Académique Documents
Professionnel Documents
Culture Documents
www.fda.gov 2
How a Regulatory Agency
Comes into Existence
13 children in St. Louis, MO die of tetanus after
1901 receiving contaminated diphtheria antitoxin and
9 children in Camden, NJ die after receiving
contaminated smallpox vaccine
www.fda.gov 4
CBER’s Regulatory Portfolio
Live
Biotherapeutic
Blood Related Products
Derivatives Devices FMT
Phage
Blood, Blood
Components
Vaccines:
Gene Tissues Preventive
Therapies &
Therapeutic
Xenotrans-
Cell Therapies plantation Allergenic
Products Products
www.fda.gov 5
Regulatory Framework for Biologics
• Constitution
• Laws/Statutes
• Public Health Service Act
• Section 351
• Section 361
• Federal Food Drug and Cosmetic Act
• Regulations/Rules
• Guidance
www.fda.gov 6
Vaccine Confidence
• Preventative vaccines have unique benefit-risk
considerations when compared to other
medical products
– Low tolerance for adverse effects
• Need to deal with the challenge of coincidence:
conditions that emerge coincident with, but
independent of vaccine administration
www.fda.gov 7
FDA and Vaccines
• Ensure adequate availability of vaccines that are
safe, pure, potent, and effective
– Conduct relevant applied scientific research
– Provide advice on vaccine development
– Evaluate submissions for licensure
– Develop and distribute reagents for vaccine production
– Lot release testing
– Post-marketing surveillance
www.fda.gov 8
Example of Vaccine Research
There has been a resurgence in
whooping cough caused by B.
pertussis
CBER scientists developed a non-human primate
model for this disease that could facilitate further
vaccine development
– Warfel JM et al. Infect Immun. 2012; 80: 1530–1536.
www.fda.gov 9
Baboon Model Suggests
Mechanism of Vaccine Failure
• Acellular pertussis vaccine (current) compared to whole cell pertussis
vaccine (older generation prior to 1980’s)
• Both vaccines induced robust antibody responses, but T cell responses
were significantly different
A 10 9
Naive
10 8
Acellular vaccine protected aP
10 4
pertussis, but failed to prevent 10 3
Day post-challenge
www.fda.gov 10
Expedited Development Programs
• Fast Track
• Priority Review
• Accelerated Approval
• Breakthrough Therapy
www.fda.gov
The product is the process 16
Vision for Manufacturing
• There will be national capacity to produce
ample quantities of high quality vaccines in a
timely and cost effective manner to address
existing and emerging infectious diseases
www.fda.gov 17
Vaccine Manufacturing Challenges
• Vaccine manufacture is a time consuming
process with constraints on agility and capacity
that limit response to emerging pathogens
including seasonal and pandemic influenza
– Working to improve yield from cell-based and
recombinant production processes
– Considering potential of advanced manufacturing
www.fda.gov 18
CATT Meetings
CBER Advanced Technology Team
• Provides an interactive mechanism to prospective
developers of novel therapies to discuss the
implementation of the needed technologies in the
development of CBER-regulated biologics products
• Allows access to early interactions on more general
topics before filing of a regulatory submission
www.fda.gov 19