Edited by DIUg M.N.G. Dukes Utilization Studies Methods and Uses m WHO Regional Publications European Series No. 45 The World Health Organization is a specialized agency of the United Nations with primary responsibility for international health matters and public health. Through this Organization, which was created in 1948, the health professions of over 180 countries exchange their knowledge and experience with the aim of making possible the attainment by all citizens of the world by the year 2000 of a level of health that will permit them to lead a socially and economically productive life. The WHO Regional Office for Europe is one of six regional offices throughout the world, each with its own programme geared to the particular health problems of the countries it serves. The European Region - embracing 850 million people living in an area stretching from Greenland in the north and the Mediterranean in the south to the Pacific shores of Russia - is unique in that a large proportion of its countries are industrialized, with advanced medical services. The European programme therefore differs from those of other regions in concentrating on the problems associated with industrial society. In its strategy for attaining the goal of "health for all by the year 2000" the Regional Office is arranging its activities in three main areas: promotion of lifestyles conducive to health, reduction of preventable conditions, and provi- sion of care that is adequate, accessible and acceptable to all. The Region is also characterized by the large number of languages spoken by its peoples and the resulting difficulties in disseminating information to all who may need it. The Regional Office publishes in four languages - English, French, German and Russian - and applications for rights of translation into other languages are most welcome. Drug utilization studies Methods and uses WHO Library Cataloguing in Publication Data Drug utilization studies : methods and uses / edited by M. N. G. Dukes (WHO regional publications. European series ; No. 45) 1. Drug utilization 2.Research I.Dukes, M. N. G. II.Series ISBN 92 890 1308 7 ISSN 0378 -2255 (NLM Classification: WB 330) World Health Organizationif Regional Office for Europe //I CopenhagenJ Drug utilization studies Methods and uses Edited by M.N.G. Dukes Former Regional Officer for Pharmaceuticals WHO Regional Office for Europe Copenhagen, Denmark WHO Regional Publications, European Series, No. 45 ICP/DSE 127 Text editing by Pamela M. Charlton ISBN 92 890 1308 7 ISSN 0378 -2255 The Regional Office for Europe of the World Health Organization welcomes requests for permission to reproduce or translate its publications, in part or in full. Applications and enquiries should be addressed to the Publications unit, WHO Regional Office for Europe, Scherfigsvej 8, DK -2100 Copenhagen 0, Denmark, which will be glad to provide the latest information on any changes made to the text, plans for new editions, and reprints and translations already available. © World Health Organization 1993 Publications of the World Health Organization enjoy copyright protection in accordance with the provisions of Protocol 2 of the Universal Copyright Convention. All rights reserved. The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of the Secretariat of the World Health Organization concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. The names of countries or areas used in this publication are those that obtained at the time the original language edition of the book was prepared. The mention of specific companies or of certain manufacturers' products does not imply that they are endorsed or recommended by the World Health Organization in preference to others of a similar nature that are not mentioned. Errors and omissions excepted, the names of proprietary products are dis- tinguished by initial capital letters. The views expressed in this publication are those of the contributors and do not necessarily represent the decisions or the stated policy of the World Health Organization. PRINTED IN FINLAND CONTENTS Page Authors vii Introduction - M.N.G. Dukes 1 1.General background - J.R. Laporte, I. Baksaas & P.K.M. Lunde 5 2.From clinical trials to drug utilization studies - G. Tognoni & J.R. Laporte 23 3.Therapeutic formularies, essential drugs, drug utilization studies - G. Tognoni & P.K.M. Lunde 43 4.Descriptive tools and analysis - D. Capellà 55 5.The quantification of drug risks in practice - D. Lee & U. Bergman 79 6.The social aspects of drug use - F.M. Haaijer -Ruskamp & E. Hemminki 97 7.The economic aspects of drug use - F.M. Haaijer - Ruskamp & M.N.G. Dukes 125 8.Health authorities and drug utilization studies - F. Garcia Alonso, A.I. Scott, L. Stika & B. Westerholm 147 9.Drug utilization and the health professions - K.H. Kimbel 169 10. Consumers as instigators and users of drug utilization research - A. Herxheimer 177 11. Drug utilization and the teaching of rational drug use - J.R. Laporte & M.L. Orme 183 12. Drug utilization studies: their transferability between industrialized and developing countries - D. Lee, K. Balasubramaniam & H.M. Ali 193 v Authors Dr Hasan M. Ali Head, Department of Pharmacology, Faculty of Pharmacy, Univer- sity of Khartoum, Sudan Dr Ingebjorg Baksaas Grenseg. 65, 3700 Skien, Norway Dr Kumariah Balasubramaniam Pharmaceutical Adviser, International Organization of Consumer Unions, Regional Office for Asia and the Pacific, Penang, Malaysia Dr Ulf Bergman Department of Clinical Pharmacology, KarolinskaInstitute, Huddinge University Hospital, Sweden Dr Dolors Capellà Division of Clinical Pharmacology, Autonomous University of Barcelona, Spain Dr M.N.G. Dukes Unit for Drug Policy Studies, University of Groningen, Netherlands Dr Fernando Garcia Alonso Ministry of Health and Consumer Affairs, Madrid, Spain Dr F.M. Haaijer -Ruskamp Department of Health Sciences, University of Groningen, Nether- lands Dr Elina Hemminki Department of Public Health, University of Helsinki, Finland vii Dr Andrew Herxheimer Editor, Drugs and therapeutics bulletin, London, United Kingdom Dr Karl H. Kimbel Oevelgoenne 92c, 2000 Hamburg 52, Germany Dr Joan Ramon Laporte Division of Clinical Pharmacology, Autonomous University of Bar- celona, Spain Dr David Lee Estafeta el Dorado, Apartment 6 2873, Panama 6, Panama Professor P.K.M. Lunde Department of Pharmacotherapeutics, University of Oslo, Norway Professor M.L. Orme Department of Pharmacology and Therapeutics, University of Liver- pool, United Kingdom Dr A.I. Scott The National Drugs Advisory Board, Dublin, Ireland Dr Ludvik Stika State Institute for Drug Control, Prague, Czechoslovakia Professor G. Tognoni Mario Negri Institute of Pharmacological Research, Milan, Italy Dr Barbro Westerholm Apoteksbolaget, Stockholm, Sweden viii Introduction M.N.G. Dukes When introducing the concept of drug utilization studies to people who are not familiar with them, one is sometimes met with blank astonish- ment. Surely, the initial reaction goes, there is no shortage of statistics on the use of medicines already? Is it truly necessary to undertake so much work in order to obtain yet more? The outsider's first view of drug utilization research is understand- able. There is something unexpected - indeed absurd - in the fact that in a world that uses so many medicines on so large a scale the pattern of their use is often so poorly known. Indeed there is no shortage of figures, but they are rarely the sort of figures that are needed to understand and interpret what is going on. Global statistics on the trade in drugs are rarely helpful; calculations based on expenditure can be highly misleading; and even individual physicians may have a mis- taken idea as to their own prescribing pattern over a period of time, to say nothing of that of their colleagues. This is a field in which, for one reason or another, people working in the interests of public health have often found themselves labouring in the dark. Yet there are in fact some identifiable reasons why drug utilization has not always had the attention it deserves. It is worth recalling that it was the pharmaceutical industry that first found itself in need of figures reflecting drug usage. Companies needed them to identify fields of opportunity for research and develop- ment, but also to monitor their own progress in a competitive market. Pioneering market research in this field was undertaken in the United States before 1939, paving the way for IMS (Intercontinental Market- ing Statistics) which became a large and successful operation by documenting drug prescribing and sales in a wide range of countries, using continuous input from health professions and the market, and 1 making the results available in detail to industrial subscribers. Systems such as that operated by IMS could have served a broader interest as well, but as a rule the data they provided remained within the walls of industry. In part that was attributable to purely commercial factors; the data were costly to collect and analyse, and they could only be made available for a substantial fee. Yet even where public health authorities expressed willingness to pay the fee, they generally encountered a polite refusal;the polarization between the pharma- ceutical industry and the public health authorities, which was widest after 1970, had perhaps created too much mistrust to expect