Case Study
Biometrics Services Accelerating clinical trials for rare diseases
The business challenge: Faster data review needed to meet urgent therapy demand With precision medicine, there is a unique sense of urgency to have patient data quickly available through the duration of a trial. This is especially the case for Sarepta Therapeutics, which develops therapies for a patient population with urgent unmet medical needs. Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness and primarily affects children. Duchenne studies are often long, following patients’ disease progression over many years. Each patient has a high volume or “avalanche” of data that is collected over the course of the treatment. Given that this is a very vulnerable patient population, it is critical for Sarepta to move quickly and rapidly analyze data to assess the safety and efficacy of its treatments. It is a common requirement from the medical team for data to be turned around within a week of a participant’s last appointment. Since the company focuses primarily on DMD, they frequently run similar studies and draw data from across these studies to build comprehensive views of patients’ experiences. In order to manage these trials effectively, Sarepta needed to work with technology and service partners who understood this sense of urgency and could meet their many data demands. This included the ability to create standard, reusable libraries for case report forms and edit checks that could be used across trials to save time during setup and to reduce build costs. To do this, Sarepta chose to have one place, in the form of a Rave EDC url, for all Sarepta studies, as this provided greater efficiencies and standardization. The team also required technology platforms that would allow them to access and review data from numerous sources in a centralized, precise and clean version of data that all stakeholders could work from for data review, exploration, and visualization.
■ Headquartered in Cambridge, MA ■ Specializes in gene therapies for Duchenne Muscular Distrophy ■ Aims to engineer precision genetic medicine for rare diseases
Products Utilized Biometrics Services elluminate Clinical Data Cloud
Success highlights 1. Rapid data cleaning and faster time to database lock 2. Instituted soft lock process and continuous data review: 95% of data is always reviewed 3. Optimized db builds with 93% reuse of forms and edit checks