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Regulation of Unrelated Cord Blood Banking Meeting the Needs of the U.S. Public

Regulation of Unrelated Cord Blood Banking Meeting the Needs of the U.S. Public. John P. Miller, MD PhD Senior Medical Director, NMDP March 30, 2007. Regulatory Issues in Unrelated Cord Blood Banking.

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Regulation of Unrelated Cord Blood Banking Meeting the Needs of the U.S. Public

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  1. Regulation of Unrelated Cord Blood Banking Meeting the Needs of the U.S. Public John P. Miller, MD PhD Senior Medical Director, NMDP March 30, 2007

  2. Regulatory Issues in Unrelated Cord Blood Banking • Existing products (prior to licensure, aka “retro” units) need to be available for transplantation • Indications for cord blood transplantation should be broadened to include non-malignant conditions • Importation of cord blood units (CBUs) is essential to meet the needs of US transplant patients; the regulatory framework needs to allow continued importation of these products

  3. Older CBUs: Today vs Unlicensed Units in the Post-licensure Era • Represent a large proportion of the current inventory and units used for transplantation • Given the size of the inventory and need for high degrees of HLA matching, these units will continue to be needed even with increased collection and banking of CBUs • Documentation of retrospective,“equivalent GMP” will be difficult or not possible for cord banks; may these units be distributed under a “perpetual” IND? • NMDP data indicates that older units have similar clinical outcomes (survival and engraftment)

  4. CBU Inventory by Collection Date 92% of CBUs for transplant 80% collected before 5/25/05 Before5/25/05 Before5/25/05 Shipped CBUs Total Inventory N=62,021 N=1,383

  5. Before 5/25/2005 (n = 267) From 5/25/2005 (n = 69) Log-rank p-value = 0.70 Survival after Primary Cord Blood Transplants(February 2000 – December 2005)

  6. Transplantation for Non-malignant disorders • Transplants of CBUs for non-malignant disorders represent 27% of total transplants from Feb 2000 to Dec 2005 • Are these units available for “off label” use by transplant physicians, and if so… • Is the cord blood bank responsible for how the TC MD uses the product after it is shipped? • As the indications for specific non-malignant disorders are rare, how would we move from IND to licensure? • NMDP data suggests similar outcomes (engraftment and survival) for transplants for malignant and non-malignant hematologic disorders

  7. Non-malignant diseases (n = 89) Hematologic malignancies (n = 245) Log-rank p-value = 0.13 Survival after Primary Cord Blood Transplants(February 2000 – December 2005)

  8. Importation of CBU • Imported CBUs are needed to meet the needs of US patients for HLA matched units, current domestic CBU inventory is not adequate • Imported CBUs represent a significant proportion of the units for transplantation, but many international banks only ship a few units and may not apply for licensure • Are CBUs importable under a “perpetual” IND or other mechanisms?

  9. Importation of CBUs: CY 2006 U.S. Total NMDP N = 891 N = 468 Domestic Domestic 19% 14% Import Import

  10. Summary • Existing cord blood units (prior to licensure, aka “retro” units) need to be available for transplantation, yet retrospective documentation of GMP necessary for licensure is unlikely • Indications for cord blood transplantation should be broadened to include non-malignant conditions • Importation of cord blood units (CBUs) is essential to meet the needs of US transplant patients; the regulatory framework needs to allow continued importation of these products

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