Title: Cellular Products for Cardiac Diseases Dwaine Rieves, MD Center for Biologics Evaluation and Research
1Cellular Products for Cardiac DiseasesDwaine
Rieves, MDCenter for Biologics Evaluation and
Research
FDA Perspective on
2Cellular Products Overview
- Topics
- Purpose
- Regulatory background
- Questions
3Cellular Products Purpose
- A scientific discussion of
- Manufacturing
- Preclinical testing
- Pilot clinical studies
4Cellular Products Purpose
- Cellular Products
- Blood or bone marrow-derived cells
- Skeletal muscle-derived cells
- Predominantly autologous
5Cellular Products Purpose
- Early Stage of Evolving Field
- Little precedent
- Limited data
- Hypothetical considerations
6Cellular Products Purpose
- A Scientific Focus
- All insights tentative
- No definitive assessment of data
- No specific product assessment
7Cellular Products Regulatory
Research
Regulatory
Cellular Products
8Cellular Products Regulatory
- Regulatory Mission
- Promote Protect Public Health by
- Pre-marketing product development
- Post-marketing product monitoring
9Cellular Products Regulatory
- Cellular Products
- for Cardiac Diseases
- Biologic/drug/device regulations
- IND
- Early clinical development
- Safety and bioactivity
10Cellular Products Regulatory
- Safety Concerns
- Manufacturing
- Sufficient preclinical testing
- Sufficient clinical study design
11Cellular Products Regulatory
- Manufacturing--Product Safety
- Cell source reagents
- Procedures
- aseptic collection, processing, cell selection,
- storage, tracking labeling
- Testing
- sterility, endotoxin, viability, enumeration
12Cellular Products Regulatory
- ManufacturingProduct Characterization
- Identification of cell populations
- Purity (non-cellular)
- Potency
13Cellular Products Regulatory
- Pre-clinical Testing
- Evolving paradigm
- Generally c/w biological products
- Catheters
- novel use of marketed
- non-marketed
14Cellular Products Regulatory
- Pre-clinical Testing
- Relevant species
- Disease model
- Administration (catheter)
15Cellular Products Regulatory
- Clinical Study Design
- Adverse event detection
- evaluation plan
- duration, frequency, extent
- safety monitoring
- sequential enrollment, stopping rules
16Cellular Products Regulatory
- Clinical Study Design
- Adverse event analysis
- --Txt-related vs natural history
- randomization
- controls (placebo vs other)
- masking
- other?
17Cellular Products Summary
- Topics
- Purpose
- Regulatory background
- Questions
18Cellular Products Questions
- Questions
- Manufacturing
- extent of testing characterization
- Pre-clinical and catheter
- extent and nature of testing
- Clinical study
- adverse event detection analysis
19Cellular Products Regulatory
- References (www.FDA.gov/cber)
- Draft Guidance for CMC Reviewers Human Somatic
Cell Therapy Investigational New Drug
Applications - ICH Guideline Preclinical Safety Evaluation of
Biotechnology-derived pharmaceuticals (S6) - ICH Guideline Good Clinical Practice (E6)