Regulatory and Economic Aspects in Oncology

205 Pages·2019·2.926 MB·English

by  Evelyn Walter

Recent Results in Cancer Research
Series Editors:
Peter-Michael Schlag · Hans-Jörg Senn
Editor
Evelyn Walter   
Regulatory
and Economic
Aspects in
Oncology
Recent Results in Cancer Research
Volume 213
Series editors
Peter-Michael Schlag, Charite Campus Mitte, Charite Comprehensive Cancer
Center, Berlin, Germany
Hans-Jörg Senn, Tumor- und Brustzentrum ZeTuP, St. Gallen, Switzerland
More information about this series at http://www.springer.com/series/392
Evelyn Walter
Editor
Regulatory and Economic
Aspects in Oncology
123
Editor
EvelynWalter
Institute for Pharmaeconomic Research
Vienna,Austria
ISSN 0080-0015 ISSN 2197-6767 (electronic)
Recent Resultsin Cancer Research
ISBN978-3-030-01206-9 ISBN978-3-030-01207-6 (eBook)
https://doi.org/10.1007/978-3-030-01207-6
LibraryofCongressControlNumber:2018957057
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Contents
Introduction and Overview . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1
Evelyn Walter
Cost of Cancer: Healthcare Expenditures and Economic Impact. . . . . . 7
Bengt Jönsson
Oncology from an HTA and Health Economic Perspective . . . . . . . . . . 25
Clement Francois, Junwen Zhou, Michał Pochopien, Leila Achour
and Mondher Toumi
Heterogeneous Recommendations for Oncology Products Among
Different HTA Systems: A Comparative Assessment . . . . . . . . . . . . . . . 39
Szymon Jarosławski, Eve Hanna, Monique Dabbous, Lylia Chachoua
and Mondher Toumi
Patient-Reported Outcomes in Oncology, Beyond Randomized
Controlled Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57
Mondher Toumi, Szymon Jarosławski, Christos Chouhaid,
Bruno Fallissard and P. Auquier
Patient-Reported Outcomes in Health Economic Decision-Making:
A Changing Landscape in Oncology . . . . . . . . . . . . . . . . . . . . . . . . . . . 67
Mandi Pratt-Chapman and Afsan Bhadelia
Approaches to Capturing Value in Oncology. . . . . . . . . . . . . . . . . . . . . 85
Evelyn Walter
Orphan Drugs in Oncology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109
Daria Korchagina, Szymon Jaroslawski, Guy Jadot and Mondher Toumi
Recent Developments in Health Economic Modelling
of Cancer Therapies. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143
William Green and Matthew Taylor
v
vi Contents
Drug Pricing and Value in Oncology . . . . . . . . . . . . . . . . . . . . . . . . . . . 153
Patricia M. Danzon
Regulatory and Evidence Requirements and the Changing
Landscape in Regulation for Marketing Authorisation . . . . . . . . . . . . . 169
Francesco Pignatti and Elias Péan
Prioritization not Rationing in Cancer Care . . . . . . . . . . . . . . . . . . . . . 189
Nikolaus Knoepffler, Jürgen Zerth and Martin O’Malley
About the Editor
Dr. Evelyn Walter has been the Head (and Co-founder) of the Institute of Phar-
maeconomic Research (IPF) in Vienna since 2003. The IPF’s main areas include
health economic evaluation, modeling, and expert advice on reimbursement deci-
sions, pharmaceutical pricing and distribution. She first studied Mathematics at
Vienna University of Technology, before switching to the Vienna University of
EconomicSciences,whereshecompletedamaster’sandadoctorateinEconomics.
Shehasauthoredorco-authoredsome60publications,chieflyinrefereedjournals,
and serves as a reviewer for several health economics journals. In Austria, she
initiatedthe “Guidelinesfor Health Evaluation.”Sheis a Member of theScientific
Advisory Board for the “System of Health Accounts” at Statistics Austria. As a
MemberoftheInternationalSocietyofPharmacoeconomicsandoutcomeResearch
(ISPOR), she co-chaired the 17th and 18th Annual European Congresses. She is a
Lecturer at the University of Applied Sciences in Burgenland, where she teaches
courses on Health Economics, Health Technology Assessment, and Pharma
Management.
vii
Introduction and Overview
Evelyn Walter
Abstract
Theoverallaimofthisbookistosetoutthemainchangesneededinthefieldof
economic and regulatory conditions as a consequence of these rapid develop-
ments in oncology. The traditional approaches of health economics, like health
economic evaluation, health technology assessment (HTA), modeling methods,
assessing value, pricing techniques, are bound to be altered in the contributions
to this book. It is understandable that with the life-threatening diagnosis of
cancer the new treatment options need to be accompanied by the best available
health economic tools. This pertains to well-implemented decision rules
concerning willingness to pay, incremental cost-effectiveness ratio thresholds,
equity, patient access, end of life criteria, etc. Their application differs with
regard to the usual health economic analyzes implemented in other treatment
areas. Overstating a bit one could ask whether we need a strongly modified
concept of oncology economics?
Keywords
(cid:1) (cid:1)
Cancer incidence Oncology cone Prices of cancer medication
The emergence of disruptive innovations is always a challenge for the systems
which are prone to be affected. Cancer therapies for decades experienced
improvements, yet no breakthroughs. They had to rely on incremental discoveries.
With the advent of advanced antibody constructs, cell therapies, nucleic acid
therapies, cancer vaccines, immunotherapies, genomic and proteomic biomarkers
andafurtherpersonalizationoftreatmentsthepicturehaschanged.Atthisstageof
substantialadvancesincancertherapies,anassessmentismandatorytoascertainto
whatextentthepresenthealthsystemiscompelledtoadapt.Inthisvein,thisbook
E.Walter(&)
InstituteforPharmaeconomicResearch,Vienna,Austria
e-mail:[email protected]
©SpringerNatureSwitzerlandAG2019 1
E.Walter(ed.),RegulatoryandEconomicAspectsinOncology,
RecentResultsinCancerResearch213,
https://doi.org/10.1007/978-3-030-01207-6_1
2 E.Walter
provides insights authored by eminent specialists in the field. The perspective
adopted is from economics of health.
Theoverallaimofthisbookistosetoutthemainchangesneededinthefieldof
economic and regulatory conditions as a consequence of these rapid developments
inoncology.The traditional approachesofhealtheconomics, like healtheconomic
evaluation, health technology assessment (HTA), modeling methods, assessing
value,pricingtechniques,areboundtobealtered inthecontributionstothisbook.
It is understandable that with the life-threatening diagnosis of cancer the new
treatment options need to be accompanied by the best available health economic
tools. This pertains to well-implemented decision rules concerning willingness to
pay, incremental cost-effectiveness ratio thresholds, equity, patient access, end of
life criteria, etc. Their application differs with regard to the usual health economic
analyzes implemented in other treatment areas. Overstating a bit one could ask
whether we need a strongly modified concept of oncology economics?
Cancerincidenceisestimatedtodoubleby2035.Thegreatestincreaseincancer
cases is expected in low-income and middle-income countries (LMICs) due to
demographic changes, such as aging of the population, and increasing exposure to
risk factors. While an estimated 60% of cancer cases occur in LMICs, only 5% of
global spending on cancer is directed to these countries (Prager et al. 2017).
Addressing the growing cancer burden as a public health priority is challenging,
because it is not a single disease but rather a multitude of diseases. Many cancers
are heterogeneous in their characteristics, with hundreds of histological and bio-
logicalsubtypes.Itrequiresspecificdiagnosticandtherapeuticstrategies,aswellas
a qualified workforce to implement them, coupled with the imperative need of
coordinated multidisciplinary patient care (Prager et al. 2017).
The dramatic advances in cancer care over the past few decades are obviously
reflectedinsignificantimprovementsinoutcome.Thesetechnologyadvancescreate
four primary opportunities to improving cancer care for patients: earlier detection,
new treatment strategies, personalization, and improved monitoring (McKinsey &
Company2016).Concerningnewtreatments,63cancerdrugs,eachapprovedinone
or more tumors, have impacted the treatment of 24 different cancer types over the
past5years.Theriseofimmuno-oncologysincethefirstlaunchesin2014hasbeen
largelycenteredonthecheckpointinhibitors(PD-1andPD-L1),whichhavebroad
efficacy across solid tumors and are used across 23 different tumor types (IQVIA
Institute 2018). An optimal insight into the changing landscape of the EU phar-
maceutical legislation concerning regulation and evidence requirements and how
new treatments receive marketing authorization is described in the article by Fran-
cesco Pignatti andElias Péan from theEuropeanMedicines Agency (EMA).
The prices of cancer medication have increased rapidly during the last years.
Spanningoveranobservationalperiodof8 years,priceswentupbyanaverageof
6% or more per year, while the inflation rate was just under 1.1% (Reuters 2017).
Spendingoncancermedicines—bothfortherapeuticandsupportivecareuse—rose
from $96 billion in 2013 up to $133 billion globally in 2017 (IQVIA Institute
2018). In the United States alone the situation is even more dramatic, spending on
cancer drugs has doubled since 2012 and reached almost $50 billion in 2017