The Lysosomal Acid Lipase (LAL) Deficiency Registry is a research and disease management observational study that gathers patient data—including health history and treatments. Its purpose is to inform questions of interest from the LAL Deficiency healthcare provider community.
To support this registry, Alexion (formerly Synageva) required a high-quality service platform that is scalable upon product approval. The platform also needed to provide an excellent research experience for the healthcare providers who care for LAL Deficiency patients.
Alexion Pharmaceuticals chose DIFZ’s Observational Research Platform, N of 1 Health Research Platform (HRP), as a technology service solution.
This platform provides tools for healthcare providers to collect, monitor, and report on data and medical knowledge about the progression of LAL Deficiency and its outcomes. It offers:
- A fully integrated portal providing single sign-on access to data collection tools and analytical graphs and reports.
- Integration with Medidata’s RAVE, the leading clinical trial data collection application.
- Real-time data interchange using CDISC standards.
- Role-based disease management for patients, physicians, and nurses.
- Reporting functionalities for sites, registry staff, and Alexion’s regulatory reports.
DIFZ’s N of 1 HRP technology service solution now provides Alexion tools for healthcare providers to collect, monitor, and report on data and medical knowledge about the progression of LAL Deficiency and its outcomes.
DIFZ customized and configured its platform, using the data collection application and an integrated website portal as the foundation of the registry.
The LAL Deficiency Registry is designed to:
- Support regulatory filings.
- Capture a broader data set on untreated patients, which will mean a faster evaluation of treatment outcomes when treated patients are included.
- Create a longitudinal data set for evaluating all patients (treated and untreated) over the long term.
- Provide relevant data for scientific publications to improve evidence-based clinical practice.
- Create a central mechanism to continuously develop a scientific community in support of LAL Deficiency.