Researchers push for stronger patient partnerships in rare disease studies
A new study underscores the critical role of patient involvement in clinical trials, particularly for rare and ultra-rare diseases. The authors argue that including patients as active partners in the research process enhances trial design, recruitment, and overall outcomes.
“Engaging patients as partners in the research process leads to more relevant and effective clinical trials,” the researchers stated, emphasizing that traditional trial models often fail to address the unique challenges faced by those with rare diseases.
The study highlights several barriers to effective rare disease research, including small patient populations, geographic dispersion, and limited understanding of disease progression. By incorporating patient perspectives early in the trial design process, researchers can ensure that studies align with real-world patient needs, improving trial feasibility and participant retention.
The authors advocate for a paradigm shift in research, calling on scientists, clinicians, and policymakers to foster stronger collaborations with patient communities. They recommend strategies such as co-developing study protocols, involving patient advocacy groups, and utilizing digital platforms to facilitate remote participation.
“Patients bring invaluable insights into trial feasibility, meaningful endpoints, and the everyday impact of treatments,” the authors notes. “Their engagement is essential for developing therapies that truly improve quality of life.”
As rare disease research continues to evolve, this study serves as a call to action for greater patient-centric approaches, ensuring that clinical trials are designed with—not just for—the patients they aim to serve.
Reference
Cribbs KA, Blackmore LTA, Banks AR, et al. Capturing Real-World Rare Disease Patient Journeys: Are Current Methodologies Sufficient for Informed Healthcare Decisions? J Eval Clin Pract. 2025;31(1):e70010. doi: 10.1111/jep.70010. PMID: 39960234; PMCID: PMC11831961.
