Today, while rare diseases are still defined by their scarcity, pharmaceutical companies are now incentivized to pursue treatments for these rare conditions. In fact, the rare disease market has grown past $150B, and more and more companies are investing resources into clinical research.
However, despite improved ROI, rare disease research is not without its obstacles. One word that defines each rare disease and its patient population would be “unique.” Companies that are new to the industry, and even those that have held rare disease clinical trials and developed treatments, need to continually grasp the nuance of symptoms, recruitment, clinical endpoints, and treatment prior to moving forward in designing clinical trial protocols.
Following are some common challenges expected during the course of researching rare diseases that must be addressed before progressing to clinical trials.