Table Of ContentPharmacoeconomics
Pharmacoeconomics
From Theory to Practice
Second Edition
Edited by
Renée J.G. Arnold, PharmD, RPh
Secondeditionpublished2021
byCRCPress
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Title:Pharmacoeconomics:fromtheorytopractice/editedbyRenéeJ.G.Arnold.
Description:Secondedition.|MiltonPark,Abingdon,Oxon;BocaRaton,FL:CRCPress,
[2020]|Includesbibliographicalreferencesandindex.
Identifiers:LCCN2020013270|ISBN9781138589834(hardback)|
ISBN9780367521363(paperback)|ISBN9780429491368(ebook)
Subjects:LCSH:Drugs–Costeffectiveness.|Pharmacy–Economicaspects.|Decisionmaking.
Classification:LCCRS100.P4332020|DDC615.1–dc23
LCrecordavailableathttps://lccn.loc.gov/2020013270
Contents
Preface.............................................................................................................vii
Acknowledgments.............................................................................................ix
Editor...............................................................................................................xi
Contributors...................................................................................................xiii
Chapter1 IntroductiontoPharmacoeconomics.............................................1
WilliamF.McGhanandRenéeJ.G.Arnold
Chapter2 DecisionModelingTechniques....................................................21
MarkS.RobertsandKenSmith
Chapter3 CostofIllness..............................................................................43
RenéeJ.G.Arnold
Chapter4 MarkovModelinginDecisionAnalysis.......................................63
J.RobertBeckandElizabethA.Handorf
Chapter5 RetrospectiveDatabaseAnalysis..................................................79
RenéeJ.G.ArnoldandSanjeevBalu
Chapter6 WhatIsCost-MinimisationAnalysis?........................................109
AlanHaycox
Chapter7 Cost-EffectivenessAnalysis........................................................121
KenSmithandMarkS.Roberts
Chapter8 BudgetImpactAnalysis.............................................................137
JosephineMauskopfandLievenAnnemans
Chapter9 MulticriteriaDecisionAnalysisfortheHealthcare
DecisionMaker..........................................................................153
SumitraSriBhashyamandKevinMarsh
v
vi Contents
Chapter10 DICESimulation:AUnifyingModelingApproachfor
Pharmacoeconomics..................................................................171
J.JaimeCaroandJörgenMöller
Chapter11 SomeProblems/AssumptionsinPharmacoeconomic
Analysis......................................................................................189
StuartBirksandAlanHaycox
Chapter12 Patient-ReportedOutcomeMeasures.........................................207
DianneBryant,AndrewFirth,GordonGuyatt,and
RenéeJ.G.Arnold
Chapter13 SensitivityAnalysis....................................................................229
MaartenJ.Postma
Chapter14 UseofPharmacoeconomicsinDrugReimbursement:
WhatCanWeLearnfromInternationalExperience?.................241
ChrisSampson,MargheritaNeri,andKyannZhang
Chapter15 Value-BasedPricingofPharmaceuticalsintheUSand
Europe.......................................................................................255
RenéeJ.G.ArnoldandNeilX.Hawkins
Chapter16 PharmacoeconomicsinDiseaseManagement:Practical
ApplicationsandPersistentChallenges......................................265
RyungSuhandDavidAtkins
Chapter17 FromMachineLearninginDrugDiscoveryto
Pharmacoeconomics..................................................................279
SeanEkinsandRenéeJ.G.Arnold
Chapter18 SpeculationsontheFutureChallengesandValueof
Pharmacoeconomics..................................................................295
J.JaimeCaro
Chapter19 PharmacoeconomicsintheEraoftheNovelCoronavirus.........305
RenéeJ.G.Arnold
Index..............................................................................................................311
Preface
The genesis of this book was the pharmacoeconomics research and other
outcomes projects that my colleagues and I have completed for our pharma-
ceutical company and government clients over the years. The chapters’ ideas
came specifically from the Introduction to Pharmacoeconomics course that
I developed and currently teach for the Icahn School of Medicine at Mount
Sinai Master of Public Health Program. I have extensively collaborated with
many of the colleagues who have written chapters for this book and I am
truly grateful to them who, despite being extremely busy, have contributed
their valuable time and collective wisdom to make this book useful and prac-
tical. This book is meant to introduce the major concepts and principles of
pharmacoeconomics, with particular emphasis on modeling, methodologies,
and data sources and applications to real-world dilemmas. Readers will learn
about the use of pharmacoeconomics in drug reimbursement and pricing
internationally. Examples of pharmacoeconomic models used to support
these purposes in government, the pharmaceutical industry, and healthcare
settings are also given.
The first edition of this book focused on pharmacoeconomic analyses of
a public health vaccination program and collaboration among members of
the pharmaceutical industry, academia, and government in the development
of the human papillomavirus vaccine. In this second edition, we broaden the
focus to include more recent examples of published analyses and/or current
thinking about relevant issues, such as new chapters on multicriteria decision
analysis (MCDA), discretely integrated condition event (DICE) simulation,
global implementation of value frameworks, international experience in use
of pharmacoeconomics in drug reimbursement, and anticipated future devel-
opments in the field. In addition to cost, the book examines a full range of
ethical and moral issues, as well as overall public health and commercial
concerns that are often involved in decisions entailing pharmacoeconomic
decision-making. Unfortunately, this book was already in galleys when the
novel severe acute respiratory syndrome (SARS-CoV-2, also referred to as
Covid-19) became a global pandemic in 2020, so has been addressed only in
a few instances here and in a final small chapter. Suffice it to say, however, that
a multitude of issues described in the book are extremely relevant to the public
health and economic consequences being faced currently and for years to come.
Lest the reader think these issues esoteric or untimely, the reader is referred
to multiple articles both in the scientific literature and lay press (e.g., The
New York Times and Wall Street Journal) on comparative cost-effectiveness of
medications for pandemic diseases, such as the novel coronavirus-19, prevalent
diseases, such as hepatitis C and atopic eczema, and also for rare diseases
such as spinal muscular atrophy.
These principles are being embodied, for example, in the much-discussed
US Institute for Clinical and Economic Review (interestingly, the same
vii
viii Preface
acronym as an oft-used concept in pharmacoeconomics – the incremental
cost-effectiveness ratio, or ICER) evaluations and in guidances rendered
by the UK’s National Institute for Health and Care Excellence (NICE).
Pharmacogenomics, or the use of personalized medicine, will be combined
with cost-effectiveness analyses, to inform and improve healthcare decision-
making. Thus, improved and cost-effective decisions, using the best available,
evidence-based medicine, will require that both clinical and economic expertise,
as epitomized in this book, be used.
Renée J. G. Arnold, PharmD, RPh
Acknowledgments
Igratefullyacknowledgemycolleagueandfriend,Dr.SeanEkins,whoprompted
me towrite the first edition of this book and also contributed a chapter to both
the first and second editions. I also acknowledge all of my illustrious coauthors
(according to chapter number) Drs. William F. McGhan, Mark Roberts, Ken
Smith, J. Robert Beck (who included updated examples and created a new
figure!),SanjeevBalu,AlanHaycox(whotookonanadditionalchapterbesides
his own), Lieven Annemans, Jo Mauskopf (who was so kind as to update
Dr. Annemans’ chapter with incredibly helpful text, examples, and illustra-
tions), Kevin Marsh (who provided a new chapter on MCDA), Sumitra Sri
Bhashyam (new chapter coauthor), Jaime Caro (with his wit and insight about
whatthefuturemayholdinourfieldandwhohaskindlyprovidedmewithpro-
fessional guidance), Stuart Birks, Gordon Guyatt, Dianne Bryant, Andrew
Firth, Maarten Postma, Chris Sampson, Neil X. Hawkins (who provided the
international counterpoint to our newvalue-basedpricing chapter), and Ryung
Suh.Lastly,intermsofthissecondedition,IgratefullythankMs.HilaryLafoe
and Ms. Jessica Poile who encouraged me to be thorough and unrelenting in
pursuing excellence and to my excellent editors, Subathra Manogaran and
SunanthaRamamoorthy.
Although not included in this newest edition, I would also like to thank
Dr. Michael Drummond for his support initially in my health economics and
outcomes research career (I recruited him as the keynote speaker for the first
International Society for Pharmacoeconomics and Outcomes Research [ISPOR]
meeting) and throughout my career in the field; he always responded in
a forthright and helpful manner. Speaking of ISPOR, I also gratefully acknow-
ledge the late Dr. Marilyn Dix Smith and Dr. Alan Bakst, who approached me
after a CEPOR meeting in North Carolina to help cofound ISPOR, originally
founded as the Association for Pharmacoeconomics and Outcomes Research
(APOR),in1995.Marilynwasmy truementorinthe organization.I wouldalso
like to thank my parents, Jack and Betty Goldberg; Jack was a true clinical
pharmacist whom everyone who went to his pharmacies called “Doc” and who
was my inspiration to enter the field. Betty continues to be an inspiration in my
scientificandpersonalendeavors.Bothhavebeenaninspirationaboutthepower
of persistence. Lastly, I would be remiss if I do not acknowledge my husband,
Michael,andson,Marc,fortheirongoingloveandsupport!
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