2023 - Rare Disease Trials and Gene Therapy: Understanding the Patient Journey

Gene therapies continue to represent an exciting treatment paradigm for patients. With an increase in regulatory approvals in the past few years, there is an expectation for this trend to continue. For rare disease patients, gene therapy trials provide a very viable path toward developing transformational therapies that could have substantial impact on halting disease progression. With any new and emerging therapy, there are specific obstacles that must be considered, especially as they relate to rare disease trials. Some of these obstacles include, but are not limited to, accessing global patient populations, site training and activation, manufacturing and regulatory considerations and minimal industry standardization of data platforms.
Understanding patient challenges and needs in rare disease trials and incorporating their feedback into protocol design is critical to advancing these therapies as part of a complex patient journey.
Join this webinar to gain insights into the challenges of accessing global patient populations, manufacturing, regulations and patient-centered protocol design in rare disease trials.