Clinical Research after Drug Approval: What is Needed and What is Not
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Drug Infunnuriorr Journal. Vol. 33. pp. 6 2 7 4 3 4 , 1999 Printed in the USA. All rights reserved.
CLINICAL RESEARCH AFTER DRUG APPROVAL: WHAT IS NEEDED AND WHAT IS NOT GENEVIEVE DECOSTER I;. Hoffmann-La Koche Ltd, Basel, Switzerland
MARCB u ~ s e International Institute for Drug Development, Brussels, Belgium
This paper delineates the purposes of clinical studies perjiormed with marketed drugs. often called “Phase IV” or “p~~stresistration ” trials. The purposes of these trials are outlined, some design issues specific to them are discussed, and the appropriateness of a full implementation of good clinical practice standards in the postregistration setting is questioned. Key Words: Phase IV studies; Postregistration studies: Clinical trial design; Good clinical
practice
INTRODUCTION PHASE IV CLINICAL STUDIES are usually defined as those performed with drugs that have been granted marketing authorization ( 1 4 ) . The term “Phase IV” is fairly standard and covers the vast majority of postregistration clinical study programs. Phase IV studies are also referred to as “marketing studies” or “experience studies” to emphasize that they are conducted once the drug is marketed, rather than prior to its approval by the regulatory authorities. Some other terms, such as “seeding trials” (5) or “observational studies” (6), have also been used, but they usually denote efforts made by marketing departments to encourage physicians to prescribe the new drug, rather than proper trials which are the focus of this paper. Phase IV
Keprint address: Marc Buyse. ScD, International Institute for Drug Development (ID’). 430 avenue Louise, 1050 Brussels. Belgium.
studies are sometimes confused with postmarketing surveillance, the process of monitoring the safety of a marketed drug (7,8). Phase IV studies are, in fact, part of this process, but their objectives include efficacy or effectiveness in addition to safety (9,lO). The distinction between Phase I11 and Phase IV studies is not always clear-cut. Many cooperative groups performing clinical trials in cardiovascular disease, cancer, or AIDS call their comparative studies “Phase 111” trials regardless of regulatory approval. Seriously compromised patients (eg, with AIDS or advanced cancer) can sometimes get access to a product prior to its approval through “expanded access,” “compassionate need,” or “treatment IND” programs and in some European countries through special license sales authorized by the health authorities. It has been argued that these programs should be an opportunity to perform largescale clinical studies addressing a question of scientific interest (11). Such studies, even though they take place before the approval
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Genevieve Dtwnrer and Marc Buyse
of the new drug, would share many characteristics with Phase IV studies. In these situations of life-threatening diseases, new drugs may also go through an “accelerated approval” process in which the new drug is marketed based on limited data and conditional on further evidence being provided in well-cont
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