Using patient outcomes data to inform and advance the development of emerging cell and gene therapiesPublished: June 3, 2019
This On Demand focuses on patient outcomes tracking through registries and the ability to leverage real world evidence and outcomes data collected from the first-generation of approved cell and gene therapies to inform the development of second-generation cell and gene therapy products, and the future of the field overall.
Innovative cell and gene therapies are progressing to market at an unprecedented pace. In the new era of personalized medicine, developers of new cell therapies are mandated by the FDA to track long-term patient outcomes, at time increments up to 15 years post-intervention, to truly measure the safety and efficacy of these treatments. As the number of patients commercially treated with these therapies continues to grow, long-term outcomes data has the potential to influence the entire industry of personalized cell and gene therapies.
But how is the industry developing protocols and secure data storage systems for collecting, maintaining, and analyzing outcomes data for patients’ post-therapeutic intervention when the process is time-consuming, costly and has no room for error?
View this On Demand Webinar for expert insights into how such data is collected and real-world examples of its utility in addressing some of the challenges that emerging cell and gene therapies are facing.
- Learn how outsourcing tracking to experts can minimize room for error, as well as time and resource burden on companies focusing on bringing therapies to market.
- Understand the impact outcomes data tracking has on ensuring innovative therapies are safe and effective, while also providing the retrospective data to expedite approvals and delivery.
- Find out how outcomes databases can be utilized to advance the design and implementation of clinical trials, regulatory approval, product quality, and patient management.
Dawn Henke, PhD Senior Technical Program Manager, Standards Coordinating Body (SCB)
Dawn Henke is the Senior Technical Program Manager at the Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-Based Drug Discovery (SCB). The SCB brings together product developers, service providers, professional societies, government entities, and academic centers with the intent to support standards development in the regenerative medicine field. Previously Dawn received her Ph.D. in genetics from University of Alabama at Birmingham and worked as a post-doctoral fellow at the NIH/NEI in stem cell research for two years.
Charles Wilfong, Global Vice President, Information Management & Facility Engineering Functions, Adaptimmune
Charles Wilfong is Vice President of Information Management and Facilities Engineering functions at Adaptimmune, where he is responsible for global strategies and operations. He has 23 years experience across the pharmaceutical, biotechnology and engineering industries supporting Research, Clinical, Manufacturing and Commercial functions. Charles has led several large cross-functional change management initiatives to digitize core business process and make better use of data and analytics. He has a Bachelor of Architectural Engineering from the Pennsylvania State University, and a Master of Business Administration and Master of Public and International Affairs from University of Pittsburgh.
Marcelo C. Pasquini, MD, Associate Professor of Medicine, Hematology, Medical College of Wisconsin
Marcelo C. Pasquini, MD, MS, is Associate Professor of Medicine in the Division of Hematology and Oncology. He completed medical school at the State University of Londrina, Brazil; postgraduate training in Internal Medicine at the University of Miami/Jackson Memorial Hospital; Hematology and Oncology at the University of Utah; and Blood and Marrow Transplantation at the Medical College of Wisconsin. He received his Masters of Science degree in Epidemiology from the Medical College of Wisconsin. Dr. Pasquini also serves as Senior Scientific Director for Clinical Trials at the Center for International Blood and Marrow Transplant Research (CIBMTR) where he focuses on clinical research in the field of transplantation and cellular therapy. Dr. Pasquini also oversees the cellular therapy outcomes database at the CIBMTR. He is also a member of the Hematopoietic cell transplant and Cellular Therapy Clinical Program at the Medical College of Wisconsin.