“This is an exciting time to be involved in clinical trial design”

At the recent stakeholder workshop, it was clear that EU-PEARL is focussed on ensuring the clinical trials of the future are going to be fit for purpose for all stakeholders to draw robust conclusions from them.

This is an exciting time to be involved in clinical trial design; understanding what the benefits and risks of newer approaches are will help them gain acceptability in the standard canon of approaches that can be taken.

One of the great strengths of the project is the multi-disciplinary approach – bringing clinicians, regulators and of course patients closer together to try and ensure such designs are acceptable to all. No patient wants to be in a trial that they feel may not benefit them or future patients, and the need to focus on measuring outcomes that are important to patients is clear. Similarly, no patient would want to be in a trial that ultimately could not answer the scientific question of interest, either because it is too small to show an effect , or because it measures something not known to actually benefit patients. And nobody wants trials that need to recruit many more patients unnecessarily because of poor design, delaying the time to market for safe and effective treatments. Finding that ‘sweet spot’ is a challenge we should all be interested in rising to, and EU-PEARL is well positioned to help do this.

Another key aspect is the inclusion and engagement of diverse therapeutic areas, outside of oncology where such trials are better known. This is key to ensuring the results of the project are generalisable to as many different therapeutic areas as possible at its conclusion. It is of course hoped that actual real sustainable platforms will be built from this project but the real strength may well be in the blueprint for the future, to allow such platforms to be set up much more efficiently in the future.

Andrew Thomson is a statistician at the European Medicines Agency where he works supporting all aspects of drug licensure. Before that, he was at the UK regulator, the MHRA, initially as a statistical assessor and latterly as Head of Epidemiology.

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