Laws and Policy Applicable to Stem Cell Products

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Laws and Policy Applicable to Stem Cell Products

• Public Health Service Act
• Food, Drug, and Cosmetic Act
• Human Somatic Cell Therapy Products and Gene
  Therapy Products (October 1993)
• Proposed Approach to Regulation of Cellular and
  Tissue-Based Products (1997)

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A New Class of Product Requiring INDs

• 1993 Somatic and Gene Therapies Federal Register
  Statement
• Somatic cell therapy is the prevention,
  treatment, cure, diagnosis, or mitigation of
  disease or injuries in humans by the
  administration of autologous, allogeneic, or
  xenogeneic cells
• Manufacture of products for somatic cell therapy
  involves the ex vivo propagation, expansion,or
  other alteration of their biological
  characteristics.

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Proposed Approach to Regulation of Cellular and
Tissue-Based Products

• Docket 97N-0068, February 28, 1997
• Outlines a risk-based system of regulation for a
  variety of cellular and tissue-based products
  including stem cells
• Considers five overarching public health and
  regulatory concerns

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Five Public Health and Regulatory Concerns

• Prevention of transmission of communicable
  diseases
• Processing controls to prevent contamination of
  cells and tissues and to preserve their integrity
  and function for safe and effective use
• Clinical safety and effectiveness
• Necessary product labeling for proper product use
• Monitor and communicate with the cell and tissue
  industry

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Implementation

• Provide public documents for the proposed
  regulatory framework
• Hold public meetings and workshops
• Notice and comment rulemaking for new rules such
  as for registration and listing, donor
  suitability requirements, and GTPs
• Phase in of new requirements over next two to
  three years

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Hematopoietic Stem Cell Standards Development

• FDA intends to promulgate establishment and
  processing controls and product standards for
  hematopoietic stem cells.
• FDA has invited the public to submit data and
  standards to the agency that would ensure safety
  and effectiveness of unrelated allogeneic stem
  cell products. Fed Register 632985-2988, Jan
  20, 1998

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If sufficient data are not available to develop
standards,
-after a specified period of time, unrelated
allogeneic hematopoietic stem cell products would
be subject to IND and marketing application
requirements.
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If FDA determines that data are available to
support the development of standards,

• FDA intends to announce such standards.
• Licensure may be granted for products certified
  as meeting promulgated standards.

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Public Workshops

• December 13, 1995 Cord Blood Banking
• February 22-23, 1996 Peripheral Blood
  Stem/Progenitor Cells
• March 17, 1997 Proposed Approach to Regulation
  of Cellular and Tissue-Based Products
• September 22-23, 1997 Ethical Issues in Cord
  Blood Banking

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Public Workshops (continued)

• September 10, 1998 Hematopoietic Stem Cell
  Products Discussion of Unrelated Allogeneic
  Placental/Umbilical Cord Blood and Peripheral
  Blood Banking and Transplantation

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Public Outreach

• Workshops
• Advisory committees
• RAC presentations
• Rule and comment process
• Posting of documents and transcripts on CBER, FDA
  web site
• Presentations at symposia, conferences