Clinical trials for rare disease therapies pose unique challenges. Rare disease studies tend to be complex, due to a lack of historical data, well-defined clinical endpoints and available comparators. Rare conditions, inherently, mean that the pool of potential study candidates will be small which causes challenges to recruitment, as does the fact that these participants are often managing debilitating conditions. All of this increases pressure on the study team to keep enrolled participants engaged and to try to prevent dropouts.