Health Technology Assessment 1998; Vol. 2: No. 14 Review Evaluating patient-based outcome measures for use in clinical trials Ray Fitzpatrick Claire Davey Martin J Buxton David R Jones Health Technology Assessment NHS R&D HTA Programme HTA Standing Group on Health Technology Chair: Professor Sir Miles Irving, Professor of Surgery, University of Manchester, Hope Hospital, Salford † Dr Sheila Adam, Professor Howard Glennester, Mr Hugh Ross, Department of Health Professor of Social Science & Chief Executive, The United Bristol Professor Martin Buxton, Administration, London School of Healthcare NHS Trust † Professor of Economics, Brunel University † Economics & Political Science Professor Ian Russell, Professor Angela Coulter, Mr John H James, Department of Health, Sciences & Director, King’s Fund, London Chief Executive, Kensington, Chelsea & Clinical Evaluation, University of York Westminster Health Authority Professor Anthony Culyer, Professor Trevor Sheldon, Deputy Vice-Chancellor, University of York Professor Richard Lilford, Director, NHS Centre for Reviews & † Dissemination, University of York † Dr Peter Doyle, Regional Director, R&D, West Midlands Executive Director, Zeneca Ltd, Professor Michael Maisey, Professor Mike Smith, ACOST Committee on Medical Research Professor of Radiological Sciences, Director, The Research School † & Health UMDS, London of Medicine, University of Leeds Professor John Farndon, Dr Jeremy Metters, Dr Charles Swan, Professor of Surgery, University of Bristol † Deputy Chief Medical Officer, Consultant Gastroenterologist, † North Staffordshire Royal Infirmary Professor Charles Florey, Department of Health Department of Epidemiology & Mrs Gloria Oates, Dr John Tripp, Public Health, Ninewells Hospital & Chief Executive, Oldham NHS Trust Department of Child Health, Royal Devon & Exeter Healthcare NHS Trust † Medical School, University of Dundee † Dr George Poste, Professor John Gabbay, Chief Science & Technology Officer, Professor Tom Walley, Director, Wessex Institute for Health SmithKline Beecham † Department of Pharmacological Therapeutics, University of Liverpool † Research & Development † Professor Michael Rawlins, Professor Sir John Grimley Evans, Wolfson Unit of Clinical Pharmacology, Dr Julie Woodin, Department of Geriatric Medicine, University of Newcastle-upon-Tyne Chief Executive, Nottingham Health Authority † Radcliffe Infirmary, Oxford † Professor Martin Roland, Dr Tony Hope, Professor of General Practice, The Medical School, University of Oxford † University of Manchester † Current members HTA Commissioning Board Chair: Professor Charles Florey, Department of Epidemiology & Public Health, Ninewells Hospital & Medical School, University of Dundee † Professor Ian Russell, Professor Sir Miles Irving Dr Tim Peters, Department of Health, Sciences & (Programme Director), Professor of Reader in Medical Statistics, Department of Clinical Evaluation, University of York * Surgery, University of Manchester, Social Medicine, University of Bristol † † Dr Doug Altman, Hope Hospital, Salford Professor David Sackett, Director of ICRF/NHS Centre for Professor Alison Kitson, Centre for Evidence Based Medicine, † Statistics in Medicine, Oxford Director, Royal College of Oxford Mr Peter Bower, Nursing Institute † Professor Martin Severs, Independent Health Advisor, Professor Martin Knapp, Professor in Elderly Health Care, Newcastle-upon-Tyne † † Director, Personal Social Services Portsmouth University Ms Christine Clark, Research Unit, London School of Dr David Spiegelhalter, Honorary Research Pharmacist, Economics & Political Science MRC Biostatistics Unit, Institute of † Hope Hospital, Salford Public Health, Cambridge Professor David Cohen, Dr Donna Lamping, Senior Lecturer, Department of Public Dr Ala Szczepura, Professor of Health Economics, Health, London School of Hygiene & Director, Centre for Health Services Studies, University of Glamorgan Tropical Medicine † University of Warwick † Mr Barrie Dowdeswell, Chief Executive, Royal Victoria Infirmary, Professor Theresa Marteau, Professor Graham Watt, Department of General Practice, Newcastle-upon-Tyne Director, Psychology & Genetics Woodside Health Centre, Glasgow † Professor Martin Eccles, Research Group, UMDS, London Professor of Clinical Effectiveness, Professor Alan Maynard, Professor David Williams, Department of Clinical Engineering, University of Newcastle-upon-Tyne † Professor of Economics, University of York † University of Liverpool Dr Mike Gill, Professor Sally McIntyre, Director of Public Health and Health Policy, Dr Mark Williams, MRC Medical Sociology Unit, Glasgow Brent & Harrow Health Authority † Public Health Physician, Bristol Dr Jenny Hewison, Professor Jon Nicholl, Dr Jeremy Wyatt, Senior Lecturer, Department of Psychology, Director, Medical Care Research Unit, Senior Fellow, Health and Public Policy, † University of Leeds † University of Sheffield School of Public Policy, University College, † Dr Michael Horlington, Professor Gillian Parker, London Head of Corporate Licensing, Smith & Nuffield Professor of Community Care, * Previous Chair Nephew Group Research Centre University of Leicester † † Current members HTA How to obtain copies of this and other HTA Programme reports. 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Evaluating patient-based outcome measures for use in clinical trials Ray Fitzpatrick1 Claire Davey1 Martin J Buxton2 David R Jones3 1 Division of Public Health and Primary Health Care, University of Oxford 2 Health Economics Research Group, Brunel University 3 Department of Epidemiology and Public Health, University of Leicester Published October 1998 This report should be referenced as follows: Fitzpatrick R, Davey C, Buxton MJ, Jones DR. Evaluating patient-based outcome measures for use in clinical trials. Health Technol Assessment 1998; 2(14). Health Technology Assessment is indexed in Index Medicus/MEDLINE and Excerpta Medica/ EMBASE. Copies of the Executive Summaries are available from the NCCHTA web site (see overleaf). NHS R&D HTA Programme he overall aim of the NHS R&D Health Technology Assessment (HTA) programme is to T ensure that high-quality research information on the costs, effectiveness and broader impact of health technologies is produced in the most efficient way for those who use, manage and work in the NHS. Research is undertaken in those areas where the evidence will lead to the greatest benefits to patients, either through improved patient outcomes or the most efficient use of NHS resources. The Standing Group on Health Technology advises on national priorities for health technology assessment. Six advisory panels assist the Standing Group in identifying and prioritising projects. These priorities are then considered by the HTA Commissioning Board supported by the National Coordinating Centre for HTA (NCCHTA). This report is one of a series covering acute care, diagnostics and imaging, methodology, pharmaceuticals, population screening, and primary and community care. It was identified as a priority by the Methodology Panel and funded as project number 93/47/09. The views expressed in this publication are those of the authors and not necessarily those