Clinical trials in small populations

Methodological challenges and solutions, 30th November-1st December 2015, Royal Statistical Society, London

Well powered randomised controlled clinical trials are firmly established as the gold standard for testing the efficacy and safety of new medicines. However, it may be infeasible to conduct such trials in a reasonable time frame when few patients are available. This will be the case if a medicine is to treat a rare disease. It is estimated that 30 million of 500 million people in the EU have a rare disease, while rare cancers account for one-fifth of new cancer cases. The movement towards genetically tailored treatment regimens will further increase the number of small populations for whom new treatments are sought.

This two-day meeting will bring together researchers and practitioners to discuss state-of-the-art methods for trials in small populations. The meeting will be held at the Royal Statistical Society, London.


Day One

Groundwork Training Workshop will be an interactive training workshop featuring:

  • Discussion sessions motivated by case studies of real trials
  • Tutorials on methods for small trials
  • Hands-on practical sessions

Day Two

Forwards-Looking Forum will comprise invited talks from:

  • Medical statisticians from industry and academia
  • Patient representatives
  • Regulators

Scholarships will be available to assist with the travel expenses of a number of researchers who are within 10 years of starting their career. Preference will be given to those wishing to contribute either a case study to either the Groundwork Training Workshop or a poster to the Forwards-Looking Forum. To apply, please submit a brief CV and an abstract of your case study or poster to by 26th October

Organising Committee

The organising committee is

  • Lisa Hampson (Lancaster University)
  • Tim Morris (MRC Clinical Trials Unit at UCL)
  • Nigel Stallard (University of Warwick)
  • Matt Sydes (MRC Clinical Trials Unit at UCL)
  • Catrin Tudur Smith (University of Liverpool)
  • Sofia S. Villar (MRC Biostatistics Unit, Cambridge)