Working with FDA: How creative one can be when working with FDA to optimize the drug and device development process.

1. How Creative One Can 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

2. 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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3. GENERAL APPROACH
Have Good Medicine
Have Good Science
Have Good Regulations
Have Pride in Your Product
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4. THE TEAM
Discovery/Development
Marketing
Clinical
Legal
Toxicology
Regulatory
FDA
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5. 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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6. AVAILABLE SERVICES
FREEDOM OF INFORMATION - FDA
FOI SERVICES
FDA SMALL BUSINESS HELP LINE
DATABASES
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7. WEB SITES
FDA.GOV
CDC.GOV
NIH.GOV
TARGETHEALTH.COM
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8. 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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9. 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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10. FORMS
1571
1572
483
356H
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11. 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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12. Innovation at FDA
Fast Track
Accelerated Approval
Priority Review
Breakthrough Therapy
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformatio
n/Guidances/UCM358301.pdf
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13. 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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14. 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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15. PRIORITY REVIEW
A 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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16. 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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17. 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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18. 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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19. 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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20. CONDUCTING THE MEETING
Take complete minutes
Make sure that all of your issues are addressed
Chat informally after the meeting
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21. 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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22. TARGET HEALTH INC.
Dr. Jules T. Mitchel, President
261 Madison Avenue, 24th Floor
New York, NY 10016
Tel: (212) 681-2100 ext 0
JMitchel@TargetHealth.com
www.TargetHealth.com
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