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LET’S GET REAL: Harnessing Non-Proprietary Patient Registries and RWE to Accelerate Rare Disease Drug Development
September 8 @ 10:00 am - 12:00 pm
Hosted by Penn Medicine Orphan Disease Center Amicus Therapeutics
Spread over four weekly 2-hour long sessions in September 2020, co-hosted by the Orphan Disease Center at the University of Pennsylvania and Amicus Therapeutics. 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, assessing data integrity, and 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.