working with the fda
TRANSCRIPT
How Creative Can One Be When Working with the FDA to Optimize the Drug and Device
Development Process?
Jules T. Mitchel, MBA, PhD
President, Target Health Inc.
WORKING WITH FDA
Target Health Inc.
TARGET HEALTH INC., founded in 1993, is a
private, New York City-based, full-service eCRO,
engaged in all aspects of Drug and Device
Development, including Regulatory Affairs
Strategic Planning, Clinical Research, Data
Management, Biostatistics, Medical Writing and
the Paperless Clinical Trial.
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GENERAL APPROACH
Have Good Medicine
Have Good Science
Have Good Regulations
Have Pride in Your Product
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THE TEAM
Discovery/Development
Marketing
Clinical
Legal
Toxicology
Regulatory
FDA
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DEVELOPMENT PHILOSOPHY
Don’t Waste Time, Time is Money
Plan Carefully
Execute Meticulously
Re-plan When Necessary
Do Only What is Needed
Hire People Who Want to Get the Job Done
And it will cost more than you think and it will take longer
than you think
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AVAILABLE SERVICES
FREEDOM OF INFORMATION - FDA
FOI SERVICES
FDA SMALL BUSINESS HELP LINE
DATABASES
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WEB SITES
FDA.GOV
CDC.GOV
NIH.GOV
TARGETHEALTH.COM
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FDA STRUCTURE
Center for Drug Evaluation and Research
(CDER)
Center for Biologics Evaluation and Research
(CBER)
Center for Devices and Radiologic Health
(CDRH)
Center for Food Science and Applied Nutrition
(CFSAN)
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DEFINITIONS
IND - INVESTIGATIONAL NEW DRUG APPLICATION
NDA - NEW DRUG APPLICATION
IDE - INVESTIGATIONAL DEVICE EXEMPTION
PMA - PREMARKET APPROVAL APPLICATION
DMF - DRUG MASTER FILE
GMP - GOOD MANUFACTURING PRACTICES
GLP - GOOD LABORATORY PRACTICES
GCP - GOOD CLINIAL PRACTICES
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FORMS
1571
1572
483
356H
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Innovation at FDA
Driving Biomedical Innovation http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM274464.pdf
Innovation at CDER http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm
Innovation and Regulatory Science http://www.fda.gov/BiologicsBloodVaccines/ScienceResearch/ucm234680.htm
CDRH Medical Device Innovation Initiative http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHI
nnovation/default.htm
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Innovation at FDA
Fast Track
Accelerated Approval
Priority Review
Breakthrough Therapy
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf
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FAST TRACK
Fast track emphasizes the critical nature of close
early communication between the FDA and
sponsors.
Fast track adds to existing programs, such as
accelerated approval, the possibility of a "rolling
review" for an application.
An applicant must submit a request with supporting
documentation for designation and FDA must
respond within 60 days.
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Accelerated Approval This program allows for earlier approval of drugs
that treat serious conditions, and fill an unmet
medical need based on a surrogate endpoint; a
marker that is thought to predict clinical benefit, but
is not itself a measure of clinical benefit.
Drug companies are still required to conduct studies
to confirm the anticipated clinical benefit. If the
confirmatory trial shows that the drug actually
provides a clinical benefit, then the FDA grants
traditional approval for the drug. If not…...
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PRIORITY REVIEWA Priority Review designation means FDA’s goal is to take
action on an application within 6 months (compared to 10
months) if the drug could provide significant advantages such
as:
evidence of increased effectiveness in treatment, prevention,
or diagnosis of condition;
elimination or substantial reduction of a treatment-limiting
drug reaction
documented enhancement of patient compliance
evidence of safety and effectiveness in a new subpopulation.
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BREAKTHROUGH THERAPY
A breakthrough therapy is a drug:
intended alone or in combination with one or more
other drugs to treat a serious or life threatening
disease or condition.
preliminary clinical evidence indicates that the drug
may demonstrate substantial improvement over
existing therapies on one or more clinically
significant endpoints, such as substantial treatment
effects observed early in clinical development.
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HOW DO WE DETERMINE WHERE OUR
PRODUCT BELONGS?
What center and what group within that center will have
primary review of our product?
Sometimes its obvious
Call the FDA and find someone who is helpful
Submit a request for designation
Submit IND or IDE and find out
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HOW TO WORK WITH FDA
What should be our initial contact with the FDA and
how should we do it?
Preparation for the initial FDA meeting.
How should we conduct an FDA meeting?
Should we view the FDA as part of our development
team?
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HOW DO WE DETERMINE
REGULATORY REQUIREMENTS?
Hire a regulatory professional
Full-time employee
Consultant
Lawyer
Other types of experts
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HOW DO WE DETERMINE CLINICAL
REQUIREMENTS?
Summary Basis of Approval
FDA Guidances
Find someone who’s done it already
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WHAT SHOULD BE OUR INITIAL CONTACT
WITH FDA AND HOW SHOULD WE DO IT?
First, do your homework
You could call the division of interest at FDA and
briefly introduce yourself
Discuss the project and its status
Discuss option of an initial meeting
Establish action items
Plan a meeting
Do initial filing and then meet
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PREPARATION FOR THE FDA MEETING
Assign one person to organize and champion the meeting
Prepare a solid document providing
rationale
chemistry issues
non-clinical pharmacology issues and data
clinical data, if available
clinical protocol
reprints
table of contents
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MEETING MATERIALS
Paginate, check quality of photocopying
Find out how many copies
Put in proposed meeting dates and blackout dates
Let FDA know when the briefing document is sent
Confirm receipt over the phone
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HOW SHOULD WE CONDUCT AN FDA
MEETING?
Be prepared
Identify one experienced scientific and one experienced clinical expert as participants
Bring in the president and a marketing person
Make sure each person knows his/her role
Make no formal presentation
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CONDUCTING THE MEETING
Take complete minutes
Make sure that all of your issues are addressed
Chat informally after the meeting
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SHOULD WE VIEW THE FDA AS PART
OF OUR DEVELOPMENT TEAM?
Share ideas
Set milestones
Send data
Maintain dialogue
No secrets
FDA is part of your team whether you like
it or not
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TARGET HEALTH INC.
Dr. Jules T. Mitchel, President
261 Madison Avenue, 24th Floor
New York, NY 10016
Tel: (212) 681-2100 ext 0
www.TargetHealth.com
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