While there is a growing recognition that the world of rare disease is different and comes with unique challenges, the body of knowledge around how to address those challenges is very much a work in progress. At rareLife solutions, we’re helping to build that knowledge base by sharing what we’ve learned from our work with patients, advocates, caregivers, healthcare professionals, treatment industry leaders, and beyond. We share our thinking with the community here and via email. To be added to our email list and get future resources directly in your inbox, please get in touch.
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.
In the following video, author Mukund Nori shares highlights from his research findings: