Smarter trial designs in surgical oncology


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Jane Blazeby1
1University of Bristol, Bristol, UK

Abstract

The overall aim of this talk is to consider optimal designs for successful RCTs in surgical oncology. Specifically it will outline pragmatic trial designs and the need for pilot or feasibility work for efficient and effective trial conduct and delivery.

There are several funding streams for randomised controlled trials (RCTs) in surgical oncology and the key to success is to design high quality studies that answer questions of importance to patients and the NHS. Recent investments from the Royal College of Surgeons of England into surgical trials centres and from the MRC into Hubs for Trials Methodology Research combined with National Institute of Health Research and Cancer Research UK funding streams means that there are unprecedented opportunities for surgeons and trials methodologists to work together to design and conduct multi-centre pragmatic trials.

Key features of pragmatic trial design include: i) broad participant, surgeon and centre inclusion, so that results are generalisable; ii) evaluation of interventions with sufficient stability to warrant multi-centre assessment; and, iii) selection of outcome measures that are of importance to patients and the health service. These issues are unfamiliar to surgeons therefore feasibility and pilot work is recommended before or part of a main trial.

The strengths and weaknesses of external pilot RCTs, standalone feasibility studies and main trials with an integrated internal pilot and pre-agreed progression criteria will be presented. The value of feasibility and pilot work to optimise recruitment, develop surgical interventions and create a functional multi-disciplinary team to deliver the main trial will be considered with examples.