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(Download PDF) Applied Methods of Cost Effectiveness Analysis in Healthcare 3Rd Edition Alistair M Gray Online Ebook All Chapter PDF
(Download PDF) Applied Methods of Cost Effectiveness Analysis in Healthcare 3Rd Edition Alistair M Gray Online Ebook All Chapter PDF
(Download PDF) Applied Methods of Cost Effectiveness Analysis in Healthcare 3Rd Edition Alistair M Gray Online Ebook All Chapter PDF
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Contents
1 Introduction
2 Economic evaluation in health care
3 Life tables and extrapolation
4 Modelling outcomes using patient-level data
5 Measuring, valuing, and analysing health outcomes
6 Defining, measuring, and valuing costs
7 Analysing costs
8 Decision analytic modelling: decision trees
9 Decision analytic modelling: Markov models
10 Representing uncertainty in decision analytic models
11 Presenting cost-effectiveness results
12 Summing up and future directions
Index
Applied Methods of
Cost-effectiveness Analysis
in Health Care
Handbooks in Health Economic Evaluation Series
Series editors: Alastair M. Gray and Andrew Briggs
Economic evaluation in health care is a thriving international activity that is increasingly used to
allocate scarce health resources, and within which applied and methodological research,
teaching, and publication are flourishing. Several widely respected texts are already well
established in the market, so what is the rationale for not just one more book, but for a series?
We believe that the books in the series Handbooks in Health Economic Evaluation share a strong
distinguishing feature, which is to cover as much as possible of this broad field with a much
stronger practical flavour than existing texts, using plenty of illustrative material and worked
examples. We hope that readers will use this series not only for authoritative views on the
current practice of economic evaluation and likely future developments, but for practical and
detailed guidance on how to undertake an analysis. The books in the series are textbooks, but
first and foremost they are handbooks.
Our conviction that there is a place for the series has been nurtured by the continuing success
of two short courses we helped develop—Advanced Methods of Cost-Effectiveness Analysis,
and Advanced Modelling Methods for Economic Evaluation. Advanced Methods was developed
in Oxford in 1999 and has run several times a year ever since, in Oxford, Canberra, and Hong
Kong. Advanced Modelling was developed in York and Oxford in 2002 and has also run several
times a year ever since, in Oxford, York, Glasgow, and Toronto. Both courses were explicitly
designed to provide computer-based teaching that would take participants not only through the
theory but also through the methods and practical steps required to undertake a robust economic
evaluation or construct a decision analytic model to current standards. The proof of concept was
the strong international demand for the courses—from academic researchers, government
agencies, and the pharmaceutical industry—and the very positive feedback on their practical
orientation.
Therefore the original concept of the Handbook series, as well as many of the specific ideas
and illustrative material, can be traced to these courses. The Advanced Modelling course is in the
phenotype of the first book in the series, Decision Modelling for Health Economic Evaluation,
which focuses on the role and methods of decision analysis in economic evaluation. The
Advanced Methods course has been an equally important influence on Applied Methods of Cost-
effectiveness Analysis in Health Care, the third book in the series which sets out the key
elements of analysing costs and outcomes, calculating cost-effectiveness, and reporting results.
The concept was then extended to cover several other important topic areas. Firstly, the design,
conduct, and analysis of economic evaluations alongside clinical trials have become a specialized
area of activity with distinctive methodological and practical issues, and their own debates and
controversies. It seemed worthy of a dedicated volume—hence the second book in the series,
Economic Evaluation in Clinical Trials. Next, while the use of cost–benefit analysis in health
care has spawned a substantial literature, this is mostly theoretical, polemical, or focused on
specific issues such as willingness to pay. We believe that the fourth book in the series, Applied
Methods of Cost–Benefit Analysis in Health Care, fills an important gap in the literature by
providing a comprehensive guide not only to the theory but also to the practical conduct of cost–
benefit analysis, again with copious illustrative material and worked out examples.
Each book in the series is an integrated text prepared by several contributing authors, widely
drawn from academic centres in the UK, the USA, Australia, and elsewhere. Part of our role as
editors has been to foster a consistent style, but not to try to impose any particular line. That
would have been unwelcome and also unwise amidst the diversity of an evolving field. News and
information about the series, as well as supplementary material for each book, can be found at
the series website (http://www.herc.ox.ac.uk/books).
Many people have been involved directly and indirectly in this book, reflecting the fact that the
Advanced Methods of Cost-Effectiveness short course from which it has grown has always been
a team effort. The course was originally conceived and developed by Andy Briggs, Philip
Clarke, Alastair Gray, Kathy Johnston, and Jane Wolstenholme. Almost every member of the
Health Economics Research Centre has subsequently been involved in teaching or tutoring, and
many incremental changes to material have resulted. We are grateful to all of these present and
past colleagues, and to the many hundreds of students who have so far attended the course,
providing valuable feedback and insight, and challenging us to express ideas as clearly as
possible.
During the preparation of this book a number of individuals contributed significantly,
providing detailed comments on the text of the book or the exercises. We would like to thank in
particular Maria Alva, James Buchanan, Helen Campbell, Helen Dakin, Boby Mihaylova, Judit
Simon, and Elizabeth Stokes. We would also like to thank Alison Hayes and Tom Lung from the
University of Sydney for their comments on Chapters 3 and 4. In addition, Jose Leal, Ramon
Luengo-Fernandez, and Oliver Rivero-Arias made valuable contributions to the chapters on
modelling, costing, and presenting results, and to the development and testing of the exercises.
For many years Alison Gater has played an invaluable role in both sustaining and organising the
course, and has dealt with permissions and many other administrative matters related to the
manuscript. In addition to his key role in getting the course off the ground, we are grateful to
Andy Briggs for his comments and support in his role as series co-editor. Finally, we would like
to thank Charlotte, Andy, and Dave for repairing the many domestic disruptions involved in
producing a book.
Abbreviations and acronyms
Introduction
The British economist Lionel Robbins famously defined economics as ‘…a science which
studies human behaviour as a relationship between ends and scarce means which have alternative
uses’ (Robbins 1932). The concepts of scarcity and choice will have resonance for anyone
involved in the planning and provision of health care: the available resources are never sufficient
to allow all available health interventions to be provided, and so choices have to be made, which
sometimes involve very difficult decisions. How scarce resources can best be allocated in order
to best satisfy human wants is in fact the basic economic problem, and it is encountered whether
the context is sub-Saharan Africa or North America, even though the absolute level of resources
available may differ enormously.
In health care, this problem can be restated as ‘How can the scarce health resources allocated
to health care best be used in order to maximise the health gain obtained from them?’ This is not
to assert that maximizing health gain is the only objective of a health care system—it may also
have interests in the fairness with which resources are used, or in other objectives such as
education, training, and research. But maximizing health gain—the efficiency objective—is
clearly important, and since the 1970s a set of analytical methods of economic evaluation has
been developed to help decision-makers address this problem.
1.2 Rationale
An important reason for devising the course, and now for translating it into a book, is that
standards of best practice in economic evaluation have become more explicit and more
demanding over time. From the early 1990s health technology assessment and reimbursement
agencies began to produce and refine guides to the way they wished economic data to be
analysed and presented, for example for Australia (Commonwealth of Australia 1995;
Pharmaceutical Benefits Advisory Committee 2008), for Ontario (Ontario Ministry of Health
1994), for Canada as a whole (Canadian Coordinating Office for Health Technology Assessment
1997; Canadian Agency for Drugs and Technologies in Health 2006), and for England and
Wales (NICE 2004, 2008).
Even without the spur of having to make formal reimbursement decisions, in 1993 the US
Public Health Service became so frustrated by the lack of consensus in the techniques used to
conduct cost-effectiveness analysis that it convened an expert group to make recommendations
that would improve quality and enhance comparability. The result was an influential textbook,
which included a strong plea for the use of a reference case or standard set of methodological
practices which, if included by analysts when reporting their results (along with other scenarios
if desired), would significantly increase comparability between studies (Gold et al. 1996).
This trend was followed by journals such as the British Medical Journal and the New England
Journal of Medicine, which began to specify how they wanted economic evaluations to be
presented, and how they expected referees to assess them (Kassirer and Angell 1994; Drummond
and Jefferson 1996, on behalf of the BMJ Economic Evaluation Working Party). More
specialized guidelines have also been produced, for example to improve standards in decision
analytic modelling (Philips et al. 2006).
Another rationale for the course and this book is that health economists are becoming involved
in more complex studies. For example, they may be conducting economic evaluations alongside
large pragmatic trials running over a long period of time with multiple comparisons, multiple
endpoints, and incomplete patient-specific data on resource use and quality of life. There has also
been a dawning recognition that even the largest and longest clinical trials do not remove the
need for modelling, which may be required before, during, after, and instead of trials (Buxton et
al. 1997).
Finally, the title—when the course was conceived it seemed reasonable to call it ‘Advanced
Methods of Cost-Effectiveness Analysis’, as the prevailing standards in the published literature
often fell well short of best practice. When Briggs and Gray (1999) surveyed 492 studies
published up to 1996 that reported results in terms of cost per life-year or cost per quality-
adjusted life-year, they found that barely 10% had used patient-level data, that fewer than 1%
reported a confidence interval for estimates of average cost, and that 20% did not report any
measure of variance at all. However, in the time that has elapsed since then, and partly as a result
of the various textbooks, guidelines, and courses mentioned above, methods have improved and
the contents of this book are, or at least should be, considered standard practice. Therefore we
have called this text ‘Applied Methods of Cost-effectiveness Analysis in Health Care’.
1.4 Exercises
As with other handbooks in this series, an important feature of this book is an emphasis on
practical examples and exercises. Some of the exercises are stand-alone activities designed to
provide experience of dealing with particular types of analysis or data issues, but running
through the outcome, cost, and presenting results chapters is an integrated exercise which uses
the same underlying dataset consisting of a set of hypothetical patients, with their associated
characteristics, complications, costs, and outcomes. The data were generated using the UK
Prospective Diabetes Study Outcome Model (Clarke et al. 2004), a simulation model developed
using patient-level data from a large prospective trial of therapies for type 2 diabetes. This
exercise gradually builds on the solutions derived in previous chapters. More details are provided
in the relevant place in each chapter. As with previous handbooks (Briggs et al. 2006), we have
chosen to base these exercises largely on Microsoft Excel® software, which is widely available,
transparent, and avoids the ‘black-box’ aspects of some other dedicated software. Some degree
of familiarity with Excel is assumed, but the step-by-step guides should permit all readers to
complete the exercises. The exercises in Chapters 8, 9, and 10 can be done using either Excel or
TreeAge™ software. Again, detailed notes are provided, but readers will have to obtain a
licensed copy of TreeAge if they do not have it already. The step-by-step guides can be found at
the end of each chapter, and supplementary material including workbooks and solution files can
also be found on the website www.herc.ox.ac.uk/books/applied that has been set up to support
this book.
References
Briggs, A.H., Claxton, K., and Sculpher, M.J. (2006). Decision Modelling for Health Economic
Evaluation. Oxford University Press.
Briggs, A.H. and Gray, A.M. (1999). Handling uncertainty in economic evaluations of healthcare
interventions. British Medical Journal, 319, 635–8.
Buxton, M.J., Drummond, M.F., Van, H.B., et al. (1997). Modelling in economic evaluation: an
unavoidable fact of life [editorial]. Health Economics, 6, 217–27.
Canadian Agency for Drugs and Technologies in Health (2006). Guidelines for Economic
Evaluation of Pharmaceuticals: Canada. Canadian Agency for Drugs and Technologies in
Health (CADTH), Ottawa.
Canadian Coordinating Office for Health Technology Assessment (1997). Guidelines for the
Economic Evaluation of Pharmaceuticals: Canada (2nd edn). Canadian Coordinating Office
for Health Technology Assessment (CCOHTA), Ottawa.
Clarke, P., Gray, A., Briggs, A., et al. (2004). A model to estimate the lifetime health outcomes
of patients with type 2 diabetes: the United Kingdom Prospective Diabetes Study (UKPDS)
Outcomes Model (UKPDS 68). Diabetologia, 47, 1747–59.
Commonwealth of Australia (1995). Guidelines for the Pharmaceutical Industry on Preparation
of Submissions to the Pharmaceutical Benefits Advisory Committee: Including Economic
Analyses. Department of Health and Community Services, Canberra.
Drummond, M.F. and Jefferson, T.O. (1996). Guidelines for authors and peer reviewers of
economic submissions to the BMJ. British Medical Journal, 313, 275–83.
Drummond, M.F., Sculpher, M.J., Torrance, G.W., O’Brien, B.J., and Stoddart, G.L. (2005).
Methods for the Economic Evaluation of Health Care Programmes (3rd edn). Oxford
University Press.
Glick, H., Doshi, J.A., Sonnad, S.S., and Polsky, D. (2007). Economic Evaluation in Clinical
Trials. Oxford University Press.
Gold, M.R., Siegel, J.E., Russell, L.B., and Weinstein, M.C. (1996). Cost-Effectiveness in Health
and Medicine. Oxford University Press, New York.
Kassirer, J.P. and Angell, M. (1994). The journal’s policy on cost-effectiveness analyses
[editorial]. New Engand Journal of Medicine, 331, 669–70.
McIntosh, E., Louviere, J.J., Frew, E., and Clarke, P.M. (2010). Applied Methods of Cost–Benefit
Analysis in Health Care. Oxford University Press.
NICE (2004). Guide to the Methods of Technology Appraisal. National Institute for Health and
Clinical Excellence, London.
NICE (2008). Guide to the Methods of Technology Appraisal. National Institute for Health and
Clinical Excellence, London.
Ontario Ministry of Health (1994). Ontario Guidelines for Economic Analysis of Pharmaceutical
Products. Ontario Ministry of Health, Toronto.
Pharmaceutical Benefits Advisory Committee (2008). Guidelines for Preparing Submissions to
the Pharmaceutical Benefits Advisory Committee (Version 4.3). Australian Government,
Department of Health and Ageing, Canberra.
Philips, Z., Bojke, L., Sculpher, M., Claxton, K., and Golder, S. (2006). Good practice guidelines
for decision-analytic modelling in health technology assessment: a review and consolidation
of quality assessment. PharmacoEconomics, 24, 355–71.
Robbins, L. (1932). An Essay on the Nature and Significance of Economic Science. Macmillan,
London.
Chapter 2
Charming Juveniles