In partnership with Amicus Therapeutics, the Orphan Disease Center invites you to a month-long websinar series!
Spread over four weekly sessions in September 2020, the series will review the role of patient registries and high quality natural history data in advancing clinical development of rare disease therapeutics, with a focus on potentially curative technologies such as gene therapy and genome editing.
While genomic technologies may raise some specific issues (particularly related to long term follow-up), the key technical issues involved in creating regulatory-quality natural history databases are likely to be similar for all therapeutic platforms in terms of the scope of information, challenges around consistent data collection and curation, and assessing data integrity.
Because of the small numbers of patients typically involved in pivotal rare disease trials, often across multiple international trial sites, the series will also address challenges related to regulatory harmonization for patient registries and regulatory applications across international jurisdictions.
Session 1 - September 8, 2020
BEYOND THE TOWER OF BABEL: FDA LEADERSHIP ON DATA STANDARDS
Session 2 - September 15, 2020
LET’S STOP REINVENTING THE WHEEL: SCALING BEST PRACTICES FOR PATIENT REGISTRIES
Session 3 - September 22, 2020
LET’S GET REAL (WORLD EVIDENCE): APPLICATIONS FROM THE ONCOLOGY COMMUNITY
Session 4 - September 29, 2020
FROM BROOKLYN TO B ING: GLOBAL RARE DISEASE REGISTRIES AS GLOBAL LEARNING PL ATFORMS
