Rare Disease Studies: A Comfortable Patient Is A Retained Patient

By Sherry Hubbard-Bednasz

What is comfort anyway? Being home. Your own bed. Your mom’s homemade anything. In rare disease studies, those things are suspended. Get through the trial and then you can seek comfort. But does it have to be this way?

One could say the cards are stacked against rare disease studies. We often talk about the challenges of clinical trials in the frame of typical studies – drug development efforts intended to improve the condition of many. The key word is many. Rare diseases do not have numbers on their side. This is not to say typical studies are cake walks. There are no guarantees with human trials. And definitely not in the midst of a global pandemic.