Exploring Novel Clinical Trial Designs for Gene-Based Therapies Proceedings of a Workshop (2020) / Chapter Skim
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6 Reflections on the Workshop and Potential Opportunities for Next Steps
6 Reflections on the Workshop and Potential Opportunities for Next Steps
Pages 69-76
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From page 69... ...
Panelists were asked to consider ideas emerging from the workshop discussions and suggest specific changes that could improve the design of gene therapy clinical trials and the overall experience for participants and their families. POSSIBLE NEXT STEPS FOR IMPROVING THE DESIGN OF GENE THERAPY CLINICAL TRIALS When asked to identify the important points that emerged during the workshop, High emphasized the benefits of understanding the natural history of a disorder that is being treated with gene therapy, developing and validating novel endpoints, and improving the experience of patients who participate in clinical trials.
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From page 70... ...
This would require shifting the paradigm of demonstrating safety of a therapy in adults first, he said, but the treatment for certain conditions may be profoundly effective in younger patients. Learning from the Past and Scaling Up Another approach to improving gene therapy clinical trials, Cho said, involves developing the infrastructure needed to scale gene therapies so that they can be appropriately integrated into clinical practice.
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From page 71... ...
Finkel added that physician education needs to include ways to frame patient expectations for these treatments so as to avoid disappointment. Ideas for Developing Clinical Endpoints in Different Ways Panelists were asked by an audience member if they saw opportunities for groups to work together in the precompetitive space to develop new endpoints for gene-based therapy clinical trials.
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From page 72... ...
Examples were provided by speakers, including one that centered on developing a novel, clinically meaningful endpoint for assessing visual improvement in patients with retinal disorders. The workshop heard that patient registries can be valuable tools for developing clinical endpoints and that educational materials tailored to patients might be important to include in a given trial, Witten said.
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From page 73... ...
Summary points from the fourth session are provided in Box 6-4. Over the years, the regulatory process has evolved with regard to gene therapies, Siegel said, and FDA is much more flexible regarding novel designs and approaches to clinical trials and the use of registries and surrogate endpoints, all while trying to maintain high scientific standards and make all of this feasible.
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From page 74... ...
(Samuels) • Earlier treatment with gene therapies often results in better patient outcomes, and newborn screening can help identify infants with rare, serious conditions.
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From page 75... ...
• Investigators or researchers who want a discussion with the Food and Drug Administration about the acceptability of their endpoint in a disease can con sider participating in the agency's drug development tools program. (Lapteva)
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From page 76... ...
• Retrospective follow-up presents several challenges, including the loss of contact with patients and patients' refusal to sign medical release documents; prospective approaches are preferable. (Robison)
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