Getting the Most Out of Rare Disease Research

Chris Hall |

In rare disease research, every patient matters, and the quality of the data that they provide is of the utmost importance. Variations within the specific rare disease population and in patients’ disease progression make it difficult for trial sponsors to understand each patient’s disease experience and to select outcome measures. And, because half of patients with rare diseases in the world are children,1 trial sponsors must rely on parents, teachers and other caregivers to complete outcome assessments based on their observations, which are subject to variability, bias and inconsistencies that can inaccurately represent the treatment effect.

In her recent article, Optimizing Rare Disease Outcomes through eCOA Technology and Training, Susan Dallabrida, PhD, presents options for improving outcome assessments in rare disease trials. Her analysis indicates that electronic Clinical Outcome Assessments (eCOA) are the most effective method for capturing validated, consistent assessments to measure rare disease trial outcomes because of their proven ability to reduce missing data, increase event reporting, and improve reporting accuracy. That’s great news because eCOA systems are preferred over traditional paper-based methods by clinical trial patients.2 Pre-set alarms remind patients to complete assessments, which consistently drives more complete data collection than paper COA – and delivers more valid data for the trial sponsor’s evaluation.

It’s remarkable how technology is helping drive new drugs to market, but sponsors of rare disease clinical trials should also consider the important role patient, caregiver and observer training plays in improving data quality.

Training site raters and patient/caregivers on the assessment and its electronic implementation is vitally important in rare disease studies because inter-rater variability is high due to the widespread geographic dispersity of sites and patients. In addition, rare disease research often requires event and severity reporting by caregivers, who do not inherently understand how to complete assessments. Training caregivers – and all raters – on how to report on an Observer Reported Outcome (ObsRO) is a critical element to successful data capture in rare disease studies.

Training patients and caregivers also promotes compliance. More than 75% of patients report that the number one factor that would motivate them to complete a daily diary in a clinical trial is training on the importance of their role, what to expect in the study, and the purpose and importance of the questionnaires.3

By taking these steps, clinical trial sponsors can generate the data required for successful regulatory submissions.

Click here for additional research on the benefits of eCOA and rater training in clinical development.

Chris Hall is the Director of eCOA Product & Clinical Science Consulting