Remodeling an existing rare disease registry to be used in regulatory context: Lessons learned and recommendations

• Main Authors: Marina Mordenti (Author), Manila Boarini (Author), Fabio D'Alessandro (Author), Elena Pedrini (Author), Manuela Locatelli (Author), Luca Sangiorgi (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Disease registries have been used as an interesting source of real-world data for supporting regulatory decision-making. In fact, drug studies based on registries cover pre-approval investigation, registry randomized clinical trials, and post-authorization studies. This opportunity has been investig...