The Foundation for Cell & Gene Medicine and RARE-X Release White Paper on Using Patient Owned Data
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.
