Session: Trials in Rare Conditions
This session will address challenges in developing and testing
therapies when there are a limited number of patients available to
enter trials.
Aims
- review real scenarios where solutions to the problem of small
patient numbers have been successfully implemented
- review cases of unsuccessful implementation but where
important lessons can be learned
- discuss issues arising post-trials, particularly collecting
and assessing safety data as well as further efficacy data
Oral Presentations
Presentations will be posted with permission from the
speakers.