March 30, 2021
This paper explores the primary issues and requirements for data gathering that cell and gene therapy developers face. It also considers solutions, and the role a standardized non-proprietary data collection platform, such as RARE-X, can play in addressing these challenges.
Foundation for Cell & Gene Medicine and Rare-X: "There is a real need for a community approach."
The Challenges of Long-Term Follow-Up Studies for Cell and Gene Therapies:
Many people seem to view long term follow up as an industry problem. How ready are cell and gene therapy makers for this task? Robust data collection is a rare disease community issue.
RARE-X recently produced a white paper with the Foundation for Cell & Gene Medicine. The Foundation for Cell & Gene Medicine is dedicated to providing education, information, and research needed to expand patient awareness of and accelerate access to transformative therapies. The white paper explored the long-term data requirements for cell and gene therapies. RARE-X and the Foundation expand on that white paper with a discussion featuring:
Betsy Bogard, who has worked in the industry for cell and gene therapy developers;
Craig Lipset, founder of Clinical Innovation Partners and an adjunct assistant professor in the department of health informatics at Rutgers University; and
Ian Winburn, global medical lead for hemophilia, endocrine, and inborn errors of metabolism, in the rare disease division of Pfizer.
RARE-X’s Daniel Levine moderates the discussion. They explore the challenges for long-term follow-up of patients who receive cell and gene therapies, why technology may represent the least of the problems, and why it’s an issue best- tackled through the collaboration of all rare disease stakeholders.
