From: Tull, Lori
Sent: Friday, May 24, 2013 11:18 AM
To: Tull, Lori
Subject: FW: SAFETY REPORTING related to your BLA 125413
From: Xu, Lei (CBER) [mailto:Lei.Xu2@fda.hhs.gov]
Sent: Wednesday, May 16, 2012 9:30 AM
To: Donna Regan
Subject: SAFETY REPORTING related to your BLA 125413
When you make changes on your safety report SOPP, please make sure the following parts are included.
ADVERSE EVENT REPORTING
You must submit adverse experience reports in accordance with the adverse experience reporting
requirements for licensed biological products (21 CFR 600.80) and you must submit distribution reports
as described in 21 CFR 600.81. You should submit postmarketing adverse experience reports and
distribution reports to the Center for Biologics Evaluation and Research, Office of Biostatistics and
Epidemiology HFM-210, Food and Drug Administration, 1401 Rockville Pike, Suite 200N, Rockville,
MD 20852-1448. Prominently identify all adverse experience reports as described in 21 CFR 600.80. Per
21 CFR 600.2(f), please refer to http://www.fda.gov/AboutFDA/CentersOffices/CBER/ucm106001.htm
for updated mailing address information.
In addition, you must submit adverse event reports for any infectious disease transmission within 15 days
after learning of the event. Infectious disease transmission refers to an adverse event that involves
suspected or confirmed transmission of an infectious agent, whether the recipient develops the infectious
disease or only has serologic or other evidence.
FDA regulations require Quarterly Periodic Adverse Experience Reports (PAERS) to contain a narrative
summary and analysis of the information in the report and an analysis of the 15-day Alert reports
submitted during the reporting interval (21 CFR 600.80(c)2). The narrative summary in PAERs for
hematopoietic progenitor cells, cord blood should include a detailed summary and assessment of all
serious infusion reactions observed during the reporting period, as well as your assessment of each case
and the overall frequency of serious infusion reactions since approval and during the reporting period.
PAERs should also include any adverse event (e.g., infusion reaction or other adverse event) information
forwarded to you from the Stem Cell Therapeutics Outcomes Database (SCTOD) during the reporting
period. PAERs should also include the number of units released for infusion and the number of patients
receiving infusions with hematopoietic progenitor cells, cord blood during the reporting period.
In addition, you have to agree to do the following:
1. Implement a safety outcomes monitoring and analysis plan. This plan will include a) maintenance
of an observational database to include, for all hematopoietic progenitor cell, cord blood units
released, information including but not limited to, time to neutrophil recovery, graft failure,
survival, cause of death, infusion reactions, and other adverse experiences, and b) aggregate
analyses of interval and cumulative adverse experience reports, and c) safety outcomes analyses
of interval and cumulative data that address early mortality, graft failure-related mortality, graft
failure, time to neutrophil recovery, infusion-related events, and other adverse
experiences. Reports will include a description of the population analyzed, results of the analyses,
whether outcomes indicators were triggered and, if so, what actions were implemented as a result.
2. Submit a 15-day ?alert report? for each serious infusion reaction associated with administration of
hematopoietic progenitor cells, cord blood.