Office of Biotechnology Products
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Office of Biotechnology Products Steven Kozlowski, Director, OBP Edward Max, Assoc. Dir. Research, OBP Site Visit Report Cellular, Tissue, and Gene Therapies Advisory Committee Meeting April 17, 2013
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Office of Biotechnology Products. Site Visit Report Cellular, Tissue, and Gene Therapies Advisory Committee Meeting April 17, 2013. Steven Kozlowski, Director, OBP Edward Max, Assoc. Dir. Research, OBP. Office of Biotechnology Products (OBP) Steven Kozlowski, MD, Director - PowerPoint PPT Presentation
Transcript of Office of Biotechnology Products
Office of Biotechnology Products
Steven Kozlowski, Director, OBPEdward Max, Assoc. Dir. Research, OBP
Site Visit Report Cellular, Tissue, and Gene Therapies
Advisory Committee Meeting April 17, 2013
Office of Biotechnology Products (OBP) Steven Kozlowski, MD, Director
Edward Max, MD,PhD, Assoc Dir Research
Division of Monoclonal Antibodies (DMA)
Division of Therapeutic Proteins (DTP)
Amy Rosenberg, PhD, DirectorGibbes Johnson PhD, Deputy
Lab of Molecular and Developmental Immunology
Lab of Cell Biology
Lab of Immunology
Lab of ChemistrySerge Beaucage, PhD, Chief
Lab of Molecular Oncology
Kula Jha, PhD
Jacek Cieslak, PhDSerge Beaucage, PhD
Gibbes Johnson PhD
Office of Biotechnology ProductsSteven Kozlowski, MD, Director
Edward Max, MD, PhD, Assoc Dir for Research
Division of Monoclonal Antibodies
Kathleen Clouse, PhD, Director
Division of Therapeutic Proteins
Amy Rosenberg, MD, Director
Monoclonal Antibodies
mAb FragmentsFab, sFv, diabodies
mAb ConjugatesRadionuclide, drug, toxin
Fc-Fusion Proteins
Enzymes
Cytokines
Growth Factors
Toxins
Rituxan
ReoPro
Bexxar
Enbrel
Fabrazyme
Avonex
Epo
Botox
OBP PRODUCTSHAVE MANY CLINICAL
INDICATIONS
OBP Product Approvals/Year
Linear fit
App
rova
ls/
Yea
r
Approvals/Yr
0
2
4
6
8
10
1980 1985 1990 1995 2000 2005 2010
5 Year Running Avg.
Yervoy ipilimumabNulojix belatacept
Challenges in OBP Product Review• Variety of Cell Substrates• microbiology (virus, prion), host cell molecules• Heterogeneity• glycosylation, oxidation, PEGylation• Higher order structure : structure/function
• receptor interaction and signaling• Manufacturing Issues• scale-up, solubility, aggregation• Mechanism of Action: sometimes unclear• design of bioactivity/potency assays• Immunogenicity: • safety and efficacy Issues• Comparability: manufacturing changes• Counter Bioterrorism: Animal Rule, stockpiles
Challenges in OBP Product Review• Variety of Cell Substrates• microbiology (virus, prion), host cell molecules• Heterogeneity• glycosylation, oxidation, PEGylation• Higher order structure : structure/function
• receptor interaction and signaling• Manufacturing Issues• scale-up, solubility, aggregation• Mechanism of Action: sometimes unclear• design of bioactivity/potency assays• Immunogenicity: • safety and efficacy Issues• Comparability: manufacturing changes• Counter Bioterrorism: Animal Rule, stockpiles
BIOSIM
ILARS
OBP Research• Protein structure/function• Biotechnology manufacturing science• Mechanism of action
– Bioassays, Biomarkers, etc.• Immunogenicity• Adventitious Agents
RESEARCH
REVIEW ®ULATION
Researcher/Reviewer Model
Advantages for all reviewersAdvantages for all reviewers
•Scientific knowledge to evaluate:Scientific knowledge to evaluate:– Mechanisms of actionMechanisms of action– Potency assaysPotency assays– Adverse eventsAdverse events– Drug-drug interactionsDrug-drug interactions
•Hands-on technical experienceHands-on technical experience– State-of-the-Art scienceState-of-the-Art science– Product developmentProduct development– Analytical techniquesAnalytical techniques– Manufacturing methodsManufacturing methods
Definitions of Research Personnel
Temporary Employee
Permanent Employee
Independent Investigator
Tenure-trackPrincipal Investigator
“Converted”Principal Investigator= Senior Investigator
Dependent Investigator
Staff Fellow Staff Scientist
Research Career Paths
Site-Visit Team: Evaluate individual PIs and Service Fellows in a Laboratory Unit
• RESEARCH accomplishments since last review cycle• RESEARCH proposals for next four years
– Novelty and originality– Mission Relevance– Independence of the candidate– Productivity relative to time for research and research support
available• Administrative/Management comments welcome• Regulatory activities and regulatory work quality NOT
assessed by Site Visit Team
Thank you!
To the Site Visit reviewers for your
time, expertise, and suggestionstime, expertise, and suggestions
to improve OBP’s research programs
Your input is critical to fulfilling our regulatory mission!
