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Update on OCTGT Guidance Development Program

Update on OCTGT Guidance Development Program . Cellular, Tissue, and Gene Therapies Advisory Committee Meeting April 11, 2008. FDA Good Guidance Practices (GGP). Ensures proper development, formatting, processing, routing, and use of FDA guidance documents

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Update on OCTGT Guidance Development Program

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  1. Update on OCTGT Guidance Development Program Cellular, Tissue, and Gene Therapies Advisory Committee Meeting April 11, 2008

  2. FDA Good Guidance Practices(GGP) • Ensures proper development, formatting, processing, routing, and use of FDA guidance documents • Mandated by “Food and Drug Administration Modernization Act of 1997" (FDAMA) • Administrative Practices and Procedures; Good Guidance Practices,” 21 CFR 10.115 • CBER SOPP 8002 • Annual Guidance Agenda published in Federal Register

  3. Guidance Documents • Documents prepared for FDA staff, applicants/sponsors, and the public that: • Describe the agency’s interpretation of or policy on a regulatory issue; • Relate to the processing, content, and evaluation/approval of submissions; • Relate to the design, production, manufacturing, testing, labeling, and promotion of regulated products;

  4. Other Documents • Guidance documents do NOT include documents related to: • Internal FDA procedures; • Agency reports; • General information provided to consumers or health professionals; • Speeches; • Journal articles and editorials; • Media interviews and press materials; • Warning letters; • Memoranda of understanding; • Other communications or actions taken by individuals at FDA or directed to individual persons or firms; • Documents directed at public health emergencies

  5. Guidance Documents • Guidance documents are intended to represent our current thinking on matters discussed in the documents. • CBER staff may depart from guidance documents only with appropriate justification and supervisory concurrence (21 CFR 10.115(d)(3)). • Any consistent deviation from a guidance document indicates that we should revise it

  6. Level 1 Guidance Documents • Federal Register announcing the availability of the draft guidance for public comment • Placed on CBER web site • FDA offers a comment period (usually 60 to 90 days) • After reviewing the public comments, CBER will revise the guidance • Federal Register, announcing the availability of the final guidance. • The public may submit comments on the final guidance at any time. • CBER may issue a level 1 guidance for immediate implementation

  7. Level 2 Guidance Documents • All guidance documents not considered to be Level 1 • Level 2 documents set forth existing practices or minor changes in interpretation or policy

  8. Communication with the Public During Guidance Development • When considering the development of guidance • CBER may freely discuss issues with the public. • If the issues to be addressed are particularly complex or controversial • Holding a public meeting • Holding advisory committee meeting on the issues • Before drafting the guidance • Once preparation of a draft guidance document has been initiated • Specifics of the guidance are not discussed with the public. • When the guidance has been issued in draft • Issues related to the draft guidance may be discussed • Issues related to specific intentions regarding the finalization of the guidance are not discussed • 21 CFR 10.80 • FDA may solicit information from the public at any time during the guidance development process.

  9. Published Guidances in Current FDA Annual Guidance Agenda (71 FR 51225, September 1, 2006) • Umbilical Cord Blood Intended For Hematopoietic Reconstitution in Patients With Hematological Malignancies • Draft, 1/2007 • CTGTAC Discussions- 2/2003, 3/2007 • Preparation of Investigational Device Exemptions and Investigational New Drugs for Products Intended to Repair or Replace Knee Articular Cartilage • Draft 7/2007 • CTGTAC Discussions- 3/2005

  10. Published Guidances in Current FDA Annual Guidance Agenda (71 FR 51225, September 1, 2006) • Validation of Rapid Microbiological Methods for Assessing Sterility of Cellular and Gene Therapy Products • Draft 2/2008 • Certain Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Recovered From Donors Who Were Tested For Communicable Diseases Using Pooled Specimens or Diagnostic Tests • Final, 1/2007

  11. Additional Published Guidances 2006-2007 • Regulation of Human Cells, Tissues and Cellular and Tissue-Based Products (HCT/Ps)- Small EntityCompliance Guidance • Immediate Implementation, 8/2007 • Eligibility Determination for Donors of Human Cells, Tissues and Cellular and Tissue-Based Products • Final 8/2007 • Cell Selection Devices for Point of Care Production of Minimally Manipulated Autologous Peripheral BloodStem Cells (PBSCs) • Draft 7/2007 • Class II Special Controls Guidance Document: Cord Blood Processing System and Storage • Immediate Implementation, 1/2007

  12. Additional Published Guidances 2006-2007 • Gene Therapy Clinical Trails – Observing Subjects for Delayed Adverse Events • Final 11/2006 • CTGTAC Discussions- 3/1999, 11/2000, 4/2001, 10/2001 • Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors • Immediate Implementation, 11/2006 • CTGTAC Discussions- 3/1999, 11/2000, 4/2001, 10/2001

  13. Recently Finalized Guidances • Content And Review Of Chemistry, Manufacturing, And Control (CMC) Information For Human Somatic Cell Therapy Investigational New Drug Applications (INDs) • Final 4/2008 • CTGTAC Discussions- Topics included in discussion at numerous meetings • Content And Review Of Chemistry, Manufacturing, And Control (CMC) Information For Human Gene Therapy Investigational New Drug Applications (INDs) • Final 4/2008 • CTGTAC Discussions- Topics included in discussion at numerous meetings

  14. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage • Written by Cross-Center Team • CDRH • CBER • Reflects CTGTAC input • CDRH participated in meeting • March 2005 • Leverages ASTM document • Written by ASTM subcommittee • FDA staff participation according to FDA policies and ASTM process

  15. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Scope • IND/IDE products to repair or replace articular cartilage of the knee • Devices • Biologics • Combinations Products • Excludes • Unicondylar and total knee implants • Meniscus replacement products • “361” HCT/P products

  16. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Manufacturing and CMC Information • Brief explanation of requirements for • Devices • Biologics • Combination Products • Tailored for specifics of product composition

  17. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Nonclinical Data and Testing • Purposes of Animal Studies • Biological response to products • Durability of response (1 year) • Toxicology • Dose response • Mechanical Testing • Compare to normal cartilage • Justify why not possible • Biocompatibility • ISO 10993 and/or ASTM F748-04

  18. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Nonclinical Data and Testing • ASTM document- F2451-05, “Standard Guide for in vivo Assessment of Implantable Devices Intended to Repair or Regenerate Articular Cartilage,” • cartilage defect locations; • discussion of articular cartilage defect preparation; • description of gross and histological assessments; • description of various mechanical evaluations and their applicability.

  19. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Clinical Study Protocols • Exploratory Trials-design features • Exploratory Trials-knowledge learned

  20. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Clinical Study Protocols • Confirmatory Trials • Recommend randomized, controlled trial • Double blind • Blinded evaluators of major endpoints • Alternative design may be considered

  21. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Study Endpoints • Clinically meaningful endpoints as primary in confirmatory studies • Improvement in pain • Improvement in physical function

  22. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Study Endpoints • Examples of secondary endpoints • Arthroscopic examination • Physical examination • Histologic examination at short and long term • Serological assessment for antibody and evidence of inflammation • Synovial fluid examination

  23. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage • Investigational Product Administration • Detailed description of procedures • Surgical technique • Plans for post-operative • Follow-up • Length of follow-up to be based on in vivo and in vitro studies • Two-year follow-up for subset of subjects

  24. Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage Adverse event (AE) reporting • AE reporting per regulations (IND or IDE) • Subsequent surgical interventions • Revision • Removal • Reoperation

  25. Resources on the Web • Consolidation of OCTGT guidances • Cellular and Gene Therapies • http://www.fda.gov/cber/genetherapy/gtpubs.htm • Tissues • http://www.fda.gov/cber/tissue/docs.htm • References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies (OCTGT) • www.fda.gov/cber/genadmin/octgtprocess.htm

  26. Summary • FDA guidance process to communicate with stakeholders • Good Guidance Practices (GGP) • Governed by regulations 21 CFR 10.115 • OCTGT Recent Guidances • Current Annual Guidance Agenda • Additional Guidances to meet needs arising between Annual Guidance Agendas • FDA Guidance on Cartilage Repair Products • CBER and CDRH Joint Guidance • CTGTAC Meeting provided input for guidance development • Leveraged FDA participation in Standards Development Organization (ASTM)

  27. Conclusions • OCTGT has an active guidance development program that includes • Strategic Guidance Planning (Annual Agenda) • Responds to changing needs • OCTGT Guidance Program leverages • Input from CTGTAC meetings • Other public meetings (NIH RAC, Workshops, Scientific Meetings, Industry Liaison Meetings) • Cross-center efforts within FDA • Participation with Standards Development Organizations

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