The Challenges of Long-Term Follow-Up Studies for Cell and Gene Therapies
March 28, 2021
Welcome to the inaugural episode of RARE-Xtra, an occasional RARE-X podcast where we take a deeper dive with experts on critical issues for the rare disease community. RARE-X recently produced a white paper in partnership with the ARM Foundation for Cell and Gene Medicine, which 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.
For this episode, RARE-X and the ARM 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-based tackled through the collaboration of all rare disease stakeholders.
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