Real-World Evidence Generation and Evaluation of Therapeutics: A Workshop

Workshop

October 19, 2016

12:00 AM - 12:00 AM (ET)

Past

Materials

About

Workshop Background and Objectives

The traditional process for evaluating new therapeutics often does not generate the evidence that patients, clinicians, and payers need to make practical real-world decisions. The volume and complexity of information about individual patients continues to grow with use of electronic records and digital health technologies. Real-world data offer numerous opportunities for improving medical product development - from enhancing the ability of decision-makers to determine both large-scale and patient-specific effects of treatments to enabling more patient-centered engagement and input throughout the research and development life cycle. However, mechanisms to facilitate efficient use of real-world data to meet the decision-making needs of myriad stakeholders have not been well established.

This one-day public workshop examined opportunities and challenges for incorporating real-world evidence into the evaluation of medical products. Workshop participants were invited to 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 from existing studies could potentially inform the design of future 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.

A Discussion Paper authored by the workshop co-chairs was released in conjunction with the workshop.

Below are materials for the workshop, including an agenda, discussion papers, and presentations

For additional information on the workshop, including the planning committee and sponsors, please visit our project page.

Related Activities

Examining the Impact of Real-World Evidence on Medical Product Development: A Workshop Series

Contact

Amanda Wagner Gee

agee@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

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Real-World Evidence Generation and Evaluation of Therapeutics: A Workshop

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Real-World Evidence Generation and Evaluation of Therapeutics: A Workshop