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

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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
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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

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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)

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CBU Inventory by Collection Date
92 of CBUs for transplant
80 collected before 5/25/05
Before 5/25/05
Before 5/25/05
Shipped CBUs
Total Inventory
N62,021
N1,383
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Survival after Primary Cord Blood
Transplants(February 2000 December 2005)
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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

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Survival after Primary Cord Blood
Transplants(February 2000 December 2005)
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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?

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