The use and importance of real-world evidence (RWE) in clinical decision-making has been frequently cited by Food and Drug Administration (FDA) Commissioner Dr. Scott Gottlieb, so it’s not surprising that the topic will be top of mind at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 23rd Annual International Meeting on May 19-23 in Baltimore, Maryland. During the meeting, National Pharmaceutical Council (NPC) staff will share their expertise on real-world evidence and related issues during panel discussions, symposia and other presentations.
Real-world data (RWD), collected from everyday health care experiences, is growing in availability, and many health care decision-makers are interested in understanding and utilizing this information. However, advanced analytics and methods are needed to make sense of this data to create RWE. Many health care decisions are made based on randomized controlled trials, which take place in highly controlled settings and may not reflect the experience of the average patient. As we transition to a learning health care system and try to understand better what “works” in quicker and more cost-efficient ways, we are learning to draw upon these lessons everywhere we can find them — in communities and clinics, in patients’ homes and nursing homes, in laboratories and living rooms.
Under the 21st Century Cures Act, the FDA is required to explore and create a framework by the end of 2018 to help guide when and how RWE studies could be used to support regulatory decisions — for example, clarifying under what conditions RWE might be used to support the approval of a new indication for already-approved treatments, or to help support or satisfy post-approval study requirements. This framework on the use of RWE will provide the backbone for FDA guidance to inform both the agency and biopharmaceutical companies. Under Commissioner Gottlieb, the agency has prioritized RWE and other types of RWD as key tools in pre- and post-market clinical safety and effectiveness, which can help evaluate and bring treatments to market more quickly. And, as Dr. Gottlieb has noted in public comments, a sharper focus on RWE is leading to rapid increases in the rigor and value of the type and use of data available.
Beyond regulatory decisions, RWE can inform coverage and reimbursement decisions as well as clinical practice guidelines, and complement the existing data. NPC has weighed in on the importance of communicating high-quality information by testifying before the FDA and submitting comments related to the communication of RWE and other health data between biopharmaceutical manufacturers and payers. NPC has longstanding research in these areas, playing a leading role in encouraging the use of high-quality methodology and standards to conduct real-world research and in helping decision-makers understand how to evaluate and use RWE. NPC has helped to facilitate the GRACE principles and checklist to guide good practices for the design, conduct, analysis and reporting of observational studies for researchers and others; developed the Comparative Effectiveness Research Collaborative, along with the Academy of Managed Care Pharmacy and ISPOR to assist decision-makers in assessing the relevance and credibility of prospective and retrospective real-world studies; and conducted numerous studies on data access, use of RWE and methods.
NPC Vice President of Comparative Effectiveness Research Jennifer Graff, PharmD, will discuss RWE and the 21st Century Cures Act at ISPOR’s Annual International Meeting in a symposium on Monday, May 21, at 12:30 p.m. She will be joined by David Thompson, PhD, Senior Vice President, Real World & Late Phase, Syneos Health; Cathy W Critchlow, PhD, Vice President, Center for Observational Research, Amgen Inc; and David Martin, MPH, Captain, U.S. Public Health Service, and Associate Director for Real World Evidence Analytics, U.S. Food and Drug Administration, Office of Medical Policy, Center for Drug Evaluation and Research. Be sure to join NPC at our other sessions at the ISPOR Annual International Meeting, and visit NPC at Exhibition Booth #426 to learn more about our latest work.