# Presentation: 836643

## Update on the Somatic Cell Therapy Letter

- Cellular, Tissue, and Gene Therapies Advisory Committee Meeting
- April 11, 2008

## Somatic Cell Therapy Letter (SCTL)

- Letter issued as of June 2002
- Requested submission of information on
- product manufacturing
- quality control procedures
- product testing
- clinical trial oversight and monitoring practices

## Goals of SCTL

- Ensure ongoing trials meet current expectations for
- Product release testing & characterization
- Identify lapses in product testing
- Product characterization & manufacturing processes
- Clinical trial oversight & monitoring
- Encourage Good Clinical Practice

## Goals of SCTL (cont.)

- Gather information on need for additional guidance other regulatory documents
- Determine need for other forms of outreach to SCT field
- Enhance the safety of cellular products
- Facilitate product development

## Somatic Cell Therapy Letter

- Consisted of 8 multi-part Chemistry, Manufacturing, and Control (CMC) questions and 3 multi-part Clinical questions
- Requested date for response was 60 days from anniversary date of the file
- Lack of response was not considered a cause for placing the file on clinical hold

## CMC Question Topics

- QC/QA program-
- description, procedures, personnel, audits
- Qualification of starting cells, reagents, equipment
- Tracking, segregation, labeling
- Cleaning and sanitization
- Control of contamination & cross contamination

## CMC Question Topics (cont.)

- Processing and testing timeline
- Product testing methods
- In-process, final product
- Sterility validation, aseptic processing
- Product characterization
- Stability program
- Cross referenced files

## Clinical Question Topics

- A complete description of the clinical monitoring program including:
- A description of the personnel responsible for monitoring
- A summary of the procedures for clinical study conduct monitoring and auditing
- A request final study reports be submitted for all studies

## Review Summary

-  Responses reviewed by IND review team
- ensure ongoing clinical trials met current expectations
- identify lapses in product safety testing
- Identified safety issues in low number of INDs
- Safety issues in a specific IND were addressed between review team and sponsor
- Observed broad differences in status of product characterization
- Identified need for additional CMC guidance documents and outreach to SCT field

## CMC- Published Guidance

## CMC- Published Draft Guidance

## GMP Guidances with Sections on SCT products

## Clinical Guidances

- Good Clinical Practice is addressed in
- 	“ICH E6
- 	Good Clinical Practice: Consolidated Guidance”

## CMC Guidance in Development

- Potency Measurements for Cell and Gene Therapy Products
- Topic of CTGTAC meeting on February 9, 2006
- Guidance document is under development

## Outreach

- OCTGT gives regulatory presentations at numerous conferences each year
-  broad audiences across SCT field
- Liaison Meetings
- CMC talks commonly focus on product characterization and potency
- CMC talks also address 21 CFR 1271, (Tissue Rules)

## HCT/P Regulations and Guidance

- 21 CFR 1271, “Tissue Rules”
- In effect in May 2005
- Focus on the prevention of transmission of infectious disease
- Parts A-D apply to SCT products

## Resources on the Web

- Consolidation of relevant information onto CBER website
- References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies (OCTGT)
- www.fda.gov/cber/genadmin/octgtprocess.htm

## Summary

- CBER experience from SCTL responses contributed to:
- multiple guidance documents
-  issued and in preparation
- numerous ongoing outreach activities broadly applicable to the field of cell therapies

## Conclusions

- Guidances are now available to address our expectations for IND submissions and clinical trial conduct
- It is not necessary to convey these expectations and request responses in a separate format
- OCTGT will discontinue issuing the SCTL and no longer request that sponsors provide updates in the format of the SCTL questions
- IND Sponsors may provide information updates in amendments and annual reports, as appropriate

## Thank you

- Somatic cell therapy IND sponsors
- CTGTAC members