Realizing The Full Value Of Patient Centricity In Rare Disease: 5 Ways To Maximize Your Engagement Efforts

Competition in an already difficult clinical research environment—where patient pools are inherently small and frequently dispersed geographically—increases as industry interest in rare diseases grows. A key component to having a successful clinical trial in this sector is making sure that the patient is engaged at every step of the study. If the patient’s voice is not incorporated throughout the drug development process, teams will ultimately risk losing the full value of patient centricity.

Gathering input from patients and family members on the specifics of the disease, the standard of care, the type and frequency of assessments, visit schedules, support needs, and even recruitment or retention strategies is essential for ensuring that the voice of the patient is incorporated into every aspect of clinical development. As a result of taking these considerations, sponsors are in a better position to develop the study in a patient-centric manner and design protocol parameters that lead to successful study enrollment and patient retention.

Ideally, patient engagement efforts should begin before the protocol is finalized so any feedback can be incorporated into the study design. However, if these efforts are underway before the study start-up, the input can still be implemented.

Sponsors looking to differentiate their trials and inspire patient interest should consider these five tips to proactively demonstrate their commitment to addressing unmet therapeutic needs.