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Clinical trial design for the 21st century

Traditionally, trials are split into three phases (I, II and III), with the trials being stopped and data analysed before starting againNick James with the next phase. This paper describes a new paradigm for trial design in the 21st Century, involving the testing of multiple drugs simultaneously using multi-arm, multi-stage trials.

It will describe the prototype trial, STAMPEDE, which is testing multiple agents in newly diagnosed prostate cancer, and involving substantial methodological innovation that has resulted in the collection of 8 phase 3 trials, with a preceding phase 2 for each one, producing effectively 16 trials involving over 6,000 patients, and making it the largest interventional study in prostate cancer.

It will be suggested that this advance on the traditional trial design permits continually evolving new questions to be addressed, linked translational science to identify the key underlying DNA abnormalities predicting benefit or lack thereof, and reduced cost associated with the development of new drugs.

Nick James

Professor James is Director of the University of Warwick Cancer Research Centre and Consultant in Clinical Oncology at the Queen Elizabeth Hospital Birmingham. He qualified in 1983 from St Bartholomew’s with the principal class medical prize and a First in Immunology. He undertook postgraduate training in London, Brussels and Tokyo.

He co-founded the leading website CancerHelp UK in 1994. He led the first UK trial of gene therapy in prostate cancer and is chief investigator on the STAMPEDE trial, the largest trial ever carried out in prostate cancer with over 6,000 participants.

In addition, he has led a series of trials of chemoradiotherapy for bladder cancer culminating in the BC2001 trial, published in the New England Journal of Medicine 2012. This trial has led to a change in thinking about bladder preservation for patients with locally advanced disease as an alternative to surgery.