Transcript Document

Seminar on……
CENTRE FOR BIOLOGICS
EVALUATION &
RESEARCH
(CBER)
Presented by:
Dabhi Mahesh R.
Department Of Pharmaceutics & Pharmaceutical Technology,
L. M. College of Pharmacy.
List of contents…….
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Introduction
Responsibilities of CBER
Structure of CBER
What are biologics ?
Role of CBER
Products regulated by CBER
Biological product deviation reporting (BPDR)
Marketing approval procedure for biologics
Biologics license application (BLA)
Refusal to file
Adverse drug reporting
FDA recall policies
Consumer / Healthcare Information & CBER
Role of CBER in Countering Bioterrorism
References
Introduction
• The Center for Biologics Evaluation and
Research (CBER) regulates biological
products.
• Current authority for this responsibility
resides in Section 351 of the Public Health
Service Act and in specific sections of the
Food Drug and Cosmetic Act.
• CBER is committed to advancing the public
health through innovative regulations that
ensure• the safety
• effectiveness and
• timely delivery to patients of biological products.
• The mission of CBER is
• to protect and enhance the public health through the
regulation of biological and related products including
blood, vaccines, tissue, allergenics and biological
therapeutics.
Responsibilities of CBER
• The safety of this nation's entire blood supply and the
products derived from it;
• The production and approval of safe and effective childhood
vaccines, including any future AIDS vaccines;
• The proper oversight of human tissue for transplantation;
• An adequate and safe supply of allergenic materials and
antitoxins;
• The safety and efficacy of biological therapeutics, including
an exciting new array of biotechnology-derived products used
to treat diseases such as cancer and aids.
Structure of CBER
• Office of communication, training and
manufacturers assistance.
• Office of management.
• Office of blood research and review.
• Office of information technology.
Structure of CBER
• Office of cellular, tissue and gene therapy.
• Office of vaccine research and review.
• Office of biostatics and epidemiology.
• Office of compliance and biologic quality.
Office of communication,
training &
manufacturers assistance
Division of
manufacturers assistance
& training
Division of
Disclosure &
Oversight management
Division of
Communication &
Consumer affairs
Office of management
Division of
Scientific advisors
& consultants
Division of planning,
Evaluation and budget
Division of
Programme service
Office of
blood research and review
Division of Emerging &
Transfusion transmitted
disease
Division of
Blood applications
Division of
Hematology
Office of
Vaccine research and review
Division of Bacterial,
Parasitic &
Allergenic products
Division of
Viral products
Division of Vaccines &
Related product applications
Office of Cellular, Tissue &
Gene therapies
Division of Cellular &
Gene therapies
Division of Evaluation &
Toxicology
Division of
Human tissue
Office of
Compliance & Biologics quality
Division of
Case management
Division of
Inspections & Surveillance
Division of
Mfg. & Product quality
Office of
Biostatistics & Epidemiology
Division of
Epidemiology
Division of
Biostatistics
Office of
Information Technology
Division of
Information technology operations
Division of
Information technology development
What are biologics ?
• Biologics, in contrast to drugs that are chemically
synthesized, are derived from living sources– humans,
– animals, and
– Microorganisms
• Biological products often represent the cuttingedge of biomedical research and offers advantage
that– Useful for treatment of medical illnesses that
presently have no other treatments available.
Role of CBER
• For product approval CBER go through the
scientific and clinical data submitted by
the applicant• To determine whether the product meets CBER’s std.
or not.
• After a thorough assessment of the data,
CBER makes a decision based on• the risk-benefit for the intended population
• the product's intended use.
Role of CBER
• CBER is committed to a product
approval process that• maximizes the benefits and
• minimizes the risks to patients of the
biological product.
• Choice to use a biological product
involves balancing the benefits to be
gained with the potential risks.
Products regulated by CBER
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Blood
Devices
Vaccines
Gene therapy
Xenotransplantation products
Allergenics
Human Tissue & Cellular products
Blood
• CBER regulates the collection of blood and blood
components & establishes standards for the
products themselves
• CBER regulates– Related products such as-
• cell separation devices,
• blood collection containers
– HIV screening tests that are used to prepare blood
products
• In 1997, the FDA initiated the Blood Action Plan
to increase the effectiveness of its scientific and
regulatory actions
Blood Action Plan
• CBER initiated a Blood Action Plan in July
1997• To increase the effectiveness of its scientific and
regulatory actions
• To ensure greater coordination with PHS partners
• The Action Plan addresses highly focused
areas of concern such as
• emergency operations,
• response to emerging diseases, and
• updating of regulations.
Blood Action Plan
• Blood action plan includes following in detail•
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Updating Blood Regulations
Reinvention of Blood Regulation
Emerging Infectious Diseases
Insuring Compliance of Plasma Fractionation
Establishments
• Notification and Lookback
• FDA Response to Emergencies and Class I Recalls
Affecting Blood Safety
• Monitoring and Increasing the Blood Supply
Blood Establishment
Registration and Product
Listing (BER)
• Blood product mfr. are required to register
with FDA under section 510 of the Federal
Food, Drug, and Cosmetic Act, unless they
are exempt under 21 CFR 607.65.
• A list of all the products processed or
manufactured must also be submitted.
• Products must be registered and listed
within 5 days of beginning operation.
Devices
• Regulates the medical devices involved in the•
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collection,
processing,
testing,
manufacture and
administration
of licensed blood, blood components and
cellular products.
• Also regulates all HIV test kits used both• to screen donor blood, blood components and cellular
products, and
• to diagnose, treat and monitor persons with HIV and AIDS.
2006 Biological Device Application Approvals
Tradename
Description and Indication for Device
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Applicant
Approval Date
HLA Visual Software version
1.0
Accessory for the evaluation of test results
from One Lambda Inc products for
molecular typing and antibodies
detection
BK050070
One Lambda, Inc
21001 Kittridge St.
Canoga Park CA 91303
1/30/2006
HLATools Software, Version
1.0
To aid in assigning Major and /or Minor
Histocompatibility Compexes
BK050071
One Lambda, Inc
21001 Kittridge St.
Canoga Park CA 91303
1/30/2006
Pall Acrodose PL System
Intended to be used to pool and store wholeblood-derived, leukocyte-reduced
platelets in the CLX HP extended
storage bag for up to 5 days
BK050074
Medsep Corporation
19 Pepperwood Lane
Pepper Pike, OH 44124
1/26/2006
ThromboType (HPA 1-6, 15)
and ThromboType 1
(HPA 1)
Alternatives to platelet serological testing and
an adjunct to HLA matching in selecting
compatible platelets for recipients
BK050040
GTI, INC
20925 Crossroads Circle
Waukesha, WI 53186
1/20/2006
Genetic Systems HbsAg EIA
3.0 Ortho Assay
Protocol Disk
Contains the pipetting parameters and analysis
algorithms for Ortho Assay Software
and/or processing method for the Ortho
Summit Processor to run Bio-Rad’s
Genetic Systems HBsAg EIA 3.0, the
assay specified on the disk label
BK050046
Ortho-Clinical Diagnostics, Inc
1001 U.S. Highway 202
P.O. Box 350
Raritan, NJ 08869-0606
1/13/2006
Vaccines
• Products regulated by FDA
• CBER and the Centers for Disease Control &
Prevention (CDC) jointly manage the Vaccine
Adverse Event Reporting System (VAERS)
• VAERS is a post-marketing safety surveillance
program, collecting information about adverse
events
Cellular Therapy
• FDA’s framework for cell & tissue regulation
focuses on– Prevent the use of contaminated tissues or cells
– Prevent improper handling or processing
– Ensure the clinical safety and effectiveness for
tissues or cells that are highly processed & used
for other than their normal function
• FDA organises Biological Response Modifiers
Advisory Committee (BRMAC) for reiveiw of
such products
Tissue
• Regulated under 21 CFR Parts 1270 and 1271
• Examples of such tissues are
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Bone,
Skin,
Corneas,
Ligaments,
Tendons,
Dura mater,
Heart valves,
Hematopoietic stem/progenitor cells,
Oocytes and
Semen.
Tissue
• FDA has published three final rules to
regulate the products effectively
• Requires firms to register and list their products
with FDA
• Requires tissue establishments to evaluate donors,
through screening and testing
• To establish current good tissue practices for
HCT/Ps
Xenotransplantation Action Plan
• Involves the transplantation, implantation, or infusion
into a human recipient of either
(A) live cells, tissues, or organs from a nonhuman
animal source or
(B) human body fluids, cells, tissues or organs that
have had ex vivo contact with live nonhuman
animal cells, tissues, or organs.
• Used experimentally to treat certain diseases such as
• neurodegenerative disorders,
• liver failure, and
• diabetes,
Human Gene Therapy
• Manufacturers of gene therapy products must test their
products extensively and meet FDA requirements for
safety, purity and potency
• To study the gene therapy product in humans, it must obtain
a special permission exemption from FDA
• This exemption is called an investigational new drug
application (IND)
• As part of the IND process, the manufacturer also must
get approval from Institutional Review Board
Human Gene Therapy
• FDA has not yet approved for sale
any human gene therapy product
• Since 1989, FDA has received about
300 requests to study gene therapy
and to develop gene therapy products
• Presently, FDA is overseeing
approximately 210 active IND gene
therapy studies
Allergenics
• Patch test used to diagnose the cause of
contact dermatitis.
• Prior to release standardized products are
compared with US reference std. for
potency.
• CBER maintains these ref. std. and
distributes them.
• There are currently 19 standardized
allergenic extracts are available.
Biological product deviation reporting
(BPDR)
• It requires reporting of any event
associated with• Mfg., testing, packaging, labeling or storage in
which the safety, purity or potency of product may
be affected.
• Report to
• CBER or
• Office of Compliance and Biologics Quality (OCBQ)
Impact of Severe Weather Conditions
on Biological Products
• Most biological products require specific
storage conditions
• For example, on August 14, 2003, the
northeast region of the US experienced a
power outage lasting from one to several
days, and some health professionals were
uncertain what to do with products that
they had in storage
• For such conditions FDA provides online
guidance & also structured a guideline for
this
Marketing approval procedure
for biologics
Biologics license application
(BLA)
• A request for permission to
introduce or deliver for introduction
for a biological product into
interstate commerce.
• 21 CFR 601.2
• Who submit a BLA?
• Manufacturer
• Any legal person who engaged in mfr. who takes
responsibility for compliance with standard
• What is in BLA?
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A form from FDA 356h
Applicant information
Product/mfg. information
Pre-clinical studies
Clinical studies
labeling
Administrative processing of BLA
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The Division of Blood Application have
responsibility for BLA issues
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Includes following events1.
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Submission of INDA to FDA
Phase I, II & III clinical trials
Submission of BLA
After submission•
Inspection of mfg. facilities
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Review of license
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During review
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Information requests
Discipline reviews
Refusal to file
• A refusal to file letter is issued when
the submission has been deemed not
sufficiently complete for a
meaningful review.
Adverse drug reporting
• Performed by• MED WATCH
• Biological Product Deviation Reporting (BPDR)
• Vaccine Adverse Event Report System (VAERS)
• VAERS collects and analyses data from
reports of adverse event following
vaccination
FDA recall policies
• Published in 21 CFR 7.40-59
• Categories of recall
• Class 1 for dangerous or defective products
that could cause serious health problems
• Class 2 for products that might cause a
temporary health problems
• Class 3 for products that are unlikely to cause
any adverse health reaction
Recall submission to FDA
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To local FDA district Recall Coordinator
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Product information
Codes
Recalling firm
Manufacturer
Identify the firm responsible for the problem
Reasons for the recall
Health hazard assessment
Volume of recall product
Distribution pattern
Recall strategy
Consumer / Healthcare Information
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CBER
• CBER is responsible for ensuring the safety and efficacy of
biologics
• CBER's regulation of biological products include a wide variety
of biological products
• If you have questions or are unable to find the information you
need, than contact:
Center for Biologics Evaluation and Research
Office of Communication,
Training & Manufacturers Assistance
Role of CBER in Countering
Bioterrorism
• CBER plays an integral role in several initiatives to
protect the Nation against bioterrorism.
• Helping to advance the development and licensing of
products to diagnose, treat, or prevent outbreaks from
exposure to bioterrorist pathogens by entering the
process at an early stage.
• Helping the products to rapidly meet the regulatory
requirements.
• Developing procedures and protocols to make possible
the safe use of promising experimental products.
References
1. www.fda.gov/cber