14 January 2010
Division of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, MD 20852
RE Docket No. FDA-2009D-0490, 20 October 2009, “Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications”, Draft Guidance.
Via electronic submission: http://www.regulations.gov/
Dear FDA Dockets Manager:
AABB is an international association dedicated to advancing transfusion and cellular therapies worldwide. Our members include more than 1,800 hospital and community blood centers and transfusion and transplantation services as well as approximately 8,000 individuals involved in activities related to transfusion, cellular therapies and transplantation medicine. For over 50 years, AABB has established voluntary standards for, and accredited institutions involved in, these activities. AABB is focused on improving health through the advancement of science and the practice of transfusion medicine and related biological therapies, developing and delivering programs and services to optimize patient and donor care and safety.
AABB appreciates this opportunity to comment on the “Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications” draft guidance document and is drawing on the expertise of its membership participation in Regulatory Affairs and Cord Blood Cellular Therapy Subsections. The HPC-C draft IND guidance is highly valued as it provides a regulatory pathway for lifesaving HPC-Cs to be transplanted when the products cannot be licensed as described in the guidance for industry “Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications” also published on 20 October 2009.
The comments submitted on behalf of AABB members are intended to provide clarity in the final document. The following format is used for the comments:
Section – language from draft guidance reprinted with identifying information.
Recommendation – recommendation with rationale.
Section – II. Background. This guidance document is applicable to certain unlicensed HPC-Cs that are made available for transplantation in cases when no satisfactory alternative treatments are available.
Recommendation – AABB recommends the term “satisfactory alternative treatments” be clarified when the recommendations are finalized. Inclusion of scenarios or examples would be informative.
Section – Table A: Minimal Information to be Included in the IND. Protocol. Provides a brief summary of the study including a description of the cord blood dosing plan…
Recommendation – AABB recommends that the guidance document, when finalized, describe or give an example of a protocol or study that would be appropriate for an IND held by a cord blood bank or registry for products to be used at many transplant centers for a variety of indications.
Section – IV. Responsibilities of sponsors and investigators.
Recommendations – AABB appreciates that the intent of this draft is to provide a pathway for continued use of HPC-C products in situations where the products cannot or are not likely to be licensable. It would be beneficial to provide a discussion of ways in which pre-licensure inventories might be shown to be comparable to the establishment’s licensed HPC-Cs as an encouragement to sponsors and investigators to obtain licensure where possible.
AABB strongly supports initiatives that improve the safety of patients and donors and stands ready to interact with FDA as necessary. Questions concerning these comments may be directed to M. Allene Carr-Greer, Director, Regulatory Affairs, email@example.com.
M. Allene Carr-Greer, MT(ASCP)SBB
Director, Regulatory Affairs