FDA did not post any warning letters to Sponsors/Monitors/CROs, IRBs or Clinical Investigators this week. From the archives, here is the Clinical Inspection summary for Brineura (cerliponase alfa) submitted by Biomarin. Two clinical sites, the sponsor and the host entity of the natural history database used as a historical control were inspected. Site inspection took place over four days and two weeks. One site received a FDA Form 483.
Dr. Specchio was cited for failing to provide the informed consent to a patient in a language understandable to the patient.
FDA notes elsewhere in the "other review package" that the genotyping information obtained from the natural history database was not accurate for five subjects. FDA also raised concerns about the lack of standard procedures for entering data into the natural history database.
FDA noted in communication with the sponsor that the data quality of the submission impeded review. Statistical methods to compare natural history versus the clinical trial were questioned.
FDA determined that the initially submitted data was not robust enough and a major amendment was requested, adding nearly a year to the review cycle. FDA notes that 33 information requests were submitted to the sponsor during this process. Pages 47-57 here show the statistical information requests alone.