From researchers to sponsors to patients to advocacy groups to clinicians, rare disease research is full of passionate, driven, and determined people. As the greatest asset to the field, all people involved must be utilized to their greatest potential for a study to be successful. That’s why significant attention must be paid to the operational challenges within rare disease research.
For studies in support of orphan drug designations, these operational concerns can be broken down into five major issues: patient identification, patient engagement, patient retention, investigative site selection, and site team support. Additionally, within rare disease, researchers frequently work with pediatric populations and older populations requiring caregivers, which adds yet another layer of complexity to the mix.