Table of Contents

Introduction
Methods in Evidence Based Healthcare and Health Technology Assessment - An Overview
PART ONE: CLINICAL TRIALS
Introduction - Richard J Lilford and Andrew Stevens
Ethical Issues in the Design and Conduct of Randomized Controlled Trials - Richard J Lilford, Sarah Edwards, David Braunholtz, Jennifer Jackson, Jim Thornton and Jenny Hewison
Ethics of Clinical Trials - Richard E Ashcroft
Social, Cultural and Economic Factors
Factors That Limit the Number, Progress and Quality of Randomized Controlled Trials - Sue Ross, Carl Counsell, William J Gillespie, Adrian Grant, Robin Prescrott, Ian Russell, Iain Colthart, Sandra Kiauka, Daphne Russell and Sue Shepard
A Systematic Review
Results of Clinical Trials and Systematic Reviews - Dianne O′Connell, Paul P Glasziou, Suzanne Hill, Jasminka Sarunac, Julia Lowe and David Henry
To Whom Do They Apply?
The Placebo Effect - Rosemary Crow, Heather Gage, Sarah E Hampson, Jo Hart, Alan C Kimber and Hilary Thomas
Methodological Process and Implications of a Structured Review
PART TWO: OBSERVATIONAL AND QUALITATIVE METHODS
Introduction - Ray Fitzpatrick
Randomized and Non-Randomized Studies - Colin Sanderson, Martin McKee, Annie Britton, Nicholas Black, Klim McPherson and Chris Bain
Threats to Internal and External Validity
A Review of Observational, Quasi-Experimental and Randomized Study Designs for the Evaluation of the Effectiveness of Healthcare Interventions - Barnaby Reeves, Rachel MacLehose, Ian Harvey, Trevor Sheldon, Ian Russell and Andrew McLaren Spiers Black
The Potential Use of Routine Data Sets in Health Technology Assessment - James Raftery, Andrew Stevens and Paul Roderick
Using Routine Data to Complement and Enhance the Results of Randomized Controlled Trials - James Lewsey, Gordon Murray, Alastair H Leyland and Andrew Boddy
Qualitative Methods in Health Technology Assessment - Elizabeth Murphy and Robert Dingwall
PART THREE: MEASUREMENT OF BENEFIT AND COST
Introduction - John Brazier
Criteria for Assessing Patient-Based Outcome Measures for Use in Clinical Trials - Ray Fitzpatrick, Claire Davey, Martin Buxton and David Jones
The Use of Health Status Measures in Economic Evaluation - John Brazier, Mark Deverill and Colin Green
Collecting Resource Use Data for Costing in Clinical Trials - Katharine Johnstone, Martin Buxton, David Jones and Ray Fitzpatrick
Eliciting Time Preferences for Health - John Cairns and Marjon van der Pol
The Conduct and Design of Questionnaire Surveys in Healthcare Research - Elaine McColl, Ann Jacoby, Lois Thomas, Jennifer Soutter, Claire Bamford, Nick Steen, Roger Thomas, Emma Harvey, Andrew Garratt and John Bond
PART FOUR: ANALYTICAL METHODS
Introduction - Keith R Abrams
Bayesian Methods - David Spiegelhalter, Jonathan Myles, David Jones and Keith Abrams
Methods for Evaluating Organization or Area-Based Health Interventions - Martin Gulliford, Obioha C Ukoummunne, Susan Chinn, Jonathan Sterne, Peter Burney and Allan Donner
Handling Uncertainty in Economic Evaluation - Andrew Briggs and Alastair Gray
A Review of the Use of the Main Quality of Life Measures, and Sample Size Determination for Quality of Life Measures, Particularly in Cancer Trials - Michael Campbell, Steven A Julious, Sarah Walker, Steve George and David Machin
Simultaneous Assessment of Quality of Life and Survival Data - Lucinda Billingham, Keith Abrams and David Jones
PART FIVE: CONSENSUS, REVIEWS AND META-ANALYSIS
Introduction - Andrew Stevens and Keith R Abrams
Publication and Related Biases - Fuijan Song, Alison Eastwood, Simon Gilbody, Lelia Buley and Alex Sutton
Meta-Analysis in Health Technology Assessment - Alexander Sutton, David Jones, Keith Abrams, Trevor Sheldon and Fujian Song
Assessing the Quality of Reports of Randomized Trials Included in Meta-Analyses - David Moher, Terry Klassen, Alison Jones Ba′Pham, Deborah Cook, Alejandro Jada, Peter Tugwell and Michael Moher
Attitudes, Practice, Evidence and Guides
Consensus Development Methods, and Their Use in Creating Clinical Guidelines - Nick Black, Maggie Murphy, Donna Lamping, Martin McKee, Colin Sanderson, Janet Askham and Theresa Marteau
PART SIX: IDENTIFYING AND FILLING GAPS IN THE EVIDENCE
Introduction - Andrew Stevens
Identifying New Health Care Technologies - Glenn Robert, Andrew Stevens and John Gabbay
Timing of Assessment of Fast-Changing Health Technologies - Graham Mowatt, Adrian Grant, D Jane Bower, John A Brebner, John Cairns and Lorna McKee
Preliminary Economic Evaluation of Health Technologies - Gareth Harper, Joy Townsend and Martin Buxton

About the Author

Andrew Stevens is Professor of Public Health and former Head of Department and Division (of Primary Care, Public and Occupational Health).

Andrew is interested in Health Services Research including health care needs assessment, health technology assessment and horizon scanning. He has edited the 4 volume Health Care Needs Assessment Series, and the Advanced Handbook of Methods in Evidence Based Healthcare.

Andrew has had a close involvement with the National Institute for Health and Clinical Excellence (NICE) since its establishment in 2000, and has been Chairman of one of its Appraisal Committees for 6 years.
Porf. Adrams′ research includes: The development and application of Bayesian methods in healthcare evaluation, systematic reviews and meta-analysis and The joint modelling of longitudinal and time-to-event data. He has considerable experience of undertaking pradmatic TCT′s and systematic reviews in a variety of settings, including cardiology, gastroenology, oncology and reproductive medicine. John Brazier is Professor of Health Economics at the Health Economics and Decision Science section of the School of Health and Related Research at the University of Sheffield. He has more than 20 years’ experience of conducting economic evaluations of health care interventions for policy makers. He has also undertaken numerous economic evaluations alongside clinical trials and decision analytic models. He has a particular interest in the measurement and valuation of health for economic evaluation where he has published widely. He is perhaps best known for his work in developing a preference-based measure of health for the SF-36, but more recently has extended these methods to a number of specific condition including measures in asthma, cancer, overactive bladder, dementia and epilepsy. More recently he has been developing ways to incorporate equity concerns such as burden of disease into the weights applied to QALYs for Value Based Pricing. He is Director of an Economic Evaluation Policy Research Unit (EEPRU) that is funded by the Department of Health in England. He was a member of the National Institute for Clinical Excellence (NICE) Technology Appraisal Committee from 2000-2004 and is a member of the Working Group on revising Methods of Economic Evaluation for Technology Appraisal in 2012.

Reviews

`As books go, this is a real heavyweight. It is physically big, with 27 chapters running to over 500 pages. It is expensive, coming in at £69. Yet it is heavyweight in another sense. Some of the chapter authors are heavyweights, leaders, in their fields and this shines through veryc learly in the text. This book aims to provide readers with an insight into some of the most advanced methods involved in evidence based healthcare. Whey we see the word "advanced" we sometimes worry about things being set out in complex, convoluted, over-your-head, impenetrable, coded and cluttered language. Happily, most of this book is clear, comprehensive and concise. A few of the chapters are more challenging to read but they are certainly worth the extra effort. The book has six sections covering 27 chapters… Inside each section lies between three-to-five chapters. Each chapter is clearly laid out and referenced. While it is a methods book, the chapter authors sprinkle a good dose of real examples through the text to reinforce some of their points. This certainly helps to keep the writing fresh and engaging. The book is essentially derived from a large collection of UK NHS Health Technology Assessment (HTA) reports, which are available free of charge from the HTA program. If you want a lot of material in one cover, and if you want to make real progress in understanding some of the latest developments and thinking in evidence based healthcare, this is the only book to buy′ -British Journal of Clinical Governance ′Among the many features of the UK National Health Services Research & Development (NHS R&D) Programme that deserve admiration is its commitment to systematically evaluating which methods to use to obtain the best possible evidence for healthcare decisions. Reviews of a wide range of methodological issues relevant to health technology assessment have been commissioned by the NHS R&D Programme. Andrew Stevens and Ray Fitzpatrick have done an admirable job organising and editing this wealth of information. The Advanced Handbook of Methods in Evidence Based Health Care provides a unique and valuable reference for anyone wanting to make well informed decisions about methods of evaluating healthcare.′ Dr Andy Oxman, Director Health Services Research Unit National Institute of Public Health Postboks 4404 Torshov 0403 Oslo NORWAY ′This book promises to become a standard work of reference for all those interested in the methods of HTA and their application. Each topic is introduced seductively through successive levels of detail, so that there is material here for a variety of readers having a variety of interests, from those who want merely to capture some of the jargon to those at the leading edge of methodological development. As a would-be multi-disciplinarian, I found enlightenment almost everywhere.′ Professor Tony Culyer, Professor of Economics and Head of the Department of Economics and Related Studies, University of York ′This handbook is an excellent reflection of the growing maturity and methodological sophistication of the field of Health Technology Assessment. The handbook covers a spectrum of issues, from primary evidence (clinical trials) through reviews and meta-analysis, to identifying and filling gaps in the evidence. Up-to-date, clearly written, and well-edited, the handbook is a needed addition to any personal or professional library dealing with Health Technology Assessment.′ Professor David Banta, TNO Prevention and Health, The Netherlands ′It is very heartening to see so much of the methodological work that the HTA programme has commissioned brought together in one volume, a volume that is technical and authoritative but also readable and accessible. As NICE gets into full swing as the key national body with responsibility for promoting clinical excellence and cost-effective care within the NHS, it is particularly timely that these groundbreaking pieces of work should be published. Clearly much remains to be done. Nevertheless, this handbook will set new standards for those undertaking health technology assessments as well as map out the next generation of methodological challenges.′ Dr Ruairidh Milne, Scientific Director, National Co-ordinating Centre for Health Technology Assessment ′Health care research is expanding rapidly, aided by the fruitful interaction of a range of academic disciplines. Since its inception in 1993, the NHS Health Technology Assessment Programme has had two distinctive themes, a multi-disciplinary approach and a strategy to develop the methodological basis for HTA. This timely volume brings together a wide range of work commissioned by the Programme on methodology. Contributions from a diverse group of active health service researchers - statisticians, health economists, clinicians, sociologists and others - give a wide ranging overview of assessment strategies in health care. The scope includes quantitative and qualitative methods, randomised and non-randomised designs and the measurement of costs and benefits in health economic analysis. A number of active themes in statistical analysis, research synthesis and horizon scanning are considered. The volume will be of value to researchers wishing to keep abreast of methodological developments across the wide field of health care. For those who plan, provide or use health services it will provide a unique insight into the development of the knowledge base upon which modern health care rests.′ Professor Kent Woods, Director NHS HTA Programme ′The impact of clinical trials on health policy development is almost as great as the impact on clinical practice. Around the world many agencies are now struggling to make the best use of data from clinical trials, experimental studies and observational studies in order to make the best decisions about what technology to subsidise and at what price. This book covers a very wide field of technical issues regarding the appropriate use of data in health technology assessment. Undoubtedly it will become a major resource for everyone working in this field.′ Professor David Henry, Professor of Clinical Pharmacology, Head of Discipline of Clinical Pharmacology, Faculty of Medicine and Health Sciences, the University of Newcastle, Australia ′This text presents the most advanced knowledge on methodology in health care research, and will form the backbone of many future studies′ - Paula Roberts, Nurse Researcher