‘Negative’ clinical trials in rare diseases and beyond: reclassification and potential solutions

Summary

Currently, categorizing clinical trials that do not demonstrate a statistically significant benefit as ‘negative’ is based solely on the main outcome of the trial. This issue, which is particularly important in rare diseases, does not account for whether there are other outcomes that could be positive for patients or other reasons for this result.

In this Perspective article, published ahead of print in one of our sister journals, Future Rare Diseases, Mukund Nori (rareLife solutions, CT, USA) proposes reclassifying ‘negative’ trials into five new categories, each of which more accurately describes the outcomes.