Individual rare diseases affect a small percentage of the population, but collectively, rare diseases impact a significant number of patients and families and have profound impacts on their lives. For most patients with rare diseases, the journey to diagnosis is long and the options for treatment are limited. Clinical trials offer hope for addressing unmet needs, but these studies must be designed with the patient perspective in mind. Asking patients and caregivers about the challenges they face and the expectations they have of study participation is critical to clinical trial success.
In this guide, we discuss strategies for building relationships with patients and families early on to incorporate their feedback into study design and integrate their perspectives, priorities, and preferences into study execution. We also explore the intricacies of designing and executing truly patient-centric rare disease studies.
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