From: Tull, Lori
Sent: Tuesday, December 13, 2011 2:09 PM
To: 'Donna Regan'
Subject: Clinical information requests for BLA
The clinical review team has the following requests for information:
1.. On the Form 356h that you submit, please specify the indication of your product. If you are limiting your indication to the following: "for use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.", you may make reference to the docket 1997N-0497 for the integrated summary of efficacy and the supporting efficacy data (the actual data need not be submitted by you). If the indication in your label goes beyond above, you will also need to submit evidence of efficacy for each additional diagnosis.
2.. Please clarify if the maternal testing sites are CLIA certified.
3.. Please submit a clinical section. Please submit a clinical summary, an integrated summary of safety and a benefit-risk analysis for the product, or identify where in the application these are located.
1.. Please include the overview of safety evaluation plan used to generate the data,
2.. Please provide the following raw safety dataset for review: degree of HLA mismatch (worst degree for multiple unit transplants), and age of cord blood unit. The dataset (including ones that you have submitted) should be accompanied by a dataset dictionary and a data dictionary.
3.. Please provide all the derived variables that you used to construct the tables and figures related to patient outcomes and product safety (e.g., the censoring variable for death). Please also specify how these variables were derived.
4.. Please provide written procedures for elicitation and handling of
1.. post donation information and
2.. recipient adverse events.
Lori A. Tull, RAC
Regulatory Project Manager
Office of Cellular, Tissue, and Gene Therapies
Center for Biologics Evaluation and Research
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