working with fda

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

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 notF...

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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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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

JMitchel@TargetHealth.com

www.TargetHealth.com

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