Rare disease clinical trials face unique challenges, such as the low prevalence of disease and few qualified investigators, resulting in higher risks to clinical development programmes for sponsors. In this whitepaper, experts share insights on mitigating the pitfalls of rare disease clinical trials, including the following:

• Recognition that motivation alone may not enable participants to “cross the finish line.”

• Identifying trial participation pressures as potential risks to trial conduct efficiency

• Engaging with patient communities early to manage risks effectively

• Addressing hurdles during the trial participation journey

• Pressures faced by patients during the trial and the impact of participation support services