Pre-Conference Workshop Day
Workshop A: Exploring Commitment to Gene Therapy Trials & Follow-Ups: Key Considerations
Tuesday, June 7 | 9.00am - 12.00pm
One of the several unique aspects of gene therapies as a therapeutic is the requirement for many years of follow-up in clinical trials, commonly for 15 years – whereas the gene therapy is only administered once. Given this high level of commitment, it’s crucial to ensure that the follow-up process is as easy to manage as possible and reduces burden on patients, ensuring high patient retention, adequate safety monitoring, and sufficient data to submit to regulators for approval. Crucial to this process is patient understanding of what commitment to a gene therapy trial will involve.
Attend this workshop to learn about:
• Understanding potential models for gene therapy trials
• Reducing patient burden over extensive follow-ups
• Detailing what the trial process will entail to make informed decisions
• Exploring examples of remote assessment
• Communicating effectively with patients on engagement with the clinical trial
• Setting expectations with trial participants and families
Workshop Leaders:
Vivian Fernandez
Senior Director, Patient
Advocacy
REGENXBIO
Sarah Friedhoff
Associate Director, Patient
Advocacy & Clinical Affairs
Abeona Therapeutics
Jodi Wolff
Senior Director, Patient
Advocacy
StrideBio
Workshop B: Engaging Patient Communities in Trial Design, Recruitment & Drug Development
Tuesday, June 7 | 13.00pm - 16.00pm
Several gene therapies currently in development are targeting rare and ultrarare diseases. Given the significant challenges associated with developing trials with small patient numbers compounded with the complexity of genetic therapies, the role of patient groups in finding patients and sharing vital input on clinical trial recruitment is even more important to utilize effectively
Attend this workshop to learn about:
• Collaborating effectively with patient groups on finding patients
• Making use of patient input to streamline recruitment processes, and understand what patients need and want to make an informed decision on enrolment
• Exploring the role of industry in progressing genetic testing and newborn screening
• Developing materials in association with patient groups
• Working alongside clinicians whilst remaining compliant
Workshop Leader:
Craig Martin
CEO
Global Genes