NORD Rare Summit: rareLife posters

The annual National Organization for Rare Disorders Summit is one of the largest multi-stakeholder events in rare disease. It includes communities ranging from from patient advocacy groups and industry, to government and academia, to discuss  topics related to rare diseases and orphan products. The rareLife solutions team was selected to include several posters at this…

“Negative” Clinical Trials in Rare Diseases and Beyond: Reclassification and Potential Solutions

Currently, all trials, including those in rare diseases, that do not demonstrate a statistically significant benefit (i.e., P < 0.05) on the primary endpoint are classified as “negative”. This classification does not take into account a myriad of factors, including whether the trial was sufficiently powered, or had other statistically significant endpoints, or was terminated before completion, or was even initiated. This paper proposes reclassification of these trials into 5 categories: true negative, underpowered, inadequate, terminated or uninitiated, and valid. These categories reflect the trial characteristics more accurately and will be more useful to all stakeholders, especially the patients who participated in the trial and their healthcare providers.