Hockey sticks and broken sticks - a design for a single-treatment, placebo controlled, double-blind, randomized clinical trial suitable for chronic diseases

Date Monday 7 May 2018
Time 4:10pm - 5pm
Where S.G.02
Presenter Hans Hockey, Biometrics Matters Ltd
Contact Steven Miller
Contact email
Admission Cost Free

This work is motivated and exemplified by a genetic disorder causing early onset diabetes, blindness and deafness, which is extremely rare, inevitably fatal and has no current direct treatment. While the standard two-arm, placebo-controlled RCT is the gold standard required by the regulatory agency for a new proposed drug study, it is conjectured that potential study participants will prefer a design which guarantees that they are always assigned to the drug under study.

A design is proposed which meets this patient need and hence probably increases recruitment and compliance. At the same time, it meets the requirement for full randomization. Analyses which follow naturally from this design are also described and were used in trial simulations, for sample sizing and for examination of the effect of underlying assumptions.

Note: this talk will include an introduction to clinical trials for those not familiar with the topic.

There will be nibbles and drinks in the G Block second floor tearoom following this talk.