Appendix B

Workshop Agenda

REAL-WORLD EVIDENCE GENERATION AND EVALUATION OF THERAPEUTICS: A WORKSHOP

October 19, 2016
National Academy of Sciences Building, Room 120
2101 Constitution Avenue, NW, Washington, DC 20418

BACKGROUND AND WORKSHOP OBJECTIVES

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 1-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 the 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 preapproval and postapproval 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; and
  • Techniques and case studies for effectively using electronic health record data.

Session Objectives:

• Examine different stakeholder evidence needs to support decision making and identify shared goals.
• Discuss priorities for facilitating use of real-world evidence to address stakeholder needs.
• Discuss aligning incentives to maximize generation and sharing of useful evidence.

Session Objectives:

• Examine different sources of real-world data (e.g., user collected, practice based) and consider their reliability in the context of different clinical/scientific questions.
• Discuss strengths and limitations of different data sources at different stages of treatment development and licensing process.

Session Objectives:

• Discuss examples of successful approaches to generating and incorporating real-world evidence into development and evaluation of medical products.
• Identify opportunities and challenges to scaling up successful practices and adapting them to new purposes.

Session Objectives:

• Outline an ideal future state for incorporating real-world evidence into evaluation of medical products.
• Identify short- and long-term next steps at any stage of clinical research to achieve seamless use of real-world evidence.
• Discuss incentives that should be explored.