Dr. Jules Mitchel, President of Target Health Inc., presents "How Creative Can One Be When Working with the FDA to Optimize the Drug and Device Development Process?" at the October 29 iCAN-Global Entrepreneur's Breakfast Forum.

1. WORKING WITH FDA
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.

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. 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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18. 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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19. HOW DO WE DETERMINE
REGULATORY REQUIREMENTS?
 Hire a regulatory professional
Full-time employee
Consultant
 Lawyer
 Other types of experts
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20. HOW DO WE DETERMINE CLINICAL
REQUIREMENTS?
 Summary Basis of Approval
 FDA Guidances
 Find someone who’s done it already
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21. 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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22. 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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23. 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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24. 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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25. CONDUCTING THE MEETING
 Take complete minutes
 Make sure that all of your issues are addressed
 Chat informally after the meeting
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26. 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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27. 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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