Long Term Follow-Up for Gene Therapies – what you need to know.
Sponsored by: Covance
- Gene Therapy
Date: 12 December
Days to go: 3
Time: 4PM London/11AM New York
Practical LTFU design considerations
Gene therapy (GT) products represent a paradigm shift in the treatment of long-term and/or life threatening illnesses. While these treatments potentially can transform the lives of these patients with a single dose, companies developing and marketing GTs will be challenged to monitor safety and outcomes over the long term, especially around vectors with a propensity for genome modification.
As the number of gene therapies in development and on the market grows, so too does the challenge of interpreting global regulator guidance on long-term follow-up (LTFU) and translating that into workable solutions that gather sufficient quality data to provide ongoing safety assurances, without which treatments risk never being approved for use.
Potential solutions include site-based long term studies, which may be inconvenient for patients over the long term. While new technologies like wearable devices, mobile applications and self-reporting are increasingly being proposed as useful tools, no single approach will succeed. Instead, a pragmatic, patient-centric approach that fits patient demographics and therapy offers the greatest prospect for success. By “designing-in” patient centricity to LTFU, it is possible to maximize participation while being robust enough to derive meaningful data.
This webinar explores current regulator guidance drawing on insights from products entering the market, and weighs up the pros and cons of different LTFU techniques, and the challenges of long-term patient consent, putting patient convenience at the heart of the solution.
Richard Williams, PhD, JD,
Executive Director Strategic Product Development Consulting, Covance Regulatory Affairs
Dr. Richard Williams is an Executive Strategist within the Drug Development Consulting group at Covance. Richard has substantial experience in multiple therapeutic areas and has direct experience interacting with FDA, EMA, and PMDA. He is the Co-Chair of Covance's Advanced Therapies Expert Working Group, which has an emphasis on gene and cell therapy. Richard is especially interested in the integration of business with science. Prior to joining Covance, Dr. Williams held positions of increasing responsibility in clinical research and regulatory affairs for large pharmaceutical companies as well as smaller successful start-up companies. He has raised >$100M for start-up companies and has taken investigational products through to positive value inflection points, including blockbuster drugs (>$1B) and cutting-edge products in emerging companies. Dr. Williams received his Ph.D. from the University of London.
Jennifer Urwongse, MBA,
Associate Director, Health Economics & Outcomes Research, Covance Market Access
Jennifer Urwongse, MBA, is an Associate Director at Covance Inc. Covance, a subsidiary of LabCorp, is a global contract research organization and drug development service company. Jennifer has overseen multinational health economic and outcomes research (HEOR) studies as part of the Covance Market Access consulting practice since 2009. Over the last four years, Jennifer has focused on the design, development and successful execution of virtual real world evidence studies, including fully "site-less" virtual studies for regulatory submissions. Prior to joining Covance, Jennifer was a management consultant for a life science consulting firm and an investment banker at a major multinational investment bank. Jennifer received a BA in Economics with a concentration in Finance from University of Virginia, and an MBA from the Massachusetts Institute of Technology.
Key Learning Objectives
- Develop an understanding of current regulator guidance re: LTFU
- Review challenges of long-term patient consent and long-term compliance
- Discuss the pros and cons of different LTFU techniques
- How putting patient convenience at the heart of the solution moves insights forward
- Director or VPs of:
- Market Access
- Patient Support
- Patient Support Strategy
- Market Access and Patient Support
- Managed Markets Strategy & Operations
- Reimbursement and Market Access
- Reimbursement and Patient Assistance
- Brand Management
- Patient Marketing
- Payer Marketing
- Marketing and advertising