‘Negative’ Clinical Trials in Rare Diseases and Beyond: Reclassification and Potential Solutions

Figures & data

Table 1. Summary of proposed reclassification of ‘negative’ trials.

Category		Trial characteristics
1	Negative	Well-designed, well-conducted trial with adequate enrollment, but no benefits of treatment in any end point
2	Underpowered	Well-conducted trial, but underpowered to determine whether or not there was any benefit of treatment under investigation
3	Inadequate	Poorly designed trial that cannot determine whether or not there was any benefit of treatment under investigation
4	Terminated or uninitiated	Trials that were terminated or not initiated for any reason
5	Justifiable	Well-designed, well-conducted trial with adequate enrollment, without treatment benefit in primary efficacy end point, but with benefit of treatment in at least one other end point