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FDA PROPOSED DISCLOSURE RULE

FDA PROPOSED DISCLOSURE RULE. Growth factors. SCF, Flt-3, IL-3, PEG-MDF. Viral vector. Fibronectin coated flasks. PBMC. CD34+ Selection. CD34+ transduction. CD34+ expressing gamma-c receptor. An Example of Complexity: Gene Therapy Products.

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FDA PROPOSED DISCLOSURE RULE

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  1. FDA PROPOSED DISCLOSURE RULE

  2. Growth factors SCF, Flt-3, IL-3, PEG-MDF Viral vector Fibronectin coated flasks PBMC CD34+ Selection CD34+ transduction CD34+ expressing gamma-c receptor An Example of Complexity: Gene Therapy Products Ex Vivo Transduced CD34+ Cells Expressing GammaC-R for X-SCID

  3. Gene Therapy and Xenotransplantation Products Come From Nature... • Natura enim non imperatur, nisi parendo: “Nature cannot be ordered about, except by obeying her” • Novum Organum (1620), Sir Francis Bacon • “…in nature there are no rewards or punishments; there are consequences.” • Some Reasons Why (1881), Robert Ingersoll • For every intended consequence, there are a hundred mostly unknown unintended consequences that we must address... • FDA reviewer’s lament

  4. Consequences From Biologics • Jenner, 1796 • Prevention of smallpox, but spread of syphilis • St. Louis, 1901 • Diphtheria antitoxin, children died with tetanus • Salk, 1950s • First successful killed vaccine, large scale manufacturing failure

  5. Consequences From Biologics • RSV Vaccine, 1960s • Killed vaccine, vaccinated died • U Penn, 1999 • 18 year old patient dies in experimental gene therapy trial • Rotavirus Vaccine, 2000 • Prevention of infantile diarrhea, but causes intussuception

  6. Proposed Rule on Public Disclosure • Availability for Public Disclosure and Submission to FDA for Public Disclosure of Certain Data and Information Related to Human Gene Therapy or Xenotransplantation • Published for comment 1/18/01 (66 FR 4688-4706; docket number 00N-0989)

  7. SCOPE and PURPORSE • Gene Therapy and Xenotransplantation • Unique areas of clinical research with potential for unique public health risks and modification of the human genome • Complexity of products • Provide consistent amount of information for public discussion and public access • Enhance patient awareness and consumer protection • Ensure accurate up to date informed consent • Allow FDA to fully participate in public discussions

  8. WHAT IS NOT DISCLOSABLE • Patient information • Trade secret information (must be productive process, not idea) • Limited amount of commercial confidential information, based on experience with RAC, DHHS Xeno Group and Xeno Subcommittee

  9. WHAT IS DISCLOSABLE, 601.52 • (1) Product and patient safety data and related information. • (2) The name and address of the sponsor. • (3) The clinical indications to be studied. • (4) A protocol for each planned study... • (5) Written informed consent form(s) as provided in 50.27 of this chapter.

  10. WHAT IS DISCLOSABLE, 601.52 • (6) Identification of the biological product(s)… • (7) IND safety reports, as provided in 312.32... • (8) Information submitted in the annual report... • (9) The regulatory status of the IND, such as on hold, in effect, inactive, or withdrawn... • (10) Other relevant data and information that the Director, CBER, determines are necessary for the appropriate consideration of the public health and scientific issues, including relevant ethical issues, raised by human gene therapy or xenotransplantation.

  11. HOW WILL THIS INFORMATION BE DISCLOSED? 601.53 • Official and redacted version of each submission to an IND • Redacted document to not contain patient information trade secrets, and certain limited confidential commercial information • FDA Public Docket for each IND • Redacted documents to specific docket through life of IND

  12. …lessons from the past enable the future:“What can be imagined, will be done” …with hope …humility …compassion

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