Extending the clinical and economic evaluations of a randomised controlled trial: The IONA Study

In modern society people are concerned about their state of health and if they do unfortunately become ill they want the best possible treatment to be made available to them. In order to satisfy these demands new treatments have to be developed. This can be a long and expensive process. Before any new treatment can come to market it has to be proved to be both clinically effective and economically cost-effective. With limited health care resources the cost-effectiveness of treatments is becoming ever more relevant. In order to show whether a treatment is clinically effective a clinical trial is carried out and this is now usually accompanied by an economic evaluation, so that the cost effectiveness of the treatment can be assessed. When a clinical trial aimed at preventing clinical events is analysed, a time-to-first event analysis is often performed together with a cost-effectiveness analysis. These analyses do not always make the best use of the large amounts of patient information recorded during the clinical trial. Using the randomised controlled trial (RCT) the Impact Of Nicorandil in Angina (IONA) as an exemplar, ways in which the clinical and economic evaluations of clinical trials can be expanded are explored. There are three main parts of this thesis. Firstly, following a more detailed introduction in Chapter 1, in Chapters 2 and 3 the IONA Study is introduced and the main clinical results of the study are given. Secondly, in Chapters 4, 5 and 6 the fact that patients could suffer more than one clinical endpoint is considered. The models that can be used to incorporate the recurrent events are introduced and then applied to the data from the IONA Study. Following on from this, through the simulation of recurrent event data, the performance of the models under different known conditions is assessed. Thirdly, in Chapters 7 and 8 an introduction to health economics is given and following this the main results of the economic evaluation of the IONA Study are presented. Areas in which the results of the economic evaluation can be expanded are then investigated. Finally, in Chapter 9 there is a discussion of the work as a whole and areas where there would be the possibility of further work