject matter experts will be invited to participate in the workshop through presentations and discussions that will consider:

· Quality of data from real-world sources, including:
-- Relevance and validity of different sources of real-world data (e.g., user-collected, practice-based) in the context of different clinical/scientific questions; and
-- Strengths and limitations of different data sources at different stages of treatment development and licensing process.

· Methodologies and best practices for high-quality real-world evidence generation and application, including:
-- Innovations in clinical trial design to maximize value of information for the full range of stakeholders;
-- Considerations of how evidence generation through postmarket studies could potentially inform the design of preapproval clinical trials and amplify understanding of product efficacy;
-- Discussion of how shared goals of payers and regulators can better align evidence generation processes used for regulatory evaluation and decisions on use by payers; and
-- Re-evaluation of traditional distinctions between goals and methods of pre-approval and post-approval research.

· Other novel methodologies and approaches to improve development and evaluation of products using real-world evidence, including:
-- Use of web-based or digital technologies to enhance clinical trial evidence collection and participation.
-- Techniques and case-studies for effectively using electronic health record (EHR) data.

The committee will develop the agenda for the workshop, select and invite speakers and discussants, and moderate or identify moderators for the discussions. A summary of the workshop will be prepared by a designated rapporteur in accordance with institutional guidelines.