Statistical methods in rare diseases and small populations

Clinical research in rare diseases and other small populations presents a range of statistical challenges as the large clinical trials that are generally used to evaluate new drugs and other healthcare interventions are infeasible or even impossible.  These challenges have necessitated the development of a range of new approaches to the statistical design and analysis of such clinical trials, particularly through international collaborations such as the International Rare Cancers Initiative (IRCI) and recent projects funded through the European Union’s Framework programme 7.

The mini-symposium on Statistical methods in rare diseases and small populations will focus on two areas of particular current activity and interest.  In the two morning sessions methodological and clinical experts will present recent examples and work on the design of clinical trials in rare cancers, in particular associated with the IRCI.  The morning will end with a panel discussion of the advantages and disadvantages of the different methodological approaches.

The afternoon session will include three talks by methodological experts considering the challenges associated with extrapolation between clinical trials between populations and in particular how the results of studies in adults can be used to inform the design and interpretation of clinical trials in paediatric populations.

Designs for Clinical Trials in Rare Cancers 
 09.30 Lucinda Billingham, Birmingham, UK
Overview of statistical issues in design of clinical trials in rare cancers and the International Rare Cancers Initiative (IRCI)
 09.45 Tim Morris, London, UK
How do you design trials in smaller populations? A framework
 10.15 Stephen Senn, Luxembourg
Approximate analysis of covariance in trials in rare diseases, in particular rare cancers
 10.45 Refreshments
 11.15 Jim Paul, Glasgow, UK 
Study Designs for Rare Cancers - The BALLAD Example
 11.45 Panel Discussion
 12.30 Lunch
  Statistical methods for extrapolation
 13.30 Lisa Hampson, Lancaster, UK 
Using historical data to inform extrapolation decisions in children
 14.00 Gerald Hlavin, Vienna, Austria 
An Extrapolation Framework to Specify Requirements for Drug Development in Children
 14.30 Sarah Zohar, Paris, France 
A unified approach for extrapolation and bridging adult information in early phase paediatric dose-finding studies
 15.00  Close