Optimizing Endpoint Selection in Ophthalmic Gene Therapy Trials
Time: 9:00 am
day: Pre-Conference Workshop Day Clinical Track AM
Details:
Choosing appropriate endpoints is pivotal for accurately evaluating the efficacy of ophthalmic gene therapy. This
workshop provides a cohesive exploration of innovative strategies to enhance endpoint selection, ensuring validity,
reliability, and patient-centricity in ocular gene therapy trials.
Key Discussion Points:
- Advancing Visual Function Assessment: Exploring advancements in visual function assessment methodologies tailored for gene therapy trials, including the integration of active machine learning algorithms to optimize data collection and analysis.
- Enhancing Endpoint Validity and Reliability: Addressing challenges in determining appropriate endpoints, evaluating novel assessment methods, and discussing collaborative efforts to establish standardized endpoint criteria for improved trial design and interpretation.
- Defining Meaningful Endpoints: Examining the discrepancy between regulatory expectations and patient perspectives on meaningful outcomes, and discussing regulatory considerations for redefining clinical endpoints.
- Navigating Regulatory Expectations: Discussing adaptive trial designs, the potential of biomarkers as surrogate endpoints, and emphasizing patient-centered outcomes in compliance with regulatory standards, ensuring flexibility and validity in endpoint selection.
By the end of this workshop, participants will gain insights into the complexities of endpoint selection and acquire practical strategies to optimize endpoint selection.