Future Medicine: Future Rare Diseases

What is a ‘negative’ trial? The term itself is very poorly defined and has an obvious undesirable connotation. It raises the specter of failure and suggests that the treatment being evaluated is not effective for that disease. It does not take into consideration the various caveats that may accompany the trial. Indeed, it is an all-encompassing term that is absolute. When so applied, or rather misapplied, it does a disservice to trial participants, other patients with that disease, their caregivers, their healthcare providers, the trial investigators and the sponsors. In short, a ‘negative’ trial impacts everyone who is engaged in finding new and better treatments for that particular disease. So, who benefits from using this terminology? Essentially, only the academic purists who characterize the results in preconceived binary pigeonholes with little to no consideration for the context of the trial or the impact on patients and caregivers.