FDA regulatory affairs : a guide for prescription drugs, medical devices, and biologics

461 Pages·2008·8.02 MB·English

by  Douglas J. Pisano

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SECOND
S E COND ED ITION
Pharmaceutical Science EDITION
about the book…
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Examines harmonization of the US Federal Food, Drug, and Cosmetic Act with international
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regulations as they apply to human drug and device development, research, manufacturing, u
and marketing. The Second Edition focuses on the new drug approval process, cGMPs, i
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GCPs, quality system compliance, and corresponding documentation requirements. Written e
in a jargon-free style, it draws information from a wide range of resources. It demystifies the f
inner workings of the FDA and facilitates an understanding of how it operates with respect to o F
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compliance and product approval. D
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FDA Regulatory Affairs: r
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(cid:115) provides a blueprint to the FDA and drug, biologic, and medical device development s
(cid:115) offers current, real-time information in a simple and concise format c
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(cid:115) contains a chapter highlighting the new drug application (NDA) process i
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(cid:115) discusses FDA inspection processes and enforcement options t e
i
(cid:115) includes contributions from experts at companies such as Millennium and Genzyme, o g
leading CRO’s such as PAREXEL and the Biologics Consulting Group, and the FDA n
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Three all-new chapters cover:
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(cid:115) clinical trial exemptions u
(cid:115) advisory committees g a
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(cid:115) provisions for fast track , t

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about the editors... o
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DOUGLAS J. PISANO is Dean of the School of Pharmacy and Professor of Pharmacy d r
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Administration, Massachusetts College of Pharmacy and Health Sciences, Boston, c y
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Massachusetts, USA. Dr. Pisano received his Ph.D. in Law, Policy, and Society at Northeastern
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University, Boston, Massachusetts, USA. He is an active member of several professional A
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organizations, including the American Association of Colleges of Pharmacy and the American
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Pharmaceutical Association. A national speaker and invited lecturer, Dr. Pisano was the v f
recipient of the Special Service Award for the Enhancement of Regulatory Education from the ic f
Regulatory Affairs Professionals Society in 2000. He has developed several courses and e a
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programs at the Massachusetts College of Pharmacy and Health Sciences in such areas as ,
i FDA
health policy, pharmacy and drug law, and regulatory affairs. Dr. Pisano, along with coeditor a r
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Dr. David S. Mantus, is also the editor of the first edition of Informa Healthcare’s FDA Regulatory
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Affairs: A Guide for Prescription Drugs, Medical Devices, and Biologics.
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DAVID S. MANTUS is Vice President of Regulatory Affairs and Program Management, Cubist i Regulatory
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Pharmaceuticals, Inc., Lexington; Adjunct Professor of Drug Regulatory Affairs, Massachusetts l
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College of Pharmacy and Health Sciences, Boston; and President, C After D Inc., Boston,
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Massachusetts, USA. Dr. Mantus received his Ph.D. in Chemistry from Cornell University, i
c Affairs
Ithaca, New York, USA. He is an active member of the Regulatory Affairs Professional Society s
and the American Chemical Society and is a frequent presenter and lecturer at national
conferences on biologics and biotechnology, regulatory affairs, and vaccine development.
Dr. Mantus has also served as chairperson for several conferences, including “Outsourcing
Pisano
Regulatory Affairs” and “Vaccine Development for the 21st Century.” A Guide for Prescription Drugs,
—
Printed in the United States of America (cid:40)(cid:23)(cid:19)(cid:21)(cid:20)
Mantus Medical Devices, and Biologics
Edited by
Douglas J. Pisano
David S. Mantus
Pisano_978-1420073546.indd 1 nC nM nY nK 7/3/08 10:20:54 AM
FDA
Regulatory
Affairs

FDA
Regulatory
Affairs
A Guide for Prescription Drugs,
Medical Devices, and Biologics
Second Edition
Edited by
Douglas J. Pisano
Massachusetts College of Pharmacy and Health Sciences
Boston, Massachusetts, USA
David S. Mantus
Cubist Pharmaceuticals, Inc.
Lexington, Massachusetts, USA
InformaHealthcareUSA,Inc.
52VanderbiltAvenue
NewYork,NY10017
#2008byInformaHealthcareUSA,Inc.
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LibraryofCongressCataloging-in-PublicationData
FDAregulatoryaffairs:aguideforprescriptiondrugs,medicaldevices,andbiologics/
editedbyDouglasJ.Pisano,DavidS.Mantus.— 2nded.
p.;cm.
Includesbibliographicalreferencesandindex.
ISBN-13:978-1-4200-7354-6(hb:alk.paper)
ISBN-10:1-4200-7354-0(hb:alk.paper) 1.Drugdevelopment—United
States.2.UnitedStatesFoodandDrugAdministration—Rulesandpractice.
3.Pharmaceuticalindustry—UnitedStates. I.Pisano,DouglasJ. II.Mantus,David.
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Preface
This book is a roadmap to the U.S. Food and Drug Administration and drug,
biologic,andmedicaldevicedevelopment.ItiswritteninplainEnglish,withan
emphasisoneasyaccesstounderstandinghowthisagencyoperateswithrespect
to the practical aspects of U.S. product approval. It is meant to be a concise
reference that offers current, real-time information. It has been written as a
handy reference for use by students, staff, and professionals at corporations,
organizations, and schools and colleges across the United States in need of a
simple,concisetextfromwhichtolearnandteach.ThetopicsinFDARegulatory
Affairs:AGuideforPrescriptionDrugs,MedicalDevices,andBiologics,Second
Editionarecovered in astraightforward format.Itisa compilationand commen-
tary of selected laws and regulations pertaining to the development and approval
ofdrugs,biologics,andmedicaldevicesintheUnitedStates.Itisnotintendedto
taketheplaceofanactualreadingoftheLawsoftheUnitedStatesofAmericaor
the regulations of the U.S. Food and Drug Administration, it’s agencies or any
bodythatregulatesthedevelopmentorapprovalofdrugs,biologics,and medical
devices in the United States.
Douglas J. Pisano
David S. Mantus
iii

Contents
Preface . . . . . . . . . .. iii
Contributors . . . . . . .. vii
1. Overview of FDA and Drug Development . . . . . . . . . . . . . . . .. 1
Josephine C. Babiarz and Douglas J. Pisano
2. What Is an IND? . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .. 33
Michael R. Hamrell
3. The New Drug Application . . . . . . . . . . . . . . . . . . . . . . . . . .. 69
Charles Monahan and Josephine C. Babiarz
4. Meeting with the FDA . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109
Alberto Grignolo
5. FDA Medical Device Regulation . . . . . . . . . . . . . . . . . . . . . . 125
Barry Sall
6. The Development of Orphan Drugs . . . . . . . . . . . . . . . . . . . 167
Tan T. Nguyen
7. CMC Sections of Regulatory Filings and CMC
Regulatory Compliance During Investigational
and Postapproval Stages . . . . . . . . . . . . . . . . . . . . . . . . . . . 187
Prabu Nambiar and Steven R. Koepke
8. Overview of the GxPs for the Regulatory Professional . . . . . . 213
Bob Buckley and Robert Blanks
v
vi Contents
9. FDA Regulation of the Advertising and Promotion
of Prescription Drugs, Biologics, and Medical Devices . . . . . . 267
Karen L. Drake
10. Electronic Submissions—A Guide for Electronic
Regulatory Submissions to FDA . . . . . . . . . . . . . . . . . . . . . . 289
Shylendra Kumar, Yolanda Hall, and Vahe´Ghahraman
11. The Practice of Regulatory Affairs . . . . . . . . . . . . . . . . . . . . 351
David S. Mantus
12. A Primer of Drug/Device Law: What’s the Law
and How Do I find It? . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369
Josephine C. Babiarz
13. FDA Advisory Committees . . . . . . . . . . . . . . . . . . . . . . . . . . 397
Christina A. McCarthy and David S. Mantus
14. Biologics . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417
Timothy A. Keutzer
Index . . . . . 435
Contributors
Josephine C. Babiarz MS Program in Regulatory Affairs, Massachusetts
College of Pharmacy and Health Sciences, Boston, Massachusetts, U.S.A.
Robert Blanks Idenix Pharmaceuticals, Inc., Cambridge, Massachusetts,
U.S.A.
Bob Buckley Idenix Pharmaceuticals, Inc., Cambridge, Massachusetts, U.S.A.
Karen L. Drake Cubist Pharmaceuticals, Inc., Lexington, Massachusetts,
U.S.A.
Vahe´ Ghahraman Regulatory Operations and Technology, Dyax Corporation,
Cambridge, Massachusetts, U.S.A.
Alberto Grignolo PAREXEL Consulting, Lowell, Massachusetts, U.S.A.
Yolanda Hall Datafarm, Inc., Marlborough, Massachusetts, U.S.A.
Michael R. Hamrell MORIAH Consultants, Yorba Linda, California, U.S.A.
TimothyA.Keutzer CubistPharmaceuticals,Inc., Lexington,Massachusetts,
U.S.A.
Steven R. Koepke SRK Consulting, LLC, Walkersville, Maryland, U.S.A.
Shylendra Kumar Datafarm, Inc., Marlborough, Massachusetts, U.S.A.
DavidS.Mantus CubistPharmaceuticals,Inc.,Lexington,Massachusetts,U.S.A.
Christina A. McCarthy Cubist Pharmaceuticals, Inc., Lexington,
Massachusetts, U.S.A.
Charles Monahan Regulatory Affairs, Molecular Insight Pharmaceuticals,
Inc., Cambridge, Massachusetts, U.S.A.
vii