Cellular Products for Cardiac Diseases Dwaine Rieves, MD Center for Biologics Evaluation and Research FDA Perspective on:

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Presentation transcript:

Cellular Products for Cardiac Diseases Dwaine Rieves, MD Center for Biologics Evaluation and Research FDA Perspective on:

Cellular Products Overview Topics Purpose Regulatory background Questions

Cellular Products Purpose A scientific discussion of – Manufacturing – Preclinical testing – Pilot clinical studies

Cellular Products Purpose Cellular Products – Blood or bone marrow-derived cells – Skeletal muscle-derived cells – Predominantly autologous

Cellular Products Purpose Early Stage of Evolving Field – Little precedent – Limited data – Hypothetical considerations

Cellular Products Purpose A Scientific Focus – All insights tentative – No definitive assessment of data – No specific product assessment

Cellular Products Regulatory A Scientific Focus ResearchRegulatory Cellular Products

Cellular Products Regulatory Regulatory Mission Promote & Protect Public Health by: – Pre-marketing product development – Post-marketing product monitoring

Cellular Products Regulatory Cellular Products for Cardiac Diseases Biologic/drug/device regulations IND Early clinical development: – Safety and bioactivity

Cellular Products Regulatory Safety Concerns Manufacturing Sufficient preclinical testing Sufficient clinical study design

Cellular Products Regulatory Manufacturing--Product Safety Cell source & reagents Procedures – aseptic collection, processing, cell selection, storage, tracking & labeling Testing – sterility, endotoxin, viability, enumeration

Cellular Products Regulatory Manufacturing—Product Characterization Identification of cell populations Purity (non-cellular) Potency

Cellular Products Regulatory Pre-clinical Testing Evolving paradigm Generally c/w biological products Catheters – novel use of marketed – non-marketed

Cellular Products Regulatory Pre-clinical Testing Relevant species Disease model Administration (catheter)

Cellular Products Regulatory Clinical Study Design Adverse event detection – evaluation plan duration, frequency, extent – safety monitoring sequential enrollment, stopping rules

Cellular Products Regulatory Clinical Study Design Adverse event analysis— --Txt-related vs natural history – randomization – controls (placebo vs other) – masking – other?

Cellular Products Summary Topics Purpose Regulatory background Questions

Cellular Products Questions Questions Manufacturing – extent of testing & characterization Pre-clinical and catheter – extent and nature of testing Clinical study – adverse event detection & analysis

Cellular Products Regulatory References ( 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)