Abstract / Summary
Abstract Background Meaningful patient engagement requires understanding lived disease and healthcare experiences. This patient-centric approach is especially valuable when applied to the design and execution of clinical trials, as participation barriers may hinder trial recruitment, retention, and detection of meaningful outcomes. Patient engagement is particularly important in the study of treatments for rare diseases, where populations are small and geographically dispersed and there are few sites with relevant clinical expertise. Additionally, protocol amendments to address hurdles are expensive and can cause delays in trial activities, which can affect downstream regulatory approvals and access to treatments. Alexion, AstraZeneca Rare Disease, worked with patients and their stakeholders to identify points of additional trial burden and enable patient-driven changes before clinical trial protocol finalization. Methods Patients, parents of pediatric patients, and patient advocacy group representatives with diverse experiences participated in virtual, study-specific perspective exchange sessions on 4 protocols (adults with generalized myasthenia gravis, pediatric patients with generalized myasthenia gravis, adults with antibody-mediated rejection after kidney transplantation, and adults with transthyretin amyloid cardiomyopathy). Participants discussed their disease journey, the protocol, and perceived barriers to clinical trial awareness, enrollment, participation, and follow-up through semistructured interviews. Results Participants raised points about the eligibility criteria, study drug self-administration, visit frequency, participant mental health, and the relevance of specific outcome measures. In response, the study sponsor proactively changed the clinical trial protocols when feasible. Changes included expanding the eligibility criteria, developing an instructional video on self-administration of the study drug, and reducing the frequency of clinic visits. In addition, the sponsor created appreciation cards and trained trial sites on a standardized approach to help participants understand the goals of specific outcome measures. Conclusions These insights led to demonstrable, advocate-recommended changes in rare disease clinical trial protocols before study delivery, underscoring how early patient-partnered drug development can be meaningfully applied to research in rare diseases. Trial registration NCT05556096, NCT06607627, NCT06744647, and NCT06183931. Clinical trial number Not applicable.