Efficient trial design for new cancer therapies

The clinical trials that provide evidence for new cancer treatments often fall sort of providing adequate evidence upon which to base a decision about reimbursement by healthcare providers. There is the potential to improve the design of trials so that they provide better information about the value of new treatments. Three examples in early breast cancer are presented that demonstrate the role of decision modelling in planning research for the evaluation of cost-effectiveness. The results show that decision modelling at this stage can make a useful contribution to the trial design process in terms of the prioritisation of research design options and the suggestion of efficient sample sizes. Bayesian decision theory and value of information analysis are particularly helpful in the interpretation of the model. There may also be a role in aiding real-time trial adaptation where alteration of a trial design can improve the efficiency of research. There are challenges in the conduct of evidence synthesis and modelling prior to a phase III trial. There are also difficulties related to the prediction of a technology's useful lifetime and its future price and the market within which it will be available. Further research is required if early modelling is to be a robust basis for public or commercial research investment decisions. JEThOS - Electronic Theses Online ServiceGBUnited Kingdo